- Conditions
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
- Interventions
- Not listed
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 7 Years to 18 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
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Showing 25–30
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- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- ARCT-810, Placebo
- Biological · Other
- Lead sponsor
- Arcturus Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 8
- States / cities
- Gainesville, Florida • Minneapolis, Minnesota • New York, New York + 5 more
- Conditions
- Metabolic Diseases
- Interventions
- human hepatocyte transplantation, Preparative Radiation Therapy
- Drug · Radiation
- Lead sponsor
- Ira Fox
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2022
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Urea Cycle Disorder
- Interventions
- KB195
- Drug
- Lead sponsor
- Kaleido Biosciences
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 6
- States / cities
- Washington D.C., District of Columbia • Tampa, Florida • Baltimore, Maryland + 3 more
- Conditions
- OTC Deficiency
- Interventions
- DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
- Genetic · Other · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2031
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
- Conditions
- Ornithine Transcarbamylase (OTC) Deficiency
- Interventions
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more