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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF-01913539] In Japanese And Western Healthy Subjects

NCT00825084
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Apr 25, 2011 · Synced Jul 21, 2026, 11:01 PM EDT
Completed No phase listed Observational

The Use of a Distress Thermometer in a Pediatric Research Setting: An Exploratory Pilot Study

NCT00824278
View directory record Official record
Conditions
Cancer, HIV Infections
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
7 Years to 21 Years
Enrollment
289 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 2 Interventional

Anti-VEGF Therapy for Acute Thyroid Eye Disease

NCT04311606
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Sub-tenon injection of saline followed by a sub-tenon injection of aflibercept, Sub-tenon injection of hyaluronidase (HA) followed by a sub-tenon injection of aflibercept, Sub-tenon injection of hyaluronidase (HA) alone
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 3 Interventional

Open-Label Phase III Long-Term Safety Trial of Liprotamase

NCT00449904
View directory record Official record
Conditions
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions
Liprotamase
Drug
Lead sponsor
Anthera Pharmaceuticals
Industry
Eligibility
7 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
39
States / cities
Oakland, California • Denver, Colorado • Jacksonville, Florida + 35 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2014 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 2 Interventional Results available

Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.

NCT02272998
View directory record Official record
Conditions
Malignant Neoplasm
Interventions
ponatinib hydrochloride, laboratory biomarker analysis
Drug · Other
Lead sponsor
Sameek Roychowdhury
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
2
States / cities
Ann Arbor, Michigan • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 23, 2025 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 3 Interventional Results available

Evaluation of Upadacitinib in Adolescent and Adult Patients With Moderate to Severe Atopic Dermatitis (Eczema)

NCT03569293
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Placebo for Upadacitinib, Upadacitinib
Drug
Lead sponsor
AbbVie
Industry
Eligibility
12 Years to 75 Years
Enrollment
912 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
58
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Bakersfield, California + 50 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional

Single-Session "Empowered Relief" Class for Marfan Syndrome and Related Conditions

NCT05980104
View directory record Official record
Conditions
Chronic Pain, Marfan Syndrome, Vascular Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome
Interventions
Empowered Relief
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 25, 2024 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional

Feasibility of a Goal-based Agenda Setting Intervention

NCT04696484
View directory record Official record
Conditions
Cystic Fibrosis, Chronic Disease
Interventions
coopeRATE Prompt
Behavioral
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years and older
Enrollment
219 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
4
States / cities
Denver, Colorado • Kansas City, Kansas • Lebanon, New Hampshire + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2021 · Synced Jul 21, 2026, 11:01 PM EDT
Active, not recruiting Phase 2 Interventional

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

NCT05418049
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Baclofen, Memantine, Roflumilast, Placebo
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 45 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Jul 21, 2026, 11:01 PM EDT
Withdrawn Phase 1 Interventional

Nivolumab for High-Risk MDS/AML Patients After Allogeneic Stem Cell Transplant With Post-Transplant Cyclophosphamide

NCT04361058
View directory record Official record
Conditions
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Myelodysplastic Syndrome Acute Myeloid Leukemia
Interventions
Nivolumab
Drug
Lead sponsor
SCRI Development Innovations, LLC
Other
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Dec 4, 2023 · Synced Jul 21, 2026, 11:01 PM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional

Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease

NCT02987725
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hypnosis, Attention Control
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
12 Years to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 3, 2020 · Synced Jul 21, 2026, 11:01 PM EDT
Recruiting Phase 2 Interventional

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

NCT06447025
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 60 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 21, 2026, 11:01 PM EDT
Not yet recruiting No phase listed Observational Accepts healthy volunteers

Multi-Modal Digital Monitoring of Disease Symptoms Huntington's Disease

NCT07315984
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
BioSensics
Industry
Eligibility
25 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
2
States / cities
Rochester, New York • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced Jul 21, 2026, 11:01 PM EDT
Terminated Phase 1Phase 2 Interventional

A Fabry Disease Gene Therapy Study

NCT04040049
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2023 · Synced Jul 21, 2026, 11:01 PM EDT
Not listed No phase listed Observational

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

NCT01772043
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced Jul 21, 2026, 11:01 PM EDT
Recruiting No phase listed Observational

Pathogenesis and Genetics of Microphthalmia, Anophthalmia and Uveal Coloboma (MAC)

NCT01778543
View directory record Official record
Conditions
Coloboma, Anophthalmia, Microphthalmia
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Year to 100 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial of Rituximab for Graves' Ophthalmopathy

NCT00595335
View directory record Official record
Conditions
Thyroid-associated Ophthalmopathy
Interventions
Rituximab, Saline, Methylprednisolone
Drug
Lead sponsor
Rebecca Bahn
Other
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 31, 2015 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Ultherapy® for Treating the Face and Neck Using Standard Versus Simulines Transducers

NCT02416076
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera Treatment at EL2, Ulthera Treatment at EL4
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2016
U.S. locations
3
States / cities
Beverly Hills, California • Saratoga, California • Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 22, 2019 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional

Bleach Bath Treatment of Adults With Atopic Dermatitis

NCT01996150
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
bleach bath (sodium hypochlorite)
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 65 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 16, 2018 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 1 Interventional

Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia

NCT00093743
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, Fanconi Anemia, Previously Treated Myelodysplastic Syndromes
Interventions
fludarabine phosphate, cyclosporine, total-body irradiation, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, mycophenolate mofetil
Drug · Radiation · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
5
States / cities
Chicago, Illinois • Indianapolis, Indiana • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 19, 2017 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Not applicable Interventional

An Open Label Evaluation of the Safety and Pharmacokinetics of Ganciclovir in Children

NCT00002015
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Roche Global Development
Other
Eligibility
3 Months to 12 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 11:01 PM EDT
Completed No phase listed Observational

Research Study on Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL)

NCT01114815
View directory record Official record
Conditions
CADASIL
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 20, 2013 · Synced Jul 21, 2026, 11:01 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

NCT02439216
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Edasalonexent, Placebo
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2022 · Synced Jul 21, 2026, 11:01 PM EDT