| 1 |
NCT02180412 |
Recruiting |
Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria |
|
- Biological: Panhematin
- Other: Glucose
|
Interventional |
Phase 2 |
- The University of Texas Medical Branch, Galveston
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Pain
- Biochemical effects of Panhematin
|
40 |
All |
18 Years to 100 Years (Adult, Senior) |
NCT02180412 |
10-203
RDCRN-7203
FD-R-03720 |
|
April 2014 |
August 31, 2020 |
August 31, 2020 |
July 2, 2014 |
August 22, 2017 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 2 |
NCT02076763 |
Completed |
Observational Study of Acute Intermittent Porphyria Patients |
- Acute Intermittent Porphyria
|
|
Observational |
|
- Digna Biotech S.L.
- Porphyria Centre Sweden
- University of Navarra
- UniQure N.V.
|
Industry / Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Changes of porphobilinogen (PBG) and delta-aminolevulinic acid (ALA) urinary levels in AIP patients
- Clinical Evolution of acute intermittent porphyria. Frecuency of hospitalizations
- Psychological evaluation of patients
- (and 3 more...)
|
9 |
All |
18 Years to 65 Years (Adult) |
NCT02076763 |
DIG-API-2011-01
261506 AIPGene |
|
August 2011 |
July 2013 |
February 2014 |
March 4, 2014 |
March 12, 2014 |
|
- Clinica Universidad de Navarra
Pamplona, Navarra, Spain - 12 Octubre Hospital
Madrid, Spain
|
| 3 |
NCT02452372 |
Completed |
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) |
- Acute Intermittent Porphyria
|
- Drug: givosiran (ALN-AS1)
- Drug: Sterile Normal Saline (0.9% NaCl)
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- The safety of givosiran evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
- Profile of Pharmacokinetics (PK) of givosiran
- The change in delta-aminolevulinic acid (ALA) from baseline
- The change in Porphobilinogen (PBG) from baseline
|
40 |
All |
18 Years to 65 Years (Adult) |
NCT02452372 |
ALN-AS1-001 |
|
May 2015 |
September 2017 |
September 2017 |
May 22, 2015 |
February 12, 2018 |
|
- Clinical Trial Site
Birmingham, Alabama, United States - Clinical Trial Site
San Francisco, California, United States - Clinical Trial Site
New York, New York, United States - (and 3 more...)
|
| 4 |
NCT02949830 |
Active, not recruiting |
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP) |
- Acute Intermittent Porphyria
|
- Drug: givosiran (ALN-AS1)
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The safety of givosiran evaluated by the proportion of patients experiencing adverse events
- The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA)
- The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG)
- (and 2 more...)
|
17 |
All |
18 Years and older (Adult, Senior) |
NCT02949830 |
ALN-AS1-002 |
|
October 2016 |
May 2020 |
August 2020 |
October 31, 2016 |
January 29, 2018 |
|
- Clinical Trial Site
San Francisco, California, United States - Clinical Trial Site
New York, New York, United States - Clinical Trial Site
Galveston, Texas, United States - (and 2 more...)
|
| 5 |
NCT02082860 |
Completed |
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria |
- Acute Intermittent Porphyria
|
- Genetic: rAAV2/5-PBGD vector dosage 1
- Genetic: rAAV2/5-PBGD vector dosage 2
- Genetic: rAAV2/5-PBGD vector dosage 3
- Genetic: rAAV2/5-PBGD vector dosage 4
|
Interventional |
Phase 1 |
- Digna Biotech S.L.
- Porphyria Centre Sweden
- University of Navarra
- (and 2 more...)
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of patients with Adverse Events and Serious Adverse Events
- Effect of the treatment on porphobilinogen (PBG) and delta-aminolevulinic acid (ALA) urinary level.
