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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetic Diseases, Inborn Clear all
Completed Not applicable Interventional Results available

Genomic Sequencing for Childhood Risk and Newborn Illness

NCT02422511
View directory record Official record
Conditions
Hereditary Disease, Genetic Predisposition to Disease
Interventions
Genomic sequencing, Family history report
Genetic · Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
1,205 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Jul 23, 2026, 10:22 PM EDT
Terminated No phase listed Observational

Comparison of Bispectral Index Values in Patients With and Without Down's Syndrome

NCT02288702
View directory record Official record
Conditions
Down Syndrome
Interventions
monitoring BIS in both groups
Device
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
2 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 22, 2023 · Synced Jul 23, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

Trial of Dextromethorphan in Rett Syndrome

NCT00593957
View directory record Official record
Conditions
Rett Syndrome
Interventions
Dextromethorphan
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
2 Years to 15 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 22, 2014 · Synced Jul 23, 2026, 10:22 PM EDT
Terminated Phase 1 Interventional

Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease

NCT01552434
View directory record Official record
Conditions
Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease, Lip and Oral Cavity Carcinoma, Lymphangioleiomyomatosis, Malignant Endocrine Neoplasm, Malignant Female Reproductive System Neoplasm, Malignant Male Reproductive System Neoplasm, Malignant Neoplasm, Malignant Respiratory Tract Neoplasm, Malignant Thoracic Neoplasm, Malignant Urinary System Neoplasm, Mesothelial Neoplasm, Metastatic Malignant Neoplasm, Metastatic Urothelial Carcinoma, Neurofibromatosis Type 2, Recurrent Adult Soft Tissue Sarcoma, Recurrent Breast Carcinoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Digestive System Carcinoma, Recurrent Female Reproductive System Carcinoma, Recurrent Male Reproductive System Carcinoma, Recurrent Malignant Neoplasm, Recurrent Pharyngeal Carcinoma, Recurrent Thyroid Gland Carcinoma, Refractory Malignant Neoplasm, Soft Tissue Neoplasm, Stage III Breast Cancer AJCC v7, Stage III Pharyngeal Cancer, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Pharyngeal Cancer, Stage IVA Pharyngeal Cancer, Stage IVB Pharyngeal Cancer, Stage IVC Pharyngeal Cancer, Thyroid Gland Neoplasm
Interventions
Bevacizumab, Cetuximab, Laboratory Biomarker Analysis, Pharmacological Study, Temsirolimus, Valproic Acid
Biological · Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jul 23, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909
View directory record Official record
Conditions
Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Fludarabine, Cyclophosphamide, Anti-Thymocyte Globulin (Rabbit), The CliniMACS device, G-CSF
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Month and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Jul 23, 2026, 10:22 PM EDT
Completed No phase listed Observational

A Natural History Study of Sanfilippo Syndrome Type D

NCT05648851
View directory record Official record
Conditions
Sanfilippo Syndrome Type D
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 31, 2025 · Synced Jul 23, 2026, 10:22 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Jul 23, 2026, 10:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Specimen and Data Study for Ovarian Cancer Early Detection and Prevention

NCT00005095
View directory record Official record
Conditions
Cervical Cancer, Endometrial Cancer, Fallopian Tube Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2), Ovarian Cancer, Sarcoma, Uterine Leiomyomata, Vaginal Cancer, Vulvar Cancer
Interventions
Ca-125, screening questionnaire administration, Surgery
Other · Procedure
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 80 Years · Female only
Enrollment
6,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2040
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 10:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry

NCT06238765
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Not listed
Lead sponsor
CorEvitas
Network
Eligibility
10 Years to 15 Years
Enrollment
1,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2099
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 23, 2026, 10:22 PM EDT
Recruiting No phase listed Observational

A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400
View directory record Official record
Conditions
Fucosidosis
Interventions
Not listed
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Jul 23, 2026, 10:22 PM EDT
Withdrawn Not applicable Interventional

FFP Versus PCC in Intracranial Hemorrhage

NCT02429453
View directory record Official record
Conditions
Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
Interventions
Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma
Drug · Biological
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Nov 15, 2016 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

NSAID Drug Interaction Study

NCT01884272
View directory record Official record
Conditions
Gout
Interventions
lesinurad 400 mg, naproxen 250 mg, indomethacin 25 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Kalamazoo, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2013 · Synced Jul 23, 2026, 10:22 PM EDT
Active, not recruiting Phase 1 Interventional

Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease

NCT03664830
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Plerixafor
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 4 Interventional Results available

A Phase 4, Open-label Study of KRYSTEXXA® (Pegloticase) Co-administered With Methotrexate (MTX) in Patients With Uncontrolled Gout (FORWARD OL)

NCT04762498
View directory record Official record
Conditions
Uncontrolled Gout, Chronic Gout
Interventions
Pegloticase, Methotrexate (MTX)
Biological · Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 80 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Anchorage, Alaska • Glendale, Arizona • San Leandro, California + 9 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Jul 23, 2026, 10:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Adolescent, Caregiver, and Young Adult Perspectives of the Transition From Pediatric to Adult Care for Sickle Cell Disease: A Preliminary Evaluation of the Sickle Cell Disease Transition Program

NCT01569971
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Assessment
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Years to 30 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 23, 2014 · Synced Jul 23, 2026, 10:22 PM EDT
Completed No phase listed Observational

Screening for Studies on Inherited Eye Diseases

NCT00001732
View directory record Official record
Conditions
Hereditary Eye Disease
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
800 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 23, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome

NCT03458416
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Cannabidiol Oral Solution
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Jun 21, 2023 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049
View directory record Official record
Conditions
Mucopolysaccharidosis IIIB
Interventions
SBC-103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 20, 2018 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

NCT01703533
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
16 Years to 45 Years · Female only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
3
States / cities
Birmingham, Alabama • Saint Paul, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 23, 2026, 10:22 PM EDT
Completed No phase listed Observational

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

NCT00499187
View directory record Official record
Conditions
HIV Infections, Fanconi Syndrome, Kidney Disease, Renal Impairment
Interventions
Blood Draws
Procedure
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
10
States / cities
Los Angeles, California • Denver, Colorado • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2014 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Evaluation of Regional Ventilation Using 19F MRI of Inert Perfluorinated Gases Mixed With Oxygen

NCT01710449
View directory record Official record
Conditions
COPD, Asthma, Cystic Fibrosis, Emphysema, Small Airways Diseases
Interventions
perfluorinated gas/oxygen mixture
Drug
Lead sponsor
Hal C Charles
Other
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 8, 2013 · Synced Jul 23, 2026, 10:22 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)

NCT05107128
View directory record Official record
Conditions
Huntington's Disease
Interventions
SAGE-718, Placebo
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
34
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced Jul 23, 2026, 10:22 PM EDT