| 1 |
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...)
|
| 2 |
NCT02262338 |
Recruiting |
Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome |
|
|
Interventional |
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- number of participants with adverse events as a measure of safety and tolerability
- plasma pharmacokinetic parameters (maximal concentration, half-life, area under the curve, mean residence time, volume of distribution and clearance of AGT-182)
- change in urinary or plasma glycosaminoglycans (GAGs)
- (and 2 more...)
|
8 |
Male |
18 Years and older (Adult, Senior) |
NCT02262338 |
AGT-182-101 |
|
April 2015 |
July 2017 |
October 2017 |
October 13, 2014 |
March 3, 2017 |
|
- Children's Hospital Oakland
Oakland, California, United States - Children's Hospital of Orange County
Orange, California, United States - Emory University
Decatur, Georgia, United States - (and 8 more...)
|
| 3 |
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
|
| 4 |
NCT02455622 |
Recruiting |
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age |
|
- Drug: Elaprase for intravenous (IV) infusion
|
Interventional |
Phase 4 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in height
- Change in weight Z-scores
- Change in neurological examination to evaluate long-term safety in patient
- (and 12 more...)
|
15 |
Male |
up to 6 Years (Child) |
NCT02455622 |
SHP-ELA-401 |
|
June 2015 |
February 2022 |
August 2022 |
May 28, 2015 |
March 13, 2017 |
|
- Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - Hospital Infantil Dr Robert Reid Cabral
Santo Domingo, Dominican Republic - Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany - (and 7 more...)
|
| 5 |
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
|
| 6 |
NCT03041324 |
Recruiting |
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II |
- Mucopolysaccharidosis II
- MPS II
|
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
- Effect of SB-913 on IDS activity
- Effect of SB-913 on urine glycosaminoglycans (GAG) levels
- AAV2/6 clearance in plasma, saliva, urine, stool, and semen
|
9 |
Male |
18 Years and older (Adult, Senior) |
NCT03041324 |
SB-913-1602 |
|
May 11, 2017 |
February 2021 |
February 2022 |
February 2, 2017 |
April 17, 2018 |
|
- UCSF Benioff Children's Hospital Oakland
Oakland, California, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - University of Minnesota
Minneapolis, Minnesota, United States - (and 4 more...)
|
| 7 |
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
|
| 8 |
NCT01449240 |
Completed
Has Results |
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome |
|
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Levels of Total Glycosaminoglycan (GAG) in CSF
- Levels of GAG in Urine
|
10 |
Male |
up to 70 Years (Child, Adult, Senior) |
NCT01449240 |
HGT-HIT-072 |
|
March 2011 |
January 2014 |
May 2014 |
October 10, 2011 |
October 28, 2015 |
December 5, 2014 |
- Emory University
Decatur, Georgia, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States - (and 4 more...)
|
| 9 |
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 |
|
|
| 10 |
NCT00882921 |
Completed
Has Results |
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients |
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients
- Change From Baseline in uGAG Levels to 109 Weeks
|
26 |
Male |
5 Years and older (Child, Adult, Senior) |
NCT00882921 |
HGT-ELA-042 |
|
October 2008 |
February 2013 |
February 2013 |
April 17, 2009 |
August 7, 2014 |
July 30, 2014 |
- Children's Hospital & Research Center Oakland
Oakland, California, United States - Children's Hospitals and Clinics of Minnesota, Division of Genetics
Minneapolis, Minnesota, United States - Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, RS, Brazil - (and 3 more...)
|
| 11 |
NCT02412787 |
Enrolling by invitation |
Long-Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Patients With Hunter Syndrome That Have Completed Study HGT-HIT-094 |
|
|
Interventional |
Phase 2
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in physical and neurological examination to evaluate long-term safety in patients
- Change in height and weight
- Change in head circumference
- (and 16 more...)
|
49 |
Male |
up to 18 Years (Child, Adult) |
NCT02412787 |
SHP609-302
2014-004143-13 |
|
April 2015 |
September 2022 |
September 2022 |
April 9, 2015 |
March 29, 2017 |
|
- Children's Hospital and Research Center at Oakland
Oakland, California, United States - Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - University of North Carolina
Chapel Hill, North Carolina, United States - (and 6 more...)
