| 1 |
NCT01733615 |
Terminated |
Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA |
- Mucopolysaccharidosis Type IVA
|
|
Observational |
|
- Emory University
- BioMarin Pharmaceutical
|
Other / Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Quantification of urinary oligosaccharides in urine from a first morning void in patients with Mucopolysaccharidosis IVA.
- Quantification of urinary oligosaccharides in urine from a random collection in patients with Mucopolysaccharidosis IVA.
|
3 |
All |
Child, Adult, Senior |
NCT01733615 |
IRB00058450 |
|
June 2012 |
February 2013 |
February 2013 |
November 27, 2012 |
September 4, 2014 |
|
- Emory University, Dept of Human Genetics
Decatur, Georgia, United States
|
| 2 |
NCT01238328 |
Unknown † |
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis |
|
- Drug: Conditioning regimen
- Drug: Graft-versus-host disease (GVHD) prophylaxis
- Procedure: Stem Cell Transplantation
|
Interventional |
Phase 2
Phase 3 |
- Tehran University of Medical Sciences
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)
- One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT)
- One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT)
- (and 2 more...)
|
10 |
All |
up to 8 Years (Child) |
NCT01238328 |
HORCSCT-0906 |
|
September 2009 |
September 2012 |
December 2012 |
November 10, 2010 |
June 4, 2012 |
|
- Hematology-Oncology & SCT Research Center
Teharn, Tehran, Iran, Islamic Republic of
|
| 3 |
NCT03488394 |
Not yet recruiting
New |
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant |
|
- Genetic: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
|
Interventional |
Phase 1
Phase 2 |
- IRCCS San Raffaele
- Fondazione Telethon
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall survival
- Achievement of haematological engraftment
- Safety of the administration of autologous haematopoietic stem cells transduced with IDUA LV - short term tolerability
- (and 11 more...)
|
6 |
All |
up to 11 Years (Child) |
NCT03488394 |
2017-002430-23 |
TigetT10_MPSIH |
April 2018 |
September 2020 |
September 2020 |
April 5, 2018 |
April 5, 2018 |
|
- Ospedale San Raffaele
Milano, Italy
|
| 4 |
NCT03161171 |
Completed |
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III |
- Mucopolysaccharidosis Type I
- Mucopolysaccharidosis Type II
- Mucopolysaccharidosis Type III
- (and 2 more...)
|
- Other: This is an observational study
|
Observational |
|
- University Hospital Heidelberg
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Coping strategies for challenging behavior and for family burden in mucopolysaccharidosis type I-III
- Quantity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
- Intensity of behavioral abnormalities in children with mucopolysaccharidosis type I-III
- Psychosocial burden in families with mucopolysaccharidosis type I-III
|
34 |
All |
up to 18 Years (Child, Adult) |
NCT03161171 |
01 |
|
July 6, 2017 |
November 13, 2017 |
November 13, 2017 |
May 19, 2017 |
December 13, 2017 |
|
- University of Heidelberg
Heidelberg, Germany
|
| 5 |
NCT03227042 |
Recruiting |
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) |
- Mucopolysaccharidosis Type IIIB
|
|
Observational |
|
|
Industry |
- Observational Model: Other
- Time Perspective: Prospective
|
- Neurocognitive function
- Behavioral function
- Quality of Life Tests
- (and 3 more...)
|
60 |
All |
up to 18 Years (Child, Adult) |
NCT03227042 |
250-902 |
|
November 16, 2017 |
July 31, 2022 |
November 15, 2022 |
July 24, 2017 |
November 22, 2017 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States - Murdoch Childrens Research Institute and Royal Children's Hospital
Melbourne, Victoria, Australia - Medical Genetics Service/HCPA, Department of Genetics/UFRGS
Pôrto Alegre, Rio Grande Do Sul, Brazil - (and 4 more...)
|
| 6 |
NCT03128593 |
Completed |
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II |
|
|
Interventional |
Phase 1
Phase 2 |
- JCR Pharmaceuticals Co., Ltd.
|
Industry |
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with Adverse Events
- Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
- Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
- (and 5 more...)
|
12 |
Male |
6 Years and older (Child, Adult, Senior) |
NCT03128593 |
JR-141-101 |
|
March 30, 2017 |
March 30, 2017 |
October 4, 2017 |
April 25, 2017 |
October 12, 2017 |
|
- Gifu Clinical site
Gifu, Japan - Fukuoka Clinical site
Kurume, Japan - Tokyo clinical site1
Minato, Japan - (and 5 more...)
|
| 7 |
NCT03173521 |
Recruiting |
Gene Therapy in Patients With Mucopolysaccharidosis Disease |
- Mucopolysaccharidosis Type VI
|
- Biological: AAV2/8.TBG.hARSB
|
Interventional |
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- safety, vital signs, laboratory tests and liver ultrasound. signs, laboratory tests and liver ultrasound.