- Clinical evolution of acute intermittent porphyria. Frequency of hospitalizations
- (and 5 more...)
|
8 |
All |
18 Years to 64 Years (Adult) |
NCT02082860 |
AAVPBGD-AIP-001
2011-005590-23 |
|
November 2012 |
July 2014 |
November 2014 |
March 10, 2014 |
December 18, 2014 |
|
- Clinica Universidad de Navarra
Pamplona, Navarra, Spain - 12 Octubre Hospital
Madrid, Spain
|
| 6 |
NCT01561157 |
Recruiting |
Longitudinal Study of the Porphyrias |
- Acute Porphyrias
- Cutaneous Porphyrias
|
|
Observational |
|
- Icahn School of Medicine at Mount Sinai
|
Other |
- Observational Model: Cohort
- Time Perspective: Other
|
- clinical analysis
- Laboratory analysis
- Relationship between disease severity and biomarkers
- Effectiveness and tolerability of currently used and new therapies for the human porphyrias
|
800 |
All |
Child, Adult, Senior |
NCT01561157 |
GCO 10-1102 |
|
November 2010 |
September 2019 |
December 2019 |
March 22, 2012 |
February 15, 2018 |
|
- University of Alabama, Birmingham
Birmingham, Alabama, United States - University of California, San Francisco
San Francisco, California, United States - University of Miami
Miami, Florida, United States - (and 7 more...)
|
| 7 |
NCT01573754 |
Recruiting |
Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda |
|
- Drug: Hydroxychloroquine
- Procedure: Phlebotomy
|
Interventional |
Phase 2 |
- The University of Texas Medical Branch, Galveston
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Plasma porphyrin concentration
|
100 |
All |
18 Years to 100 Years (Adult, Senior) |
NCT01573754 |
FDA-2604
R01FD002604 |
|
April 2003 |
August 2020 |
August 2022 |
April 9, 2012 |
February 28, 2018 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 8 |
NCT01617642 |
Not yet recruiting |
Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP) |
- Acute Intermittent Porphyria
|
|
Observational |
|
- Nordlandssykehuset HF
- The Royal Norwegian Ministry of Health
|
Other |
- Observational Model: Case Control
- Time Perspective: Cross-Sectional
|
- Blood pressure
- Diet registration
- Iron status
- (and 2 more...)
|
150 |
All |
18 Years and older (Adult, Senior) |
NCT01617642 |
2011/2197/REK
ID/7462 SFP 1068-12 |
|
July 2012 |
August 2017 |
December 2021 |
June 12, 2012 |
June 12, 2012 |
|
- Nordlandssykehuset HF
Bodø, Nordland, Norway
|
| 9 |
NCT00599326 |
Completed
Has Results |
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda |
|
|
Interventional |
Phase 3 |
- University of Texas Southwestern Medical Center
- Novartis Pharmaceuticals
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants Showing Reduction or Elimination of Skin Blistering
- Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
|
10 |
All |
18 Years and older (Adult, Senior) |
NCT00599326 |
CICL670A US17
IRB File Number 062007-047 |
|
January 2008 |
April 2010 |
April 2010 |
January 23, 2008 |
February 10, 2014 |
February 10, 2014 |
- UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
Dallas, Texas, United States
|
| 10 |
NCT02240784 |
Recruiting |
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP) |
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Medical history of AHP patients (Part A only)
- Medication usage of AHP patients (Part A only)
- Plasma biomarkers (Part A only)
- (and 8 more...)
|
300 |
All |
18 Years and older (Adult, Senior) |
NCT02240784 |
ALN-AS1-NT-001 |
|
August 2014 |
December 2020 |
December 2020 |
September 16, 2014 |
December 20, 2017 |
|
- Clinical Trial Site
San Francisco, California, United States - Clinical Trial Site
Ann Arbor, Michigan, United States - Clinical Trial Site
New York, New York, United States - (and 16 more...)