|
| 12 |
NCT00920647 |
Completed
Has Results |
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® |
|
- Other: Control
- Drug: Idursulfase IT (1 mg)
- Drug: Idursulfase IT (10 mg)
- Drug: Idursulfase IT (30 mg)
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Serious Adverse Event (SAE)
- Number of Treatment Emergent Adverse Event (AE)
- Safety Changes in Cerebrospinal Fluid (CSF)- White Blood Cells (WBC)
- (and 8 more...)
|
16 |
Male |
3 Years to 18 Years (Child, Adult) |
NCT00920647 |
HGT-HIT-045
2010-020048-36 |
|
November 2009 |
October 2012 |
October 2012 |
June 15, 2009 |
October 28, 2015 |
May 16, 2014 |
- University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States - Birmingham Children's Hospital
Birmingham, United Kingdom - Birmingham Children's Hospital NHS Foundation Trust
Birmingham, United Kingdom
|
| 13 |
NCT02055118 |
Completed |
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment |
|
- Biological: idursulfase-IT
- Other: No IT treatment
|
Interventional |
Phase 2
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from baseline in the GCA score after 12 months of treatment at visit week 52, as obtained by DAS-II testing
- Change from baseline in the adaptive behavior composite (ABC) score after 12 months of treatment at visit week 52, as obtained by VABS-II testing
- Change from baseline in the GCA score at visit weeks 16, 28 and 40 as obtained by DAS-II testing
- (and 6 more...)
|
48 |
Male |
up to 18 Years (Child, Adult) |
NCT02055118 |
HGT-HIT-094
2013-002885-38 |
AIM-IT |
February 1, 2014 |
September 28, 2017 |
September 28, 2017 |
February 4, 2014 |
November 8, 2017 |
|
- Children's Hospital and Research Center at Oakland
Oakland, California, United States - Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - University of North Carolina
Chapel Hill, North Carolina, United States - (and 6 more...)
|
| 14 |
NCT03292887 |
Recruiting |
Hunter Outcome Survey (HOS) |
|
|
Observational |
|
|
Industry |
- Observational Model: Other
- Time Perspective: Prospective
|
- Number of Participants With Infusion-related Reactions (IRRs)
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Number of Participants With Positive Antibody Response
- (and 19 more...)
|
2000 |
All |
Child, Adult, Senior |
NCT03292887 |
HOS |
HOS |
August 1, 2005 |
September 1, 2022 |
September 1, 2022 |
September 26, 2017 |
September 26, 2017 |
|
- Shire
Lexington, Massachusetts, United States
|
| 15 |
NCT01506141 |
Active, not recruiting |
Extension of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase (IT)in Conjunction With Elaprase in Pediatric Patients With Hunter Syndrome and Cognitive Impairment |
|
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety of intrathecal idursulfase-IT administration
- Pharmacokinetic (PK) parameters of idursulfase-IT administered in conjunction with Elaprase in CSF and blood.
- Change from baseline in CSF biomarkers.
- Change from baseline in urinary GAGs and GAG-degradation products
|
20 |
Male |
3 Years to 18 Years (Child, Adult) |
NCT01506141 |
HGT-HIT-046
2011-000212-25 |
|
August 2010 |
July 2021 |
July 2021 |
January 9, 2012 |
February 15, 2017 |
|
- Ann & Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States - University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States - Legacy Emanuel Hospital
Portland, Oregon, United States - (and 6 more...)
|
| 16 |
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...)
|
| 17 |
NCT00937794 |
Completed
Has Results |
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase® |
|
- Behavioral: Neurobehavioral testing
- Other: Visual and auditory assessments
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent
- Number of Participants With a Score of at Least 90 on The General Conceptual Ability (GCA) Sub-Scale of The Differential Ability Scale (DAS)
|
33 |
Male |
32 Months to 18 Years (Child, Adult) |
NCT00937794 |
HGT-HIT-050 |
|
July 2009 |
July 2011 |
July 2011 |
July 13, 2009 |
May 16, 2016 |
May 16, 2016 |
- University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States - Birmingham Children's Hospital
Birmingham, United Kingdom
|
| 18 |
NCT00630747 |
Completed
Has Results |
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase |
- Hunter Syndrome
- Mucopolysaccharidosis II (MPS II)
|
|
Interventional |
Phase 2
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105
- Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105
- Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105
- (and 3 more...)