- safety, laboratory tests and liver ultrasound.
- safety, laboratory test for kidney fuction
- (and 4 more...)
|
10 |
All |
4 Years to 65 Years (Child, Adult) |
NCT03173521 |
TIGEM1-MPS VI |
|
July 17, 2017 |
April 30, 2020 |
June 1, 2022 |
June 2, 2017 |
October 4, 2017 |
|
- Federico II University
Italy, Naples, Italy
|
| 8 |
NCT02437253 |
Completed
Has Results |
Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI |
- Mucopolysaccharidosis Type I
- Mucopolysaccharidosis Type II
- Mucopolysaccharidosis Type VI
|
- Drug: Adalimumab
- Other: Placebo
|
Interventional |
Phase 1
Phase 2 |
- Los Angeles Biomedical Research Institute
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score
- Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score
- Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)
- (and 2 more...)
|
2 |
All |
5 Years and older (Child, Adult, Senior) |
NCT02437253 |
30535 |
|
May 2015 |
July 2016 |
July 2016 |
May 7, 2015 |
September 15, 2017 |
September 15, 2017 |
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States
|
| 9 |
NCT01856218 |
Completed
Has Results |
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) |
- Mucopolysaccharidosis Type 7
|
|
Interventional |
Phase 1
Phase 2 |
- Ultragenyx Pharmaceutical Inc
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate
- Percentage Change From Baseline in uGAG Chondroitin Sulfate
- Number of Participants With Any ≥ 50% Decrease in uGAG
- (and 8 more...)
|
3 |
All |
5 Years to 30 Years (Child, Adult) |
NCT01856218 |
UX003-CL201 |
|
November 2013 |
July 2016 |
July 2016 |
May 17, 2013 |
March 13, 2018 |
January 5, 2018 |
- Manchester Academic Health Science Centre
Manchester, United Kingdom
|
| 10 |
NCT02037880 |
Completed |
Natural History Studies of Mucopolysaccharidosis III |
- Mucopolysaccharidosis Type IIIA
- Mucopolysaccharidosis Type IIIB
|
- Procedure: Lumbar puncture
- Device: Magnetic Resonance Imaging (MRI) of the brain
|
Observational |
|
- Nationwide Children's Hospital
- Sanfilippo Children's Research Foundation
- The Sanfilippo Research Foundation
- The Children's Medical Research Foundation
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Cognitive function
- Adaptive functioning
- Emotional/behavioral function
- White and grey matter brain volumes
|
25 |
All |
2 Years and older (Child, Adult, Senior) |
NCT02037880 |
MPSIII-1 |
|
February 2014 |
November 2015 |
November 2015 |
January 16, 2014 |
November 16, 2015 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 11 |
NCT01707433 |
Completed |
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease |
- Mucopolysaccharidosis IV A
- Mucopolysaccharidosis VI
|
|
Observational |
|
- Children's Hospitals and Clinics of Minnesota
- BioMarin Pharmaceutical
- Greenwood Genetic Center
- Gillette Children's Specialty Healthcare
|
Other / Industry |
- Observational Model: Cohort
|
- Subjects identified with MPS IVA or VI
|
22 |
All |
up to 21 Years (Child, Adult) |
NCT01707433 |
MPSHIP |
|
October 2012 |
June 2015 |
June 2015 |
October 16, 2012 |
August 4, 2015 |
|
- Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States - Gillette Children's Specialty Healthcare
St Paul, Minnesota, United States
|
| 12 |
NCT00144794 |
Recruiting |
Mucopolysaccharidosis I (MPS I) Registry |
- Mucopolysaccharidosis I (MPS I)
|
|
Observational |
|
- Genzyme, a Sanofi Company
- Sanofi
|
Industry |
- Observational Model: Cohort
- Time Perspective: Other
|
- To evaluate the long-term effectiveness of Aldurazyme
|
1500 |
All |
Child, Adult, Senior |
NCT00144794 |
MPS I Registry
DIREGC07008 |
|
November 20, 2003 |
December 31, 2020 |
December 31, 2020 |
September 5, 2005 |
April 12, 2018 |
|
- Investigational Site Number 840109
Little Rock, Arkansas, United States - Investigational Site Number 840108
Los Angeles, California, United States - Investigational Site Number 840042
Sacramento, California, United States - (and 111 more...)
