- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
- Interventions
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Behavioral
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 12 Months to 99 Years
- Enrollment
- 480 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–6
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- Conditions
- Thymidine Kinase 2 Deficiency
- Interventions
- Observational retrospective data
- Other
- Lead sponsor
- Zogenix MDS, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Indianapolis, Indiana • Akron, Ohio + 1 more
- Conditions
- Thymidine Kinase 2 Deficiency
- Interventions
- doxecitine and doxribtimine
- Drug
- Lead sponsor
- UCB BIOSCIENCES, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Mitochondrial DNA Depletion Syndrome 2 Myopathic Type, Thymidine Kinase 2 Deficiency
- Interventions
- Thymidine
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2026
- U.S. locations
- 16
- States / cities
- San Diego, California • Stanford, California • Aurora, Colorado + 12 more
- Conditions
- Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 6,900 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York