|
94 |
Male |
5 Years and older (Child, Adult, Senior) |
NCT00630747 |
TKT024EXT
2004-002743-27 |
|
September 2004 |
January 2008 |
January 2008 |
March 7, 2008 |
August 7, 2015 |
March 17, 2014 |
- St. Joseph's Hospital
Phoenix, Arizona, United States - Pediatric Clinical Research Center, Children's Hospital Oakland
Oakland, California, United States - The Children's Hospital
Denver, Colorado, United States - (and 49 more...)
|
| 19 |
NCT01822184 |
Completed |
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II) |
- Mucopolysaccharidosis (MPS)
- Hunter Syndrome
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Neurodevelopmental parameters of cognitive function over time in pediatric patients with MPS II
- Neurodevelopmental parameters of adaptive function over time in pediatric patients with MPS II
- Reported adverse events
- (and 2 more...)
|
100 |
Male |
2 Years to 18 Years (Child, Adult) |
NCT01822184 |
HGT-HIT-090 |
|
January 2013 |
October 2016 |
October 2016 |
April 2, 2013 |
October 20, 2016 |
|
- Childrens Hospital & Research Center Oakland
Oakland, California, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - University of North Carolina Division of Genetics and Metabolism
Chapel Hill, North Carolina, United States - (and 4 more...)
|
| 20 |
NCT00607386 |
Completed
Has Results |
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy |
- Hunter Syndrome
- Mucopolysaccharidosis II
- MPS II
|
|
Interventional |
Phase 4 |
- Shire
- Covance
- PharmaNet
- PRA Health Sciences
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety Evaluation
- Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
- Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
- (and 7 more...)
|
28 |
Male |
up to 5 Years (Child) |
NCT00607386 |
HGT-ELA-038
2007-006044-22 |
|
December 2007 |
July 2011 |
July 2011 |
February 5, 2008 |
October 28, 2015 |
November 7, 2013 |
- Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, RS, Brazil - Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii
Warsaw, Poland - National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics
Taipei, Taiwan
|
| 21 |
NCT01645189 |
Completed |
Safety and Efficacy of Hunterase |
|
|
Interventional |
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of adverse events
- change of anti-idursulfase-beta antibody status
- Percent Change of Urine GAG
|
6 |
Male |
up to 5 Years (Child) |
NCT01645189 |
GC1111C |
GC1111 |
July 2012 |
September 2013 |
September 2013 |
July 20, 2012 |
July 8, 2014 |
|
- Samsug Medical Center
Seoul, Korea, Republic of
|
| 22 |
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
|
| 23 |
NCT01330277 |
Recruiting |
Biomarker for Hunter Disease |
- Lysosomal Storage Diseases
- Hunter Disease
- Mucopolysaccharidoses
|
|
Observational |
|
- University of Rostock
- Centogene AG Rostock
|
Other / Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Development of a new MS-based biomarker for the early and sensitive diagnosis of Hunter disease from plasma and saliva
- Testing for clinical robustness, specificity and long-term stability of the biomarker
|
80 |
All |
Child, Adult, Senior |
NCT01330277 |
BH03/2011 |
BioHunt |
March 2011 |
September 2018 |
October 2018 |
April 6, 2011 |
May 4, 2017 |
|
- Pediatric practice
Oran, Algeria - Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica Recruiting
Porto Alegre, Brazil - Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil - (and 8 more...)
|
| 24 |
NCT01602601 |
Completed |
A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 |
|
- Drug: Idursulfase
- Drug: GSK2788723
|
Observational |
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Cross-Sectional
|
- Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase
|
10 |
All |
Child, Adult, Senior |
NCT01602601 |
116406 |
|
April 9, 2012 |
June 11, 2012 |
June 11, 2012 |
May 21, 2012 |
September 25, 2017 |
|
- GSK Investigational Site
Tokyo, Japan
|
| 25 |
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
|
| 26 |
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
|
| 27 |
NCT00748969 |
Terminated
Has Results |
Clinical Trial of Growth Hormone in MPS I, II, and VI |
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
- Mucopolysaccharidosis VI
|
- Drug: Somatropin (DNA origin)
|
Interventional |
Phase 2
Phase 3 |
- Los Angeles Biomedical Research Institute
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in Growth Velocity From Baseline to End of Study Year 1.