|
| 13 |
NCT01675674 |
Terminated |
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics |
- Mucopolysaccharidoses
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
- (and 2 more...)
|
- Other: Dried blood spot test for MPS
|
Observational |
|
- National MPS Society
- MediResource Inc.
|
Other |
|
- Incidence of previously unrecognized MPS I, II, IVA, and VI in children presenting to pediatric rheumatology, hand, or skeletal dysplasia clinics
- Utility of DBS testing to screen for MPS in pediatric patients
|
3000 |
All |
6 Months to 18 Years (Child, Adult) |
NCT01675674 |
RHE-001 |
|
September 2011 |
March 2014 |
March 2014 |
August 30, 2012 |
May 24, 2013 |
|
- University of Medicine and Dentistry of New Jersey
New Brunswick, New Jersey, United States - Hospital for Special Surgery
New York, New York, United States
|
| 14 |
NCT03359213 |
Not yet recruiting |
A Study of JR-141 in Patients With Mucopolysaccharidosis II |
|
|
Interventional |
Phase 2 |
- JCR Pharmaceuticals Co., Ltd.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with Adverse Events
- Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
- Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
- (and 6 more...)
|
18 |
Male |
Child, Adult, Senior |
NCT03359213 |
JR-141-BR21 |
|
February 1, 2018 |
December 31, 2018 |
December 31, 2018 |
December 2, 2017 |
December 2, 2017 |
|
- Grupo de Pesquisa Clínica em Genética Médica - HCPA
Porto Alegre, Brazil - Igeim - Unifesp
São Paulo, Brazil
|
| 15 |
NCT00104234 |
Completed
Has Results |
Study of rhASB in Patients With Mucopolysaccharidosis VI |
|
- Drug: N-acetylgalactosamine 4-sulfatase
- Drug: Placebo/rhASB
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- 12-Minute Walk Test
- 3-Minute Stair Climb
- Change in Urinary Glycosaminoglycans (GAG) Level
|
39 |
All |
7 Years and older (Child, Adult, Senior) |
NCT00104234 |
ASB-03-06 |
|
February 2004 |
October 2006 |
October 2006 |
February 25, 2005 |
February 2, 2010 |
March 6, 2009 |
- BioMarin Pharmaceutical Inc.
Novato, California, United States
|
| 16 |
NCT00215527 |
Terminated |
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I |
- Mucopolysaccharidosis I
- Lysosomal Storage Diseases
- Spinal Cord Compression
|
|
Interventional |
Phase 1 |
- Patricia I. Dickson, M.D.
- The Ryan Foundation
- University of California, Los Angeles
- (and 2 more...)
|
Other / U.S. Fed |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
- improvement in spinal cord compression due to mucopolysaccharidosis I
|
4 |
All |
8 Years and older (Child, Adult, Senior) |
NCT00215527 |
MIRC-001
12069-01 |
|
November 2005 |
October 2011 |
October 2011 |
September 22, 2005 |
February 21, 2013 |
|
- Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )
Torrance, California, United States - Helsinki University Central Hospital
Helsinki, Finland
|
| 17 |
NCT03017677 |
Not yet recruiting |
A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis |
|
|
Observational |
|
- Mackay Memorial Hospital
- BioMarin Pharmaceutical
|
Other / Industry |
- Observational Model: Other
- Time Perspective: Prospective
|
- To evaluate the positive screening rate of MPS subject (I, II VI and IV) under ENT screening algorithm in Taiwan
- To evaluate the data collection of patient profile which include urine GAG data, disaccharides patterns, enzyme activity, genetic pattern and medical history of MPS (I, II VI and IV) patients.
|
100 |
All |
up to 20 Years (Child, Adult) |
NCT03017677 |
16MMHIS127 |
|
January 1, 2018 |
December 2020 |
December 2020 |
January 11, 2017 |
January 3, 2018 |
|
|
| 18 |
NCT03315182 |
Enrolling by invitation |
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB |
- Mucopolysaccharidosis Type 3 B
|
- Biological: rAAV9.CMV.hNAGLU
|
Interventional |
Phase 1
Phase 2 |
- Kevin Flanigan
- Abeona Therapeutics, Inc
- Nationwide Children's Hospital
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Determination of safety based on the development of unacceptable toxicity: defined as the occurrence of two or more unanticipated Grade III or higher treatment-related toxicity.
- Reduction from baseline values of glycosaminoglycans or their subunit, heparan sulfate, at 6 and/or 12 months after treatment, in any of the following: Cerebrospinal fluid, plasma or urine.