|
| 11 |
NCT00213772 |
Completed |
Risk Factors of Porphyria Cutanea Tarda (PCT) |
|
|
Observational |
|
- University Hospital, Strasbourg, France
|
Other |
- Observational Model: Case Control
- Time Perspective: Longitudinal
- Time Perspective: Prospective
|
|
280 |
All |
Child, Adult, Senior |
NCT00213772 |
2030 |
|
May 1999 |
|
|
September 21, 2005 |
September 21, 2005 |
|
|
| 12 |
NCT02935400 |
Enrolling by invitation |
Acute Porphyria Biomarkers for Disease Activity |
- Acute Intermittent Porphyria
- Hereditary Coproporphyria
- Variegate Porphyria
|
|
Observational |
|
- The University of Texas Medical Branch, Galveston
|
Other |
- Observational Model: Other
- Time Perspective: Prospective
|
- Potential biomarkers Biomarkers
|
50 |
All |
18 Years and older (Adult, Senior) |
NCT02935400 |
14-0001 |
|
January 2014 |
December 2020 |
December 2020 |
October 17, 2016 |
January 25, 2018 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 13 |
NCT02922413 |
Recruiting |
Panhematin for Prevention of Acute Attacks of Porphyria |
- Acute Intermittent Porphyria
- Hereditary Coproporphyria
- Variegate Porphyria
|
- Biological: Hemin for injection
- Other: Placebo
|
Interventional |
Phase 2 |
- The University of Texas Medical Branch, Galveston
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Prevention
|
- Occurance of an acute attack of porphyria after treatment
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
- Effects on levels of porphobilinogen
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT02922413 |
14-0188 |
|
August 2015 |
September 2019 |
September 2020 |
October 4, 2016 |
January 25, 2018 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 14 |
NCT01284946 |
Unknown † |
Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda |
|
|
Interventional |
Phase 2 |
- Assistance Publique - Hôpitaux de Paris
- Association pour l'Etude des Fonctions Digestives (AEFD)
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
- Related drug adverse events
- The change from baseline in serum ferritin after 12 and 24 weeks of treatment,The change from baseline in iron burden after 24 weeks of treatment measured by liver MRI T2,The evolution of clinical symptoms
- Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels
|
45 |
All |
18 Years and older (Adult, Senior) |
NCT01284946 |
AEFD2010-01 |
|
January 2011 |
December 2012 |
December 2012 |
January 27, 2011 |
January 27, 2011 |
|
- Hopital Louis Mourier, GI unit,
Colombes, Ile de France, France
|
| 15 |
NCT00004788 |
Completed |
Study of Nutritional Factors in Porphyria |
|
|
Observational |
|
- National Center for Research Resources (NCRR)
- University of Texas
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Primary Purpose: Screening
|
|
30 |
All |
Child, Adult, Senior |
NCT00004788 |
199/11886
UTMB-328 |
|
May 1988 |
|
|
February 25, 2000 |
June 24, 2005 |
|
|
| 16 |
NCT00004331 |
Unknown † |
Studies in Porphyria I: Characterization of Enzyme Defects |
|
|
Observational |
|
- National Center for Research Resources (NCRR)
- University of Texas
|
NIH / Other |
- Primary Purpose: Screening
|
|
25 |
All |
Child, Adult, Senior |
NCT00004331 |
199/11888
UTMB-399 |
|
November 1992 |
|
|
October 19, 1999 |
June 24, 2005 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 17 |
NCT00004396 |
Completed |
Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias |
|
- Drug: heme arginate
- Drug: tin mesoporphyrin
|
Interventional |
Phase 2 |
- National Center for Research Resources (NCRR)
- University of Texas
|
NIH / Other |
- Primary Purpose: Treatment
|
|
32 |
All |
18 Years and older (Adult, Senior) |
NCT00004396 |
199/13185
UTMB-96-476
UTMB-96-318
UTMB-FDR000710
UTMB-FDR001459 |
|
September 1997 |
|
|
October 19, 1999 |
June 24, 2005 |
|
- Rockefeller University Hospital
New York, New York, United States - University of Texas Medical Branch
Galveston, Texas, United States
|
| 18 |
NCT00004330 |
Completed |
Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks |
|
- Drug: luteinizing hormone-releasing factor