- Safety of Nutropin AQ® in Children With MPS I, II, and VI. Safety Endpoints Are: • Physical Examinations • Fundoscopic Examinations • Adverse Events • Radiographic Examinations of Spine and Lower Extremities
- Change in Bone Mineral Density, Content, and Strength by DXA and pQCT, and Change in Serum Markers of Bone Metabolism.
- (and 3 more...)
|
2 |
All |
5 Years to 17 Years (Child) |
NCT00748969 |
0808M43681 |
|
November 2008 |
September 2013 |
September 2013 |
September 9, 2008 |
November 20, 2017 |
November 24, 2014 |
- University of Minnesota
Minneapolis, Minnesota, United States
|
| 28 |
NCT01870375 |
Recruiting |
Longitudinal Studies of Brain Structure and Function in MPS Disorders |
- Mucopolysaccharidosis Type I
- Mucopolysaccharidosis Type II
- Mucopolysaccharidosis Type VI
- (and 2 more...)
|
|
Observational |
|
- University of Minnesota - Clinical and Translational Science Institute
- Rare Diseases Clinical Research Network
- National Center for Advancing Translational Science (NCATS)
- (and 3 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in Cognitive Ability (IQ)
- Change in Quality of Life
- Change in Neuropsychological Status
- (and 3 more...)
|
100 |
All |
6 Years to 25 Years (Child, Adult) |
NCT01870375 |
0905M65804
U54NS065768 |
|
September 2009 |
August 2019 |
August 2019 |
June 6, 2013 |
October 20, 2017 |
|
- Oakland Children's Hospital
Oakland, California, United States - University of Minnesota
Minneapolis, Minnesota, United States - New York University
New York, New York, United States - Hospital for Sick Children
Toronto, Ontario, Canada
|
| 29 |
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
|
| 30 |
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...)
|
| 31 |
NCT01938014 |
Unknown † |
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children |
- Mucopolysaccharidosis Type I (MPS I)
- Mucopolysaccharidosis Type II (MPS II)
- Mucopolysaccharidosis Type III (MPS III)
- (and 2 more...)
|
|
Observational |
|
- University of Chicago
- Rare Diseases Clinical Research Network
- National Center for Advancing Translational Science (NCATS)
- (and 4 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in Health Status of the Lysosomal Disease-Affected Child Measured at 6-month Intervals for 5.5 Years
- Change in the Behavioral Outcomes of the Immediate Family of the Lysosomal Disease-Affected Child Measured at 6-month Intervals for 5.5 Years
- Change in Developmental Status of the Lysosomal Disease-Affected Child Measured at 6-month Intervals for 5.5 Years
- (and 4 more...)
|
150 |
All |
up to 18 Years (Child, Adult) |
NCT01938014 |
RDCRN-LDN-6710
U54NS065768 |
|
January 2009 |
August 2017 |
August 2017 |
September 10, 2013 |
August 11, 2015 |
|
- University of Chicago
Chicago, Illinois, United States - University of Minnesota
Minneapolis, Minnesota, United States - Hunter James Kelly Institute
Buffalo, New York, United States
|
| 32 |
NCT01586871 |
Completed |
Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network |
- MPS I
- MPS II
- MPS VI
- Mucopolysaccharidoses
|
|
Observational |
|
- University of Minnesota - Clinical and Translational Science Institute
- Rare Diseases Clinical Research Network
- National Center for Advancing Translational Science (NCATS)
- (and 2 more...)