- Increase in CSF or plasma NAGLU enzyme activity levels at 6 and/or 12 months
- (and 4 more...)
|
9 |
All |
2 Years and older (Child, Adult, Senior) |
NCT03315182 |
MPSIIIB |
MPSIIIB |
October 16, 2017 |
October 2020 |
October 2020 |
October 20, 2017 |
November 6, 2017 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
| 19 |
NCT02095015 |
Terminated |
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project) |
- Mucopolysaccharidosis
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
- Mucopolysaccharidosis VI
|
|
Observational |
|
|
Industry |
- Time Perspective: Prospective
|
- The status of diagnosis of MPS II (either positive or negative) of each subject
- The status of diagnosis of MPS I or VI (either positive or negative) of each subject
|
160 |
Male |
up to 7 Years (Child) |
NCT02095015 |
SHP-001-801 |
|
March 2014 |
August 2018 |
September 2018 |
March 24, 2014 |
June 25, 2015 |
|
- Children's Hospital Los Angeles
Los Angeles, California, United States - Children's National Medical Center
Washington, District of Columbia, United States - Nemours Children's Clinic
Jacksonville, Florida, United States - (and 16 more...)
|
| 20 |
NCT02493998 |
Recruiting |
A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) |
- Mucopolysaccharidosis Type IIIB
- Mucopolysaccharidosis Type 3 B
- MPS III B
- MPS 3 B
|
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Neurocognitive function
- Imaging characteristics
- Behavioral function
- (and 4 more...)
|
30 |
All |
1 Year to 10 Years (Child) |
NCT02493998 |
250-901 |
|
November 2015 |
December 2018 |
April 2019 |
July 10, 2015 |
April 2, 2018 |
|
- Children's Hospital and Research Center Oakland
Oakland, California, United States - Melbourne Children's Trials Centre
Melbourne, Victoria, Australia - Fundacion Cardioinfantil-Instituto de Cardiologia
Bogota, Colombia - (and 5 more...)
|
| 21 |
NCT02153255 |
Withdrawn |
Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa |
- Mucopolysaccharidosis IV
- Morquio A Disease
|
|
Observational |
|
- Birmingham Women's and Children's NHS Foundation Trust
- BioMarin Pharmaceutical
|
Other / Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Evidence of abnormal walking pattern and surface EMG activity as assessed by Dynamic Gait Analysis
- Change in gait pattern over one year
- Effect on gait pattern of using wrist splints
- Effect on gait pattern of lower limb surgery
|
0 |
All |
6 Years to 18 Years (Child, Adult) |
NCT02153255 |
14/WM/0120 |
|
July 2016 |
July 2016 |
July 2016 |
June 3, 2014 |
July 28, 2016 |
|
- Birmingham Children's Hospital NHS Foundation Trust
Birmingham, West Midlands, United Kingdom
|
| 22 |
NCT00741338 |
Completed
Has Results |
Immune Tolerance Study With Aldurazyme® (Laronidase) |
|
- Biological: Laronidase
- Drug: Cyclosporine A (CsA)
- Drug: Azathioprine (Aza)
|
Interventional |
Phase 1
Phase 2 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants Who Achieved Immune Tolerance Induction
- Percent Reduction of Urinary Glycosaminoglycan (uGAG) Level From Baseline to the End of Treatment/Early Withdrawal
|
7 |
All |
up to 5 Years (Child) |
NCT00741338 |
ALID02307
2007-001163-30 |
|
September 2008 |
September 2012 |
September 2012 |
August 26, 2008 |
July 2, 2014 |
July 2, 2014 |
- HCPA
Porto Alegre, Brazil - Moscow Research Institute for Pediatrics and Children Surgery
Moscow, Russian Federation
|
| 23 |
NCT01415427 |
Completed
Has Results |
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) |
- Mucopolysaccharidosis IV A
- Morquio A Syndrome
- MPS IVA
|
- Drug: BMN 110 - Weekly
- Drug: BMN 110 - Every Other Week
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change From Baseline in 6-minute Walk (6MW) Test - ITT
- Change From Baseline in 6-minute Walk (6MW) Test - MPP
- Change From Baseline in 3-minute Stair Climb Test - ITT
- (and 3 more...)
|
173 |
All |
5 Years and older (Child, Adult, Senior) |
NCT01415427 |
MOR-005 |
|
July 2011 |
June 16, 2016 |
June 16, 2016 |
August 12, 2011 |
May 19, 2017 |
May 19, 2017 |
- Phoenix, Arizona, United States
- Oakland, California, United States
- Orange, California, United States
- (and 40 more...)