|
Interventional |
Not Applicable |
- National Center for Research Resources (NCRR)
- University of Texas
|
NIH / Other |
- Primary Purpose: Prevention
|
|
|
Female |
18 Years to 55 Years (Adult) |
NCT00004330 |
199/11885
UTMB-445
UTMB-312 |
|
March 1987 |
|
|
October 19, 1999 |
June 24, 2005 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 19 |
NCT00004789 |
Completed |
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria |
|
- Drug: heme arginate
- Drug: tin mesoporphyrin
|
Interventional |
Phase 1
Phase 2 |
- National Center for Research Resources (NCRR)
- University of Texas
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Primary Purpose: Treatment
|
|
59 |
All |
18 Years and older (Adult, Senior) |
NCT00004789 |
199/11887
UTMB-398 |
|
July 1993 |
|
|
February 25, 2000 |
June 24, 2005 |
|
|
| 20 |
NCT00004398 |
Completed |
Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria |
|
- Drug: heme arginate
- Drug: tin mesoporphyrin
|
Interventional |
Phase 1 |
- National Center for Research Resources (NCRR)
- University of Texas
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Allocation: Randomized
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
|
24 |
All |
18 Years and older (Adult, Senior) |
NCT00004398 |
199/13191
UTMB-97-118
UTMB-FDR001459 |
|
January 1998 |
|
May 2000 |
October 19, 1999 |
June 24, 2005 |
|
|
| 21 |
NCT00004397 |
Completed |
Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria |
|
- Drug: heme arginate
- Drug: tin mesoporphyrin
|
Interventional |
Phase 1 |
- National Center for Research Resources (NCRR)
- University of Texas
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Primary Purpose: Treatment
|
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT00004397 |
199/13187
UTMB-97-117
UTMB-FDR001459 |
|
January 1998 |
|
May 2000 |
October 19, 1999 |
June 24, 2005 |
|
|
| 22 |
NCT03118674 |
Recruiting |
Harvoni Treatment Porphyria Cutanea Tarda |
- Porphyria Cutanea Tarda
- Hepatitis C
|
|
Interventional |
Phase 2 |
- Wake Forest University Health Sciences
- Gilead Sciences
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- National Institutes of Health (NIH)
|
Other / Industry / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Resolution of active PCT by 7 months after start of therapy
- Time to resolution of active PCT
|
49 |
All |
18 Years and older (Adult, Senior) |
NCT03118674 |
IRB00043341
U54DK083909 |
|
August 10, 2017 |
August 30, 2022 |
August 30, 2022 |
April 18, 2017 |
February 20, 2018 |
|
- University of Alabama, Birmingham
Birmingham, Alabama, United States - University of California, San Francisco
San Francisco, California, United States - Icahn School of Medicine at Mount Sinai
New York, New York, United States - (and 3 more...)
|
| 23 |
NCT01568554 |
Recruiting |
Clinical Diagnosis of Acute Porphyria |
- Hereditary Coproporphyria (HCP)
- Acute Intermittent Porphyria (AIP)
- Variegate Porphyria (VP)
|
|
Observational |
|
- University of California, San Francisco
- University of Texas
- University of Alabama at Birmingham
- (and 5 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Presence of positive biochemical features by first-line testing in subjects suspected of being a genetic carrier of acute porphyria
- Presence of positive biochemical features by second-line testing in subjects suspected of being a genetic carrier of acute porphyria
- Clinical features suggestive of the acute porphyria carrier state
- (and 6 more...)
|
600 |
All |
15 Years and older (Child, Adult, Senior) |
NCT01568554 |
PC7204
1U54DK083909 |
|
December 2011 |
December 2018 |
December 2018 |
April 2, 2012 |
September 24, 2013 |
|
- UAB Porphyria Center, University of Alabama at Birmingham
Birmingham, Alabama, United States - UCSF Porphyria Center, University of California at San Francisco
San Francisco, California, United States - Mount Sinai Porphyria Comprehensive Diagnostic & Treatment Center, Mount Sinai School of Medicine
New York, New York, United States - (and 3 more...)
