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ClinicalTrials.gov public records Last synced Oct 21, 2026, 12:53 PM EDT

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Showing 385–408 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 3 Interventional Results available

Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy

NCT00419341
View directory record Official record
Conditions
Primary Immune Deficiency
Interventions
Human Normal Immunoglobulin for Subcutaneous Administration
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
2 Years to 75 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
13
States / cities
Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2013 · Synced Oct 21, 2026, 12:53 PM EDT
Withdrawn Phase 1 Interventional

A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease

NCT02630394
View directory record Official record
Conditions
Sickle Cell Disease, Acute Chest Syndrome
Interventions
Azithromycin
Drug
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
6 Years to 16 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Jackson, Mississippi • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 21, 2018 · Synced Oct 21, 2026, 12:53 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Triple Negative Breast Cancer and Germline Hereditary Breast and Ovarian Cancer Mutation Carrier Registry

NCT02302742
View directory record Official record
Conditions
Breast Cancer, Hereditary Breast and Ovarian Cancer
Interventions
Not listed
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
11
States / cities
Hays, Kansas • Kansas City, Kansas • Overland Park, Kansas + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Terminated No phase listed Observational Results available

Efficacy of TOBI Podhaler vs. Tobramycin Inhalation Solution

NCT02038803
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
TOBIpodhaler
Drug
Lead sponsor
Rush University Medical Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 6, 2024 · Synced Oct 21, 2026, 12:53 PM EDT
Recruiting Phase 1Phase 2 Interventional

Cannabidiol in Sickle Cell Disease

NCT06930703
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Cannabidiol, Placebo
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Manhattan, New York
Source: ClinicalTrials.gov public record
Updated Jun 25, 2025 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Not applicable Interventional Results available

Blue vs Red Light During Levulan Based Photodynamic Therapy in Patients With Basal Cell Nevus Syndrome

NCT02157623
View directory record Official record
Conditions
Basal Cell Nevus Syndrome
Interventions
Levulan, Red Light PDT, Blue Light PDT
Drug · Other
Lead sponsor
Edward Maytin, MD, PhD
Other
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 21, 2026, 12:53 PM EDT
Active, not recruiting No phase listed Observational

ESIS in Pediatric DRE

NCT05469373
View directory record Official record
Conditions
Epilepsy
Interventions
Not listed
Lead sponsor
Sarah Katie Ihnen
Other
Eligibility
1 Year to 30 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Terminated Not applicable Interventional

The Effect of Low Intensity Exercise on Quality of Life and Hemodynamics in Patients With Permanent Atrial Fibrillation

NCT00680290
View directory record Official record
Conditions
Atrial Fibrillation
Interventions
Exercise
Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 16, 2019 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Phase 3 Interventional Results available

Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally

NCT01380197
View directory record Official record
Conditions
Pain, Sickle Cell Disease
Interventions
Morphine, Nubain
Drug
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
6 Years to 19 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 20, 2018 · Synced Oct 21, 2026, 12:53 PM EDT
Completed No phase listed Observational

Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease

NCT00368446
View directory record Official record
Conditions
Cystic Fibrosis, Pseudohypoaldosteronism, Nontuberculous Mycobacterial Infection, Chronic Granulomatous Disease, Primary Ciliary Dyskinesia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 65 Years
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Approved For Marketing No phase listed Expanded access

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

NCT00954460
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa
Drug
Lead sponsor
Shire
Industry
Eligibility
3 Years and older
U.S. locations
32
States / cities
Phoenix, Arizona • Beverly Hills, California • La Jolla, California + 28 more
Source: ClinicalTrials.gov public record
Updated May 21, 2021 · Synced Oct 21, 2026, 12:53 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Combination Chemotherapy With or Without Atezolizumab in Treating Patients With Stage III Colon Cancer and Deficient DNA Mismatch Repair

NCT02912559
View directory record Official record
Conditions
Colon Adenocarcinoma, DNA Repair Disorder, Lynch Syndrome, Stage III Colon Cancer AJCC v8
Interventions
Atezolizumab, Biospecimen Collection, Computed Tomography, Fluorouracil, Leucovorin Calcium, Magnetic Resonance Imaging, Oxaliplatin, Quality-of-Life Assessment
Drug · Procedure · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years and older
Enrollment
712 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1026
States / cities
Anchorage, Alaska • Fairbanks, Alaska • Phoenix, Arizona + 683 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Not listed No phase listed Observational

Identification Genetic, Immunologic and Microbial Markers of Hirschsprung Associated Enterocolitis in Children With Hirschsprung Disease

NCT02193685
View directory record Official record
Conditions
Hirschsprung Disease, Enterocolitis
Interventions
Not listed
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
1 Day to 17 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 12, 2016 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Not applicable Interventional

Investigating Trunk Control in Young Children With Down Syndrome

NCT03544840
View directory record Official record
Conditions
Down Syndrome, Gross Motor Development Delay
Interventions
Physical Therapy Home Program
Other
Lead sponsor
Texas Woman's University
Other
Eligibility
6 Months to 24 Months
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 22, 2019 · Synced Oct 21, 2026, 12:53 PM EDT
Terminated No phase listed Observational

Genetics and Clinical Characteristics of Bardet-Biedl Syndrome

NCT00078091
View directory record Official record
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Oct 21, 2026, 12:53 PM EDT
Recruiting No phase listed Observational

MEASuRE: Metreleptin Effectiveness And Safety Registry

NCT02325674
View directory record Official record
Conditions
Generalised Lipodystrophy, Partial Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2031
U.S. locations
16
States / cities
Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Recruiting Phase 1Phase 2 Interventional

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813
View directory record Official record
Conditions
Nephropathic Cystinosis
Interventions
DFT383
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
6
States / cities
Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Approved For Marketing No phase listed Expanded access

3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)

NCT00872950
View directory record Official record
Conditions
Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndrome
Interventions
3,4-DIAMINOPYRIDINE
Drug
Lead sponsor
Lahey Clinic
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Burlington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Phase 3 Interventional Results available

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
19
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Not yet recruiting Phase 1 Interventional

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

NCT07531940
View directory record Official record
Conditions
Down Syndrome, Intellectual Disability, Alzheimer Dementia (AD)
Interventions
Memantine
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years to 32 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Not applicable Interventional Results available

Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications

NCT04419168
View directory record Official record
Conditions
Chronic Pain, Depression, Anxiety, Opioid Use
Interventions
cCBT, m-Education
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
359 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
7
States / cities
Chicago, Illinois • Baltimore, Maryland • Durham, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 19, 2025 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Phase 2 Interventional

Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)

NCT05143190
View directory record Official record
Conditions
Recessive Dystrophic Epidermolysis Bullosa
Interventions
PTR-01
Drug
Lead sponsor
Phoenix Tissue Repair, Inc.
Industry
Eligibility
12 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
2
States / cities
Redwood City, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 21, 2022 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease

NCT02307513
View directory record Official record
Conditions
Behçet's Syndrome
Interventions
Apremilast, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Sacramento, California • Ormond Beach, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2021 · Synced Oct 21, 2026, 12:53 PM EDT
Completed Phase 3 Interventional

Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment

NCT00008359
View directory record Official record
Conditions
Cancer
Interventions
caspofungin acetate, liposomal amphotericin B
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 18, 2013 · Synced Oct 21, 2026, 12:53 PM EDT