|
| 24 |
NCT01242111 |
Terminated
Has Results |
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) |
- MPS IV A
- Mucopolysaccharidosis IVA
- Morquio A Syndrome
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety Evaluation
- Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
- Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
- (and 3 more...)
|
20 |
All |
Child, Adult, Senior |
NCT01242111 |
MOR-100 |
|
November 2010 |
July 2014 |
July 2014 |
November 16, 2010 |
September 30, 2015 |
September 30, 2015 |
- Birmingham, United Kingdom
- Dumfries, United Kingdom
- London, United Kingdom
- Manchester, United Kingdom
|
| 25 |
NCT01521429 |
Active, not recruiting |
Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI |
|
|
Observational |
|
- Los Angeles Biomedical Research Institute
- Rare Diseases Clinical Research Network
- National Institute of Neurological Disorders and Stroke (NINDS)
- (and 3 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Annual change in dual energy x-ray absorptiometry (DXA)
- Annual change in Peripheral quantitative computer tomography (pQCT)
- Annual change in Biomarkers of bone remodeling
- (and 3 more...)
|
55 |
All |
5 Years to 35 Years (Child, Adult) |
NCT01521429 |
0906M68810
U54NS065768 |
|
August 2009 |
September 2019 |
September 2019 |
January 30, 2012 |
May 5, 2017 |
|
- Children's Hospital & Research Center Oakland
Oakland, California, United States - Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States - University of Minnesota
Minneapolis, Minnesota, United States
|
| 26 |
NCT02044692 |
Recruiting |
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients |
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Incidence of adverse event and adverse drug reaction.
- Change of vital sign, physical and clinical examination, anti-idurasulfase-beta antibody status.
|
34 |
All |
Child, Adult, Senior |
NCT02044692 |
GC1111_OS |
|
January 2014 |
January 2020 |
January 2020 |
January 24, 2014 |
June 20, 2014 |
|
- Samsung Medical Center
Seoul, Korea, Republic of
|
| 27 |
NCT00787995 |
Terminated |
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) |
- MPS IV A
- Mucopolysaccharidosis IVA
- Morquio A Syndrome
|
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
|
- Endurance
- Respiratory Function
|
353 |
All |
Child, Adult, Senior |
NCT00787995 |
MOR-001 |
|
October 2008 |
July 2014 |
July 2014 |
November 10, 2008 |
December 8, 2014 |
|
- Oakland, California, United States
- Chicago, Illinois, United States
- Cordoba, Argentina
- (and 12 more...)
|
| 28 |
NCT02754076 |
Recruiting |
A Treatment Study of Mucopolysaccharidosis Type IIIB |
- MPS III B
- Mucopolysaccharidosis Type IIIB
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety Evaluation of weekly infusions of BMN 250 (Part 1 & Part 2) - Number of participants with abnormal clinical laboratory values and/or Adverse Events that are related to treatment.
- Development Quotient (DQ) as efficacy variable with analysis of rate of change of DQ score on treatment vs. rate of change of DQ score prior to treatment.
- Characterize maximum concentration (Cmax) of BMN 250 in cerebrospinal fluid (CSF) and plasma as relevant through completion of Part 1 and Part 2
- (and 6 more...)
|
33 |
All |
1 Year to 10 Years (Child) |
NCT02754076 |
250-201 |
MPS IIIB |
April 2016 |
March 2019 |
March 2019 |
April 28, 2016 |
December 7, 2017 |
|
- University Medical Center Hamburg Eppendorf, Department of Pediatrics
Hamburg, Germany - Hospital Clinico Universitario de Santiago
Santiago de Compostela, Spain - Mackay Memorial Hospital
Taipei, Taiwan - (and 2 more...)
|
| 29 |
NCT02097251 |
No longer available |
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient |
- Mucopolysaccharidosis Type 7
|
|
Expanded Access |
|
- Joyce Fox
- Ultragenyx Pharmaceutical Inc
- Northwell Health
|
Other / Industry |
|
|
|
Male |
Child, Adult, Senior |
NCT02097251 |
13-606A |
|
|
|
|
March 27, 2014 |
February 6, 2017 |
|
- Cohen Children's Medical Center
New Hyde Park, New York, United States
|
| 30 |
NCT03370653 |
Recruiting |
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil |
|
- Drug: Odiparcil
- Other: Placebo
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of patients with modified clinical signs
- Number of patients with modified biological values
- Incidence of AEs/SAEs
- (and 14 more...)