|
| 24 |
NCT00005103 |
Completed |
Study of the Pathogenesis of Porphyria Cutanea Tarda |
|
|
Observational |
|
- National Center for Research Resources (NCRR)
- University of Texas
|
NIH / Other |
- Primary Purpose: Screening
|
|
120 |
All |
Child, Adult, Senior |
NCT00005103 |
199/14875
UTMB-433
UTMB-95-173 |
|
November 2000 |
|
|
April 7, 2000 |
June 24, 2005 |
|
- University of Texas Medical Branch
Galveston, Texas, United States
|
| 25 |
NCT00418795 |
Completed |
Porphozym in the Treatment of Acute Attacks in AIP |
- Acute Intermittent Porphyria
|
- Drug: recombinant human porphobilinogen deaminase (Porphozym)
|
Interventional |
Phase 2
Phase 3 |
- Zymenex A/S
- Chiesi Farmaceutici S.p.A.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double
- Primary Purpose: Treatment
|
|
36 |
All |
18 Years and older (Adult, Senior) |
NCT00418795 |
rhPBGD-02 |
|
June 11, 2003 |
June 20, 2006 |
June 20, 2006 |
January 5, 2007 |
March 9, 2018 |
|
- Univercity Texas Medical Branch
Galveston, Texas, United States
|
| 26 |
NCT01422915 |
Completed
Has Results |
Sorbent Therapy of the Cutaneous Porphyrias |
- Erythropoietic Protoporphyria
|
|
Interventional |
Phase 2
Phase 3 |
- Brigham and Women's Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
- Protoporphyrin Concentration in Blood
|
4 |
All |
22 Years to 60 Years (Adult) |
NCT01422915 |
2010P002253 |
EPP |
May 2011 |
March 2012 |
March 2012 |
August 25, 2011 |
April 25, 2017 |
April 25, 2017 |
- Brigham & Women's Hospital
Boston, Massachusetts, United States
|
| 27 |
NCT01880983 |
Active, not recruiting |
Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC)) |
- Erythropoietic Protoporphyria (EPP)
|
|
Observational |
|
- University of Alabama at Birmingham
- Porphyria Rare Disease Clinical Research Consortium
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
150 |
All |
7 Years and older (Child, Adult, Senior) |
NCT01880983 |
7202 |
|
November 2011 |
December 2018 |
December 2018 |
June 19, 2013 |
January 17, 2018 |
|
- The University of Alabama at Birmingham
Birmingham, Alabama, United States
|
| 28 |
NCT03338816 |
Recruiting |
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) |
- Acute Hepatic Porphyria
- Acute Intermittent Porphyria
- Porphyria, Acute Intermittent
- (and 4 more...)
|
- Drug: Givosiran
- Drug: Placebo
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The annualized rate of porphyria attacks in patients with AIP.
- The pharmacodynamic (PD) effect of Givosiran on urine levels of delta-aminolevulinic acid (ALA) in patients with AIP.
- The pharmacodynamic (PD) effect of Givosiran on urine levels of Porphobilinogen (PBG) in patients with AIP.
- (and 6 more...)
|
74 |
All |
12 Years and older (Child, Adult, Senior) |
NCT03338816 |
ALN-AS1-003 |
|
November 1, 2017 |
March 2019 |
September 2021 |
November 9, 2017 |
February 28, 2018 |
|
- Clinical Trial Site
Miami, Florida, United States - Clinical Trial Site
New York, New York, United States - Clinical Trial Site
Winston-Salem, North Carolina, United States - (and 8 more...)
|
| 29 |
NCT00006057 |
Completed |
Diagnostic and Screening Study of Genetic Disorders |
- Tay-Sachs Disease
- Porphyria, Erythropoietic
- Leukodystrophy, Globoid Cell
- Metabolism, Inborn Errors
|
|
Observational |
|
- National Center for Research Resources (NCRR)
- Icahn School of Medicine at Mount Sinai
|
NIH / Other |
- Primary Purpose: Screening
|
|
50 |
All |
Child, Adult, Senior |
NCT00006057 |
199/15151
MTS-GCO-88-459 |
|
December 1999 |
|
|
July 6, 2000 |
June 24, 2005 |
|
- Mount Sinai School of Medicine
New York, New York, United States
|
| 30 |
NCT02943213 |
Completed
Has Results |
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride |
- Anti-Psychotic
- Management of Manifestations of Psychotic Disorders
- Treatment of Schizophrenia
- (and 6 more...)
|
- Drug: Chlorpromazine Hydrochloride
|
Interventional |
Phase 1 |
- Cycle Pharmaceuticals Ltd.