|
Other / NIH |
- Observational Model: Case-Control
- Time Perspective: Cross-Sectional
|
- Carotid Artery Thickness
- Carotid Artery Stiffness
|
27 |
All |
3 Years to 18 Years (Child, Adult) |
NCT01586871 |
1202M09721
U54NS065768 |
|
March 2012 |
August 2016 |
June 2017 |
April 27, 2012 |
September 5, 2017 |
|
- Children's Hospital of Orange County
Orange, California, United States - University of Minnesota
Minneapolis, Minnesota, United States
|
| 33 |
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
|
| 34 |
NCT02254863 |
Recruiting |
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells |
- Adrenoleukodystrophy
- Batten Disease
- Mucopolysaccharidosis II
- (and 9 more...)
|
|
Interventional |
Phase 1 |
- Joanne Kurtzberg, MD
- The Marcus Foundation
- Duke University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluate for Infusional Toxicity
- Evaluate for Neuro Toxicity
- Efficacy determination
|
12 |
All |
up to 22 Years (Child, Adult) |
NCT02254863 |
Pro00050198 |
DUOC-01 |
September 2014 |
September 2018 |
September 2018 |
October 2, 2014 |
March 16, 2018 |
|
- Duke University Medical Center
Durham, North Carolina, United States
|
| 35 |
NCT02171104 |
Recruiting |
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis |
- Mucopolysaccharidosis Disorders
- Hurler Syndrome
- Hunter Syndrome
- (and 26 more...)
|
- Biological: Stem Cell Transplantation
- Drug: IMD Preparative Regimen
- Drug: Osteopetrosis Only Preparative Regimen
- (and 5 more...)
|
Interventional |
Phase 2 |
- Masonic Cancer Center, University of Minnesota
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percent of subjects who achieve high-level donor hematopoietic engraftment
- Graft-versus-host disease
- Transplant-related mortality
- (and 2 more...)
|
100 |
All |
up to 55 Years (Child, Adult) |
NCT02171104 |
2013LS104 |
|
July 10, 2014 |
September 2019 |
September 2019 |
June 23, 2014 |
January 23, 2018 |
|
- Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
|
| 36 |
NCT01043640 |
Completed
Has Results |
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders |
- Mucopolysaccharidosis
- Hurler Syndrome
- Hunter Syndrome
- (and 10 more...)
|
- Drug: Campath-1H
- Drug: Cyclophosphamide
- Drug: Busulfan
- (and 3 more...)
|
Interventional |
Phase 2 |
- Masonic Cancer Center, University of Minnesota
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Patients With Donor Derived Engraftment
- Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
- Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
- (and 5 more...)
|
46 |
All |
up to 21 Years (Child, Adult) |
NCT01043640 |
2009LS088
MT2009-19 |
|
December 2009 |
June 2015 |
June 2017 |
January 7, 2010 |
February 5, 2018 |
May 12, 2017 |
- Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
|
| 37 |
NCT01372228 |
Active, not recruiting |
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders |
- Hurler Syndrome (MPS I)
- Hurler-Scheie Syndrome
- Hunter Syndrome (MPS II)
- (and 9 more...)
|
- Biological: hematopoietic stem cell infusion
|
Interventional |
Phase 1
Phase 2 |
- University of Louisville
- Duke University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Production of missing enzyme at levels greater than or equal to 10% of normal
- Enriched Hematopoetic Stem Cell Engraftment
|
30 |
All |
Child, Adult, Senior |
NCT01372228 |
ICT-14070-010611 |
|
April 2011 |
April 2025 |
April 2028 |
June 13, 2011 |
November 17, 2017 |
|
- Duke University Medical Center
Durham, North Carolina, United States
|
| 38 |
NCT03333200 |
Recruiting |
Longitudinal Study of Neurodegenerative Disorders |
- MLD
- Krabbe Disease
- ALD
- (and 24 more...)
|
- Other: Palliative Care
- Biological: Hematopoetic Stem Cell Transplantation
|
Observational |
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Cognitive development
- Language development
- Gross Motor development .