|
24 |
All |
16 Years and older (Child, Adult, Senior) |
NCT03370653 |
IVA_01_ODI_HMPS_17_002 |
iMProveS |
December 13, 2017 |
August 2019 |
March 2020 |
December 12, 2017 |
April 12, 2018 |
|
- Hôpital Femme-Mère-Enfant
Bron, France - Villa Metabolica
Mainz, Germany - Royal Free Hospital
London, United Kingdom
|
| 31 |
NCT00144781 |
Completed
Has Results |
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease |
- Mucopolysaccharidosis I
- Hurler's Syndrome
- Hurler-Scheie Syndrome
- Scheie Syndrome
|
- Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
|
Interventional |
Phase 4 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
- Percent Change From Baseline to Week 26 in Liver Organ Volume
- Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
|
34 |
All |
Child, Adult, Senior |
NCT00144781 |
ALID-017-03 |
|
December 2004 |
January 2006 |
January 2006 |
September 5, 2005 |
April 3, 2015 |
June 16, 2009 |
- Hospital Infantil Joana de Gusmao
Florianopolis, Santa Catarina, Brazil - Universidade Federal de Minas Gerais
Belo Horizonte, Brazil - Hospital de Clinical de Porto Alegre
Porto Alegre, Brazil - (and 2 more...)
|
| 32 |
NCT00004454 |
Completed |
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) |
|
- Genetic: lymphocyte gene therapy
|
Interventional |
Phase 1
Phase 2 |
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- University of Minnesota - Clinical and Translational Science Institute
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Primary Purpose: Treatment
|
|
2 |
All |
18 Years and older (Adult, Senior) |
NCT00004454 |
199/13577
UMN-HUNTER
UMN-5P01HD32652 |
|
October 1996 |
|
|
October 19, 1999 |
June 24, 2005 |
|
- University of Minnesota Medical School
Minneapolis, Minnesota, United States
|
| 33 |
NCT00884949 |
Completed
Has Results |
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA |
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Subject Incidence of Treatment Emergent AEs
- Change From Baseline in 6MWT
- Change From Baseline in 3MSCT
- (and 3 more...)
|
20 |
All |
5 Years to 18 Years (Child, Adult) |
NCT00884949 |
MOR-002 |
|
April 2009 |
February 2011 |
March 2011 |
April 21, 2009 |
June 30, 2014 |
June 30, 2014 |
- Birmingham, United Kingdom
- London, United Kingdom
- Manchester, United Kingdom
|
| 34 |
NCT03153319 |
Recruiting |
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II |
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
|
- Drug: Adalimumab Injection [Humira]
- Drug: Saline Solution for Injection
|
Interventional |
Phase 1
Phase 2 |
- Los Angeles Biomedical Research Institute
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Pain - 16 weeks
- Adalimumab trough
- Joint range-of-motion - 16 weeks
- (and 3 more...)
|
14 |
All |
5 Years and older (Child, Adult, Senior) |
NCT03153319 |
31041-01 |
|
June 5, 2017 |
June 2020 |
June 2020 |
May 15, 2017 |
August 15, 2017 |
|
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States
|
| 35 |
NCT01515956 |
Completed
Has Results |
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) |
- Mucopolysaccharidosis IVA
- Morquio A Syndrome
- MPS IVA
|
|
Interventional |
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
- Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures
- Change From Baseline in Normalized Growth Rate Z-Scores
|
15 |
All |
up to 5 Years (Child) |
NCT01515956 |
MOR-007 |
|
October 2011 |
February 2016 |
February 2016 |
January 24, 2012 |
August 10, 2017 |
July 6, 2017 |
- Oakland, California, United States
- Manhasset, New York, United States
- Monza, Italy
- (and 2 more...)
|
| 36 |
NCT01301898 |
Completed |
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients |
|
- Drug: GC1111_0.5mg/kg
- Drug: GC1111_1.0mg/kg
- Drug: Elaprase_0.5mg/kg
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Primary Outcome
- Secondary Outcome
|
31 |
Male |
6 Years to 35 Years (Child, Adult) |
NCT01301898 |
GC1111_P1/2 |
|
May 2010 |
March 2011 |
March 2011 |
February 23, 2011 |
February 14, 2012 |
|
- Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, Korea, Republic of
|
| 37 |
NCT01275066 |
Completed
Has Results |
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) |
|
- Drug: BMN 110 Weekly
- Drug: Placebo
- Drug: BMN 110 Every Other Week
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change From Baseline in Endurance as Measured by the 6-minute Walk Test
- Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test
- Percent Change From Baseline in Urine Keratan Sulfate Normalized for Urine Creatinine
|
177 |
All |
5 Years and older (Child, Adult, Senior) |
NCT01275066 |
MOR-004
2010-020198-18
10/H1306/87
18972/0213/001-0001
2011_038#B201129
145240
2011-01-09
20110012889
0999935174 |
|
February 2011 |
August 2012 |
August 2012 |
January 12, 2011 |
July 7, 2014 |
April 15, 2014 |
- Oakland, California, United States
- Wilmington, Delaware, United States
- Washington, District of Columbia, United States
- (and 25 more...)