- Parexel
|
Industry |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Maximum Observed Plasma Concentration (Cmax) - Chlorpromazine
- Maximum Observed Plasma Concentration (Cmax) - 7-Hydroxy-Chlorpromazine
- Area Under the Plasma Concentration Versus Time Curve, From Time Zero to t, Where t is the Time of the Last Quantifiable Concentration (AUC(0-t)) - Chlorpromazine
- (and 9 more...)
|
20 |
All |
18 Years to 65 Years (Adult) |
NCT02943213 |
CT-004
PXL231486 |
|
November 2016 |
December 2016 |
December 2016 |
October 24, 2016 |
November 14, 2017 |
October 13, 2017 |
- Farmovs Parexel
Bloemfontein, Kampuslaan Suid, South Africa
|
| 31 |
NCT01550705 |
Terminated
Has Results |
Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria |
- Erythropoietic Protoporphyria (EPP)
- X Linked Erythropoietic Protoporphyria
|
|
Interventional |
Not Applicable |
- University of Utah
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Icahn School of Medicine at Mount Sinai
- (and 3 more...)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in Plasma Protoporphyrin IX Level
- Participants With Increased Sun Sensitivity
|
11 |
All |
18 Years and older (Adult, Senior) |
NCT01550705 |
UTINH
U54DK083909 |
INHEPP |
March 2012 |
December 2015 |
December 2015 |
March 12, 2012 |
January 16, 2017 |
January 16, 2017 |
- University of Utah School of Medicine
Salt Lake City, Utah, United States
|
| 32 |
NCT01688895 |
Recruiting |
Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact |
- Erythropoietic Protoporphyria
- EPP
- X-Linked Protoporphyria
- (and 5 more...)
|
|
Observational |
|
- Icahn School of Medicine at Mount Sinai
- Rare Diseases Clinical Research Network
- Office of Rare Diseases (ORD)
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Other
|
- Clinical Analysis
- Laboratory Analysis
- Relationship between disease severity and biomarkers
|
150 |
All |
Child, Adult, Senior |
NCT01688895 |
GCO 08-0959-04
HSM12-00307
U54DK083909 |
|
July 2012 |
September 2019 |
December 2019 |
September 20, 2012 |
February 15, 2018 |
|
- University of Alabama, Birmingham
Birmingham, Alabama, United States - University of California, San Francisco
San Francisco, California, United States - Icahn School of Medicine at Mount Sinai
New York, New York, United States - (and 4 more...)
|
| 33 |
NCT00004940 |
Completed |
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria |
- Erythropoietic Protoporphyria
|
- Drug: cysteine hydrochloride
|
Interventional |
Phase 3 |
- Brigham and Women's Hospital
- FDA Office of Orphan Products Development
|
Other |
- Primary Purpose: Treatment
|
|
50 |
All |
18 Years to 65 Years (Adult) |
NCT00004940 |
199/13376
BWH-FDR000996-DR |
|
May 1996 |
|
September 2001 |
February 25, 2000 |
March 25, 2015 |
|
|
| 34 |
NCT00004831 |
Completed |
Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria |
- Erythropoietic Protoporphyria
|
- Drug: cysteine hydrochloride
|
Interventional |
Not Applicable |
- FDA Office of Orphan Products Development
- St. Luke's-Roosevelt Hospital Center
|
U.S. Fed / Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Double
- Primary Purpose: Treatment
|
|
20 |
All |
18 Years to 65 Years (Adult) |
NCT00004831 |
199/13413
BWH-FDR000996-EF
SLRH-CU-FDR000996-EF |
|
October 1996 |
|
July 1998 |
February 25, 2000 |
March 25, 2015 |
|
|
| 35 |
NCT02979249 |
Recruiting |
Oral Iron for Erythropoietic Protoporphyrias |
- Erythropoietic Protoporphyria
- EPP
- X-linked Protoporphyria
- XLP
|
|
Interventional |
Not Applicable |
- Icahn School of Medicine at Mount Sinai
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in erythrocyte protoporphyrin levels
- EPP-specific Quality of Life Questionnaire
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT02979249 |
GCO 08-0959-1001
U54DK083909 |
EPP |
December 2016 |
August 2019 |
August 2019 |
December 1, 2016 |
February 2, 2018 |
|
- University of Alabama, Birmingham
Birmingham, Alabama, United States - University of California
San Francisco, California, United States - Icahn School of Medicine at Mount Sinai
New York, New York, United States - (and 3 more...)