- (and 4 more...)
|
1500 |
All |
Child, Adult, Senior |
NCT03333200 |
PRO11050036 |
|
January 11, 2012 |
January 2030 |
January 2035 |
November 6, 2017 |
February 1, 2018 |
|
- The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
| 39 |
NCT00654433 |
Terminated |
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases |
- Inherited Metabolic Diseases
- Lysosomal Storage Disorders
- Peroxisomal Storage Diseases
- (and 2 more...)
|
|
Interventional |
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases
- To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
- To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
|
40 |
All |
up to 16 Years (Child) |
NCT00654433 |
UCBT-002
BB-IND#13502 |
UCBT-002 |
March 2008 |
July 2011 |
November 2011 |
April 8, 2008 |
July 8, 2014 |
|
- Mattel Children's Hospital at UCLA
Los Angeles, California, United States - Mt. Sinai Medical Center
New York, New York, United States - Duke University
Durham, North Carolina, United States
|
| 40 |
NCT01962415 |
Recruiting |
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT |
- Primary Immunodeficiency (PID)
- Congenital Bone Marrow Failure Syndromes
- Inherited Metabolic Disorders (IMD)
- (and 2 more...)
|
- Drug: Hydroxyurea
- Drug: Alemtuzumab
- Drug: Fludarabine
- (and 2 more...)
|
Interventional |
Phase 2 |
- Paul Szabolcs
- University of Pittsburgh
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Post-transplant treatment-related mortality (TRM)
- Neurodevelopmental milestones
- Immune Reconstitution
- (and 9 more...)
|
30 |
All |
2 Months to 35 Years (Child, Adult) |
NCT01962415 |
PRO13100018 |
HSCT+RIC |
February 4, 2014 |
November 2019 |
November 2020 |
October 14, 2013 |
April 5, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
| 41 |
NCT02060526 |
Completed
Has Results |
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease |
|
- Drug: Recombinant human heparan N-sulfatase [rhHNS]
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III)
- Number of Participants With Serious Adverse Events (SAE)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs)
- (and 8 more...)
|
21 |
All |
12 Months to 48 Months (Child) |
NCT02060526 |
HGT-SAN-093
2013-003450-24 |
|
February 2014 |
May 2016 |
May 2016 |
February 12, 2014 |
May 15, 2017 |
March 3, 2017 |
- Los Angeles Biomedical Research
Torrance, California, United States - University of Minnesota Department of Pediatrics
Minneapolis, Minnesota, United States - University of North Carolina
Chapel Hill, North Carolina, United States - (and 7 more...)
|
| 42 |
NCT00744692 |
Completed
Has Results |
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders |
- Non Malignant Disorders
- Immunodeficiencies
- Congenital Marrow Failures
- (and 5 more...)
|
- Biological: Unrelated Umbilical Cord Blood Transplant
- Drug: Reduced Intensity Conditioning
|
Interventional |
Phase 1 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.
- To Describe the Pace of Neutrophil Recovery
- To Evaluate the Pace of Immune Reconstitution.
- (and 6 more...)
|
22 |
All |
up to 21 Years (Child, Adult) |
NCT00744692 |
Pro00008753 |
|
October 2008 |
December 2012 |
April 2014 |
September 1, 2008 |
August 13, 2014 |
August 13, 2014 |
- Duke University Medical Center Pediatric Blood and Marrow Transplant Program
Durham, North Carolina, United States
|
| 43 |
NCT00692926 |
Completed |
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells |
- MDS
- Anemia, Aplastic
- Inborn Errors of Metabolism
- (and 2 more...)
|
- Biological: ALDHbr Umbilical Cord Blood Cells
|
Interventional |
Phase 1 |
- Joanne Kurtzberg, MD
- Aldagen
- Duke University
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To assess the safety of infusing ex vivo cytokine-primed ALDHbr cells isolated from banked unrelated umbilical cord blood (CB) into patients also receiving a standard unmanipulated unrelated CB transplant delivering >2.5x10e7 nucleated cells per kg.
- To describe the biological effect of ex vivo cytokine-primed ALDHbr cells in these patients on neutrophil and platelet engraftment and immune reconstitution.
- To describe the clinical outcomes in these patients: - incidence of infections -Non relapse mortality -incidence of acute and chronic graft-vs-host disease -overall survival at 180 days
|
37 |
All |
up to 55 Years (Child, Adult) |
NCT00692926 |
Pro00008970
7177 |
|
October 2005 |
July 2012 |
July 2012 |
June 6, 2008 |
June 29, 2017 |
|
- Duke University Medical Center
Durham, North Carolina, United States
|
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