|
| 38 |
NCT01509768 |
Completed |
Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) |
- Sanfilippo Syndrome Type B
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- evaluate the course of disease progression in patients with MPS IIIB who are untreated with any investigational products to inform possible future treatment studies
|
19 |
All |
1 Year to 10 Years (Child) |
NCT01509768 |
HGT-SNB-088 |
|
March 2012 |
November 2014 |
November 2014 |
January 13, 2012 |
November 25, 2014 |
|
- Children's Hospital and Research Center
Oakland, California, United States - University of Minnesota
Minneapolis, Minnesota, United States - Johannes Gutenberg University Medical Center
Mainz, Germany - Great Ormond Street Hospital for Children
London, United Kingdom
|
| 39 |
NCT00912925 |
Completed
Has Results |
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I |
- Mucopolysaccharidosis I
- Hurlers Syndrome
- Hurler-Scheie Syndrome
|
- Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)
- Biological: Placebo
|
Interventional |
Phase 3 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)
- Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
- Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)
- (and 4 more...)
|
45 |
All |
5 Years and older (Child, Adult, Senior) |
NCT00912925 |
ALID-003-99 |
|
December 2000 |
September 2001 |
September 2001 |
June 3, 2009 |
April 7, 2015 |
August 19, 2009 |
- New York, New York, United States
- Chapel Hill, North Carolina, United States
- Vancouver, British Columbia, Canada
- Mainz, Germany
|
| 40 |
NCT02293382 |
Completed |
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB |
- MPS IIIB (Sanfilippo Syndrome)
|
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Retrospective
|
- Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients.
|
30 |
All |
Child, Adult, Senior |
NCT02293382 |
NGLU-NH01 |
|
July 2013 |
January 2015 |
January 2015 |
November 18, 2014 |
February 3, 2017 |
|
- Pittsburg, Pennsylvania, United States
- Porte Alegre, Brazil
- Amsterdam, Netherlands
- (and 2 more...)
|
| 41 |
NCT02293408 |
Active, not recruiting |
Natural History Study to Characterise the Course of Disease Progression in Patients With Mucopolysaccharidosis Type IIIB |
- MPS IIIB (Sanfilippo B Syndrome)
|
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Evaluation of the course of disease progression in patients with MPS IIIB as measured by medical history data and prospective clinical, biochemical and neurocognitive assessments.
|
30 |
All |
1 Year and older (Child, Adult, Senior) |
NCT02293408 |
NGLU-NH02 |
|
September 2014 |
April 2018 |
April 2018 |
November 18, 2014 |
July 13, 2017 |
|
- Minneapolis, Minnesota, United States
- Pittsburgh, Pennsylvania, United States
- Porte Alegre, Brazil
- (and 4 more...)
|
| 42 |
NCT00258011 |
Completed
Has Results |
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease |
- Mucopolysaccharidosis I
- Hurler Syndrome
- Hurler-Scheie Syndrome
- Scheie Syndrome
|
- Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
|
Interventional |
Phase 3 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety Evaluation
- Urinary Glycosaminoglycan (GAG) Excretion
|
3 |
All |
Child, Adult, Senior |
NCT00258011 |
ALID02205 |
|
December 2005 |
October 2006 |
October 2006 |
November 24, 2005 |
March 7, 2014 |
February 12, 2009 |
- Osaka City University Hospital
Osaka, Japan - National Center for Child Health and Development
Tokyo, Japan
|
| 43 |
NCT00146770 |
Completed
Has Results |
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients |
- Mucopolysaccharidosis I
- Hurler's Syndrome
- Hurler-Scheie Syndrome
- Scheie Syndrome
|
- Biological: Aldurazyme
- Biological: placebo
|
Interventional |
Phase 3 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)
- Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)
- Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)
- (and 3 more...)
|
45 |
All |
Child, Adult, Senior |
NCT00146770 |
ALID-006-01 |
|
May 2001 |
March 2005 |
March 2005 |
September 7, 2005 |
April 3, 2015 |
June 16, 2009 |
- University of South Alabama
Mobile, Alabama, United States - Emory University School of Medicine
Atlanta, Georgia, United States - University of Rochester
Rochester, New York, United States - (and 22 more...)
