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ClinicalTrials.gov public records Last synced Oct 10, 2026, 8:53 AM EDT

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Showing 25–48 of 2,393 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hereditary Conditions Clear all
Terminated Phase 2 Interventional Results available

Rasburicase and Allopurinol in Treating Patients With Hematologic Malignancies

NCT01564277
View directory record Official record
Conditions
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), Blastic Phase Chronic Myelogenous Leukemia, Contiguous Stage II Adult Burkitt Lymphoma, de Novo Myelodysplastic Syndromes, Noncontiguous Stage II Adult Burkitt Lymphoma, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Stage I Adult Burkitt Lymphoma, Stage III Adult Burkitt Lymphoma, Stage IV Adult Burkitt Lymphoma, Untreated Adult Acute Lymphoblastic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
rasburicase, allopurinol
Drug
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 10, 2026, 8:53 AM EDT
Terminated Phase 2 Interventional Results available

A Two-week Open-label Pharmacodynamic and Pharmacokinetic Study of Multiple Doses of a Darifenacin Liquid Oral Suspension in Children (2 - 15 Years) With Neurogenic Detrusor Overactivity

NCT00712322
View directory record Official record
Conditions
Neurogenic Detrusor Overactivity
Interventions
Darifenacin
Drug
Lead sponsor
Warner Chilcott
Industry
Eligibility
2 Years to 15 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
9
States / cities
San Diego, California • Boston, Massachusetts • Detroit, Michigan + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Oct 10, 2026, 8:53 AM EDT
Terminated No phase listed Observational

MPS II Immunophenotyping

NCT04976231
View directory record Official record
Conditions
MPS II, Mucopolysaccharidosis II, Hunter Syndrome
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 10, 2026, 8:53 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Eltrombopag for Fanconi Anemia

NCT03206086
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
6 Years to 99 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Completed No phase listed Observational

Development of a Next Generation Sequencing (NGS) -Based Assay to Detect Preeclampsia Molecular Markers

NCT02808494
View directory record Official record
Conditions
Preeclampsia
Interventions
Not listed
Lead sponsor
Illumina, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
242 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
10
States / cities
Newark, Delaware • Boston, Massachusetts • New Brunswick, New Jersey + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2022 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting Phase 4 Interventional

Tirzepatide in PWS, HO and GNSO

NCT06901245
View directory record Official record
Conditions
Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
Interventions
Tirzepatide
Drug
Lead sponsor
Grace Kim
Other
Eligibility
18 Years to 26 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Saint Paul, Minnesota • Nashville, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 16, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Hyperpolarized Noble Gas MR Imaging for Pulmonary Disorders

NCT00846573
View directory record Official record
Conditions
COPD, Cystic Fibrosis, Asthma, Healthy
Interventions
Hyperpolarized Helium-3
Drug
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
5 Years and older
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2011
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 15, 2012 · Synced Oct 10, 2026, 8:53 AM EDT
Completed Phase 1 Interventional

A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington Disease

NCT01485965
View directory record Official record
Conditions
Huntington's Disease
Interventions
SEN0014196
Drug
Lead sponsor
Siena Biotech S.p.A.
Industry
Eligibility
25 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
7
States / cities
La Jolla, California • Sacramento, California • St Louis, Missouri + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2013 · Synced Oct 10, 2026, 8:53 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCT06109181
View directory record Official record
Conditions
Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
Interventions
LX2020
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Stanford, California • Baltimore, Maryland • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting Not applicable Interventional

Fetal Endoscopic Tracheal Occlusion (FETO) for Severe Congenital Diaphragmatic Hernia

NCT05461222
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Balt "Goldballoon"
Device
Lead sponsor
Yair Blumenfeld
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting Not applicable Interventional

Forehead Temperature-Regulating Therapy for Insomnia in Adults With Tourette's Disorder

NCT05499741
View directory record Official record
Conditions
Tourette's Disorder, Insomnia
Interventions
Forehead Temperature-Regulating Therapy
Device
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Enrolling by invitation No phase listed Observational

RNA to the RESCUE: Evaluation to Assess the Clinical Utility of RNA Sequencing in Establishing a Genetic Diagnosis or Adjudicating a Previously Established Genetic Diagnosis.