|
| 36 |
NCT01605136 |
Unknown † |
Phase III Confirmatory Study in Erythropoietic Protoporphyria |
- Erythropoietic Protoporphyria
|
- Drug: Afamelanotide
- Drug: Placebo
|
Interventional |
Phase 3 |
- Clinuvel Pharmaceuticals Limited
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Duration of direct sunlight exposure between 10:00 and 18:00 hours on days when no pain was experienced (pain score of 0).
- Combined sun exposure and phototoxic pain
- Sun exposure
- (and 4 more...)
|
93 |
All |
18 Years and older (Adult, Senior) |
NCT01605136 |
CUV039 |
|
May 2012 |
April 2013 |
April 2013 |
May 24, 2012 |
March 22, 2013 |
|
- University of Alabama
Birmingham, Alabama, United States - University of California, San Francisco
San Francisco, California, United States - Henry Ford Medical Center
Detroit, Michigan, United States - (and 4 more...)
|
| 37 |
NCT01097044 |
Completed |
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) |
- Erythropoietic Protoporphyria
|
- Drug: Afamelanotide
- Drug: Placebo
|
Interventional |
Phase 2 |
- Clinuvel Pharmaceuticals Limited
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Severity of phototoxic reaction measured by visual analogue scale
- Number of phototoxic reactions
- Quality of life measured by patient completed questionnaire
- (and 2 more...)
|
77 |
All |
18 Years and older (Adult, Senior) |
NCT01097044 |
CUV030 |
|
April 2010 |
April 2011 |
April 2011 |
April 1, 2010 |
March 25, 2013 |
|
- University of Alabama
Birmingham, Alabama, United States - University of California, San Francisco
San Francisco, California, United States - Mt. Sinai
New York, New York, United States - (and 3 more...)
|
| 38 |
NCT00979745 |
Completed |
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) |
- Erythropoietic Protoporphyria
|
- Drug: Afamelanotide
- Drug: Placebo
|
Interventional |
Phase 3 |
- Clinuvel Pharmaceuticals Limited
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Prevention
|
- Severity of phototoxic reaction measured by visual analogue scale
- Number of phototoxic reactions
- Quality of life measured by patient completed questionnaire
- (and 2 more...)
|
70 |
All |
18 Years to 70 Years (Adult, Senior) |
NCT00979745 |
CUV029 |
|
September 2009 |
May 2011 |
May 2011 |
September 18, 2009 |
August 4, 2011 |
|
- HUS:n Iho-ja allergiasairaala (Skin and Allergy Hospital)
Helsinki, Finland - Centre Français des Porphyries, Hôpital Louis Mourier
Colombes, Cedex, France - Department of Dermatology , Heinrich-Heine-University Duesseldorf
Duesseldorf, Germany - (and 5 more...)
|
| 39 |
NCT00206869 |
Unknown † |
Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria |
- Erythropoietic Protoporphyria
|
- Behavioral: exercise
- Behavioral: heat
- Procedure: red light illumination
|
Interventional |
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
|
|
All |
14 Years to 70 Years (Child, Adult, Senior) |
NCT00206869 |
KF 01-169/02 |
|
January 2003 |
|
|
September 21, 2005 |
October 11, 2006 |
|
- Bispebjerg Hospital
Copenhagen, Copenhagen NV, Denmark
|
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