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ClinicalTrials.gov public records Last synced Jul 21, 2026, 12:39 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Disorders Clear all
Terminated Phase 2 Interventional Results available

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

NCT02093221
View directory record Official record
Conditions
Type 1 Diabetes Mellitus
Interventions
180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI, Placebo
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
6 Years to 35 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
36
States / cities
Tucson, Arizona • Los Angeles, California • Orange, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2018 · Synced Jul 21, 2026, 12:39 PM EDT
Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

NCT01050855
View directory record Official record
Conditions
Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
Interventions
Reduced-Intensity Conditioning
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
6 Months to 25 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 21, 2026, 12:39 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Identification of Biomarkers in Spinocerebellar Ataxia 3

NCT03885167
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
Specimen Collection
Other
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 21, 2023 · Synced Jul 21, 2026, 12:39 PM EDT
Not yet recruiting Phase 1 Interventional

NAD Augmentation to Prevent or Reverse Alzheimer's Disease in People With Down Syndrome

NCT07278492
View directory record Official record
Conditions
Down Syndrome (DS)
Interventions
MIB-626, Placebo
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 21, 2026, 12:39 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluating Color Perception Under LED Red/Green and Green Dominant Light

NCT01927536
View directory record Official record
Conditions
Color Blindness, Acquired
Interventions
White LED Flashlight, Red/Green Green Dominant LED Flashlight
Device
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 66 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2019
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 14, 2019 · Synced Jul 21, 2026, 12:39 PM EDT
Active, not recruiting Phase 1 Interventional Accepts healthy volunteers

This Study Consists of Two Study Parts Conducted Under a Single IRB. Part I: Short Term ApoE-dependent Cerebral Blood Flow Response to Sirolimus in Cognitively Normal Adults Part II: Short Term ApoE-dependent Cerebral Blood Flow and Lung Perfusion Response to Sirolimus in Cognitively Normal Adults

NCT05386914
View directory record Official record
Conditions
Genetic Predisposition to Disease, Healthy Volunteers
Interventions
Sirolimus
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
45 Years to 65 Years
Enrollment
205 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Jul 21, 2026, 12:39 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Jul 21, 2026, 12:39 PM EDT
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, 12:39 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, 12:39 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, 12:39 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, 12:39 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Genetic Testing for Breast, Ovarian, Pancreatic, and Prostate Cancers

NCT04330716
View directory record Official record
Conditions
Breast Cancer, Ovarian Cancer, Pancreatic Cancer, Metastatic Prostate Cancer
Interventions
Standard Genetic Counseling, Educational Video
Behavioral
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
269 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
6
States / cities
Boston, Massachusetts • Brighton, Massachusetts • Milford, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2024 · Synced Jul 21, 2026, 12:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available

CNTF Implants for CNGB3 Achromatopsia

NCT01648452
View directory record Official record
Conditions
Eye Disease, Achromatopsia
Interventions
NT-501 CNTF-releasing implant
Biological
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 20, 2016 · Synced Jul 21, 2026, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 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, 12:39 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy for Achromatopsia (CNGA3)

NCT03758404
View directory record Official record
Conditions
Achromatopsia
Interventions
adeno-associated virus vector AAV- CNGA3
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 30, 2022 · Synced Jul 21, 2026, 12:39 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, 12:39 PM EDT