|
| 44 |
NCT00418821 |
Recruiting |
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants |
- Mucopolysaccharidosis I
- Hurler's Syndrome
- Hurler-Scheie Syndrome
- Scheie
|
- Biological: Aldurazyme (laronidase)
|
Interventional |
Phase 4 |
- Genzyme, a Sanofi Company
- BioMarin/Genzyme LLC
- Sanofi
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Breast milk samples for laronidase, Immunogenicity Testing, and Urinary glycosaminoglycans (uGAG).
|
10 |
Female |
Child, Adult, Senior |
NCT00418821 |
ALID01803
2007-007003-33 |
|
October 2010 |
January 2020 |
January 2020 |
January 5, 2007 |
August 22, 2017 |
|
- Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo
Rome, Italy
|
| 45 |
NCT00069641 |
Completed
Has Results |
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) |
|
- Biological: Iduronate-2-sulfatase enzyme replacement therapy
- Biological: iduronate-2-sulfatase enzyme replacement therapy
- Biological: Placebo
|
Interventional |
Phase 2
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53
- Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53
- Mean Combined Liver and Spleen Volume at Baseline
- (and 4 more...)
|
96 |
Male |
5 Years to 25 Years (Child, Adult) |
NCT00069641 |
TKT024 |
|
September 2003 |
March 2005 |
March 2005 |
October 1, 2003 |
May 13, 2015 |
May 13, 2015 |
- Children's Hospital Oakland
Oakland, California, United States - St. Louis Children's Hospital, Washington University
St. Louis, Missouri, United States - University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States - (and 6 more...)
|
| 46 |
NCT02746341 |
Recruiting |
Natural History Study of Patients With MPS IIIA |
- Mucopolysaccharidosis IIIA
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition
- Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale
- Sleep disturbances measured by Actigraphy
- (and 2 more...)
|
25 |
All |
up to 9 Years (Child) |
NCT02746341 |
P3-LYS-SAF |
|
April 2016 |
December 2018 |
March 2019 |
April 21, 2016 |
June 20, 2017 |
|
- Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil - Armand Trousseau Public Hospital
Paris, France - University Medical Center Hamburg-Eppendorf
Hamburg, Germany - (and 2 more...)
|
| 47 |
NCT03071341 |
Enrolling by invitation |
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I |
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- number of patients with adverse events as a measure of safety and tolerability
- change in total urinary glycosaminoglycans (GAGs)
- change in urinary heparan sulfate and dermatan sulfate
- (and 3 more...)
|
11 |
All |
2 Years and older (Child, Adult, Senior) |
NCT03071341 |
AGT-181-101E |
|
October 2016 |
July 2019 |
October 2019 |
March 6, 2017 |
March 6, 2017 |
|
- HCPA - Hospital das Clinicas de Porto Alegre
Porto Alegre, RS, Brazil
|
| 48 |
NCT02583152 |
Recruiting |
New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis |
|
|
Observational |
|
- Manchester Royal Eye Hospital
- BioMarin Pharmaceutical
|
Other / Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Corneal densitometry scores in participants on treatment
- Corneal clouding score over time in patients on treatment.
|
50 |
All |
3 Years and older (Child, Adult, Senior) |
NCT02583152 |
R04002 |
|
November 2015 |
November 2020 |
November 2020 |
October 22, 2015 |
November 28, 2017 |
|
- Manchester Royal Eye Hospital
Manchester, United Kingdom
|
| 49 |
NCT03053089 |
Active, not recruiting |
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I |
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Stage 1: number of patients with adverse events as a measure of safety and tolerability of a single dose
- Stage 2: number of patients with adverse events as a measure of safety and tolerability of repeat weekly doses
- PK parameters (maximal concentration, half-life, AUC, distribution and clearance) of AGT-181
- (and 4 more...)
|
21 |
All |
2 Years and older (Child, Adult, Senior) |
NCT03053089 |
AGT-181-101 |
|
October 2015 |
July 2017 |
October 2017 |
February 14, 2017 |
March 1, 2017 |
|
- HCPA - Hospital das Clinicas de Porto Alegre
Porto Alegre, RS, Brazil
|
| 50 |
NCT02663024 |
Not yet recruiting |
Study of Idursulfase-beta (GC1111) in Hunter Syndrome |
|
- Biological: idursulfase beta
- Biological: idursulfase
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
- Change from baseline in urinary GAG at Week 25
- Change from baseline in Six Minute Walk Test at Week 25
- (and 11 more...)
|
20 |
Male |
5 Years to 35 Years (Child, Adult) |
NCT02663024 |
GC1111B_P2 |
|
December 2016 |
December 2019 |
June 2020 |
January 26, 2016 |
January 26, 2016 |
|
|
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