NCT07787975
View directory record Official record
Conditions
Genetic Conditions, Genetic Disorders
Interventions
RNA sequencing
Device
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

NCT05462106
View directory record Official record
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT07601425
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
Interventions
ATV-1601, Placebo
Drug
Lead sponsor
Atavistik Bio, Inc
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2030
U.S. locations
6
States / cities
Phoenix, Arizona • Augusta, Georgia • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers

The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Very Low Birth Weight Infants

NCT01203514
View directory record Official record
Conditions
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Anemia, Neonatal
Interventions
Erythropoietin, Sham Comparator
Drug · Other
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
24 Hours to 96 Days
Enrollment
318 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1997 – 2000
U.S. locations
9
States / cities
New Haven, Connecticut • Washington D.C., District of Columbia • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2017 · Synced Oct 10, 2026, 8:53 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Lung Ultrasound to Diagnose Transient Tachypnea of the Newborn (TTN) Versus Respiratory Distress Syndrome (RDS) in Neonates

NCT01517958
View directory record Official record
Conditions
Transient Tachypnea of the Newborn, TTN, Respiratory Distress Syndrome, RDS
Interventions
Respiratory Distress Group, Control Group
Other
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
28 Weeks and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2016 · Synced Oct 10, 2026, 8:53 AM EDT
Terminated Phase 2 Interventional Results available

Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers

NCT02863068
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Topical Sodium Nitrite, Placebo
Drug · Other
Lead sponsor
caterina P minniti
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
3
States / cities
The Bronx, New York • Durham, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of PI3Kinase Inhibition (Copanlisib) and Anti-PD-1 Antibody Nivolumab in Relapsed/Refractory Solid Tumors With Expansions in Mismatch-repair Proficient (MSS) Colorectal Cancer

NCT03711058
View directory record Official record
Conditions
Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
Interventions
Copanlisib, Nivolumab
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Completed No phase listed Observational

Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders

NCT03967743
View directory record Official record
Conditions
Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease, Development, Infant, Development, Child
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 4 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 10, 2026, 8:53 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography

NCT01683825
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F-18 florbetapir PET
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 10, 2026, 8:53 AM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Safety and Efficacy Study of Intramuscular Uricase-PEG 20

NCT01038947
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Uricase-PEG 20
Biological
Lead sponsor
EnzymeRx
Industry
Eligibility
40 Years to 75 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
2
States / cities
Miami, Florida • Duncansville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 24, 2009 · Synced Oct 10, 2026, 8:53 AM EDT
Completed Phase 2 Interventional

Fludarabine Phosphate, Busulfan, and Anti-Thymocyte Globulin Followed By Donor Peripheral Blood Stem Cell Transplant, Tacrolimus, and Methotrexate in Treating Patients With Myeloid Malignancies

NCT01056614
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, 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), Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Hematopoietic/Lymphoid Cancer, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Relapsing Chronic Myelogenous Leukemia
Interventions
fludarabine phosphate, busulfan, anti-thymocyte globulin, tacrolimus, methotrexate, peripheral blood stem cell transplantation, allogeneic hematopoietic stem cell transplantation, laboratory biomarker analysis
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 60 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 6, 2016 · Synced Oct 10, 2026, 8:53 AM EDT
Recruiting No phase listed Observational

Adaptive Optics Imaging of Outer Retinal Diseases

NCT05355415
View directory record Official record
Conditions
Retinal Degeneration, Age-Related Macular Degeneration, Retinitis Pigmentosa, Hydroxychloroquine Retinopathy, Usher Syndromes, Late-Onset Retinal Degeneration, Cone Dystrophy, Cone Rod Dystrophy, Rod Cone Dystrophy, Rod Dystrophy
Interventions
Adaptive optics imaging
Device
Lead sponsor
Food and Drug Administration (FDA)
Federal
Eligibility
21 Years and older
Enrollment
100 participants
Timeline
2021 – 2028
U.S. locations
2
States / cities
Bethesda, Maryland • Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 10, 2026, 8:53 AM EDT