- Conditions
- Neurodegeneration With Brain Iron Accumulation (NBIA)
- Interventions
- Not listed
- Lead sponsor
- Susan J. Hayflick
- Other
- Eligibility
- Not listed
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Portland, Oregon
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Showing 1–7
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- Conditions
- Pantothenate Kinase-Associated Neurodegeneration
- Interventions
- Deferiprone oral solution
- Drug
- Lead sponsor
- ApoPharma
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Pantothenate Kinase-Associated Neurodegeneration
- Interventions
- CoA-Z, Placebo
- Other
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 3 Months to 89 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Pantothenate Kinase-Associated Neurodegeneration
- Interventions
- Deferiprone oral solution, Placebo
- Drug
- Lead sponsor
- ApoPharma
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 89 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Pantothenate Kinase-associated Neurodegeneration (PKAN)
- Interventions
- Not listed
- Lead sponsor
- Susan J. Hayflick
- Other
- Eligibility
- 7 Years to 75 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2018
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Pantothenate Kinase-Associated Neurodegeneration
- Interventions
- Fosmetpantotenate, Placebo
- Drug
- Lead sponsor
- Travere Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 65 Years
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 9
- States / cities
- Irvine, California • Washington D.C., District of Columbia • Miami, Florida + 6 more