- Conditions
- Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- New York, New York
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Showing 1–24
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Porphyria Cutanea Tarda, Hepatitis C
- Interventions
- Harvoni
- Drug
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 3
- States / cities
- San Francisco, California • Winston-Salem, North Carolina • Galveston, Texas
- Conditions
- Erythropoietic Protoporphyria (EPP)
- Interventions
- Not listed
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 7 Years and older
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2020
- U.S. locations
- 1
- States / cities
- Birmingham, Alabama
- Conditions
- Acute Intermittent Porphyria
- Interventions
- givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Birmingham, Alabama • San Francisco, California • New York, New York + 1 more
- Conditions
- Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
- Interventions
- Not listed
- Lead sponsor
- Portal Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 5
- States / cities
- Boston, Massachusetts • New York, New York • Wake Forest, North Carolina + 2 more
- Conditions
- Porphyrias
- Interventions
- Web-based Survey
- Other
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria, Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP)
- Interventions
- Givosiran, Placebo
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 10
- States / cities
- Little Rock, Arkansas • San Francisco, California • Boston, Massachusetts + 7 more
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA Dehydratase Deficient Porphyria (ADP), Porphyria Acute
- Interventions
- Not listed
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 212 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 12
- States / cities
- Pasadena, California • Jacksonville, Florida • Margate, Florida + 9 more
- Conditions
- Acute Porphyrias
- Interventions
- Panhematin, Glucose
- Biological · Other
- Lead sponsor
- The University of Texas Medical Branch, Galveston
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2022
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Erythropoietic Protoporphyria, X-linked Protoporphyria
- Interventions
- Cimetidine, Placebo
- Drug
- Lead sponsor
- Amy K. Dickey, M.D.
- Other
- Eligibility
- 15 Years and older
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Winston-Salem, North Carolina • Galveston, Texas
- Conditions
- Porphyria Cutanea Tarda
- Interventions
- Deferasirox
- Drug
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
- Interventions
- Autonomic Function Testing, Genetic Testing, Urine Testing, Blood laboratory Testing
- Diagnostic Test
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 70 participants
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Interventions
- Hemin for injection, Placebo
- Biological · Other
- Lead sponsor
- The University of Texas Medical Branch, Galveston
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2023
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Afamelanotide, Placebo
- Drug
- Lead sponsor
- Clinuvel Pharmaceuticals Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 93 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • San Francisco, California • Detroit, Michigan + 4 more
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- Colestipol
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 22 Years to 60 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
- Interventions
- DISC-1459
- Drug
- Lead sponsor
- Disc Medicine, Inc
- Industry
- Eligibility
- 12 Years and older
- U.S. locations
- 5
- States / cities
- Miami, Florida • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Porphyria
- Interventions
- heme arginate, tin mesoporphyrin
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1997
- U.S. locations
- 2
- States / cities
- New York, New York • Galveston, Texas
- Conditions
- Acute Hepatic Porphyria
- Interventions
- Not listed
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 136 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2021
- U.S. locations
- 8
- States / cities
- San Francisco, California • Miami, Florida • Ann Arbor, Michigan + 5 more
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- DISC-1459
- Drug
- Lead sponsor
- Disc Medicine, Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 230 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • San Francisco, California • Miami, Florida + 7 more
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Interventions
- Hemin
- Drug
- Lead sponsor
- The University of Texas Medical Branch, Galveston
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2026
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Acute Intermittent Porphyria
- Interventions
- recombinant human porphobilinogen deaminase (Porphozym)
- Drug
- Lead sponsor
- Zymenex A/S
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2006
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Porphyria
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1992
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Porphyria Cutanea Tarda
- Interventions
- Hydroxychloroquine, Phlebotomy
- Drug · Procedure
- Lead sponsor
- The University of Texas Medical Branch, Galveston
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2021
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- Congenital Erythropoietic Porphyria (CEP)
- Interventions
- ATL-001 (Ciclopirox oral solution)
- Drug
- Lead sponsor
- Atlas Molecular Pharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio