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ClinicalTrials.gov public records Last synced Oct 6, 2026, 7:06 PM EDT

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Showing 49–72 of 10,522 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Completed Phase 2 Interventional Results available

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

NCT01530659
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
Interventions
NT-501, Sham
Drug · Procedure
Lead sponsor
Neurotech Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 14, 2023 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 3 Interventional Results available

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

NCT05139810
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Donidalorsen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
15
States / cities
Scottsdale, Arizona • San Diego, California • Santa Monica, California + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2025 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 3 Interventional Results available

A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn

NCT01720524
View directory record Official record
Conditions
Pulmonary Hypertension, Familial Persistent, of the Newborn
Interventions
placebo, iv sildenafil
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
0 Days to 4 Days
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
20
States / cities
Little Rock, Arkansas • Sacramento, California • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2021 · Synced Oct 6, 2026, 7:06 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

The INFORM Study: Rotational Fractional Resection for Submental Contouring

NCT03853980
View directory record Official record
Conditions
Skin Laxity
Interventions
Focal Contouring System
Device
Lead sponsor
Recros Medica, Inc.
Industry
Eligibility
30 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
14
States / cities
Campbell, California • Encino, California • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Not applicable Interventional

Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?

NCT03581682
View directory record Official record
Conditions
Marfan Syndrome
Interventions
Tele-Clinic Visits Using Parent-Acquired Echos
Other
Lead sponsor
Stanford University
Other
Eligibility
5 Years to 19 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluation and Treatment of Pulmonary Vascular Disease in Moderate to Severe CF

NCT02626182
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
sildenafil, placebo
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 24, 2019 · Synced Oct 6, 2026, 7:06 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Oct 6, 2026, 7:06 PM EDT
Terminated Phase 3 Interventional Results available

The Hemophilia Inhibitor Prevention Trial

NCT04303559
View directory record Official record
Conditions
Hemophilia A Without Inhibitor
Interventions
Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
Drug
Lead sponsor
Margaret Ragni
Other
Eligibility
4 Months to 4 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
3
States / cities
Little Rock, Arkansas • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 11, 2023 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 6, 2026, 7:06 PM EDT
Not yet recruiting Phase 2 Interventional

Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

NCT07328529
View directory record Official record
Conditions
Aggressive Behavior in Fragile X Syndome
Interventions
SRX246, Placebo
Drug
Lead sponsor
Azevan Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

NCT02247739
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Recombinant human C1 inhibitor, Placebo
Biological · Other
Lead sponsor
Pharming Technologies B.V.
Industry
Eligibility
13 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
4
States / cities
Tampa, Florida • St Louis, Missouri • Lake Oswego, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 8, 2017 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

NCT02055157
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years to 14 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Oakland, California • Torrance, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2021 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional

Phase 2b Study of EVO756 in Adults With Atopic Dermatitis

NCT07150845
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
EVO756, Placebo control
Drug
Lead sponsor
Evommune, Inc.
Industry
Eligibility
18 Years and older
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
24
States / cities
Phoenix, Arizona • Fremont, California • Los Angeles, California + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional Results available

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

NCT01756157
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
CINRYZE with rHuPH20
Biological
Lead sponsor
Shire
Industry
Eligibility
12 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013
U.S. locations
17
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bentonville, Arkansas + 14 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2021 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of BAX 888 in Male Adults With Severe Hemophilia A

NCT03370172
View directory record Official record
Conditions
Hemophilia A
Interventions
BAX 888
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 6, 2026, 7:06 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 23, 2014 · Synced Oct 6, 2026, 7:06 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome

NCT00004345
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 6, 2026, 7:06 PM EDT
Active, not recruiting No phase listed Observational

Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease

NCT06747572
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
Not listed
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 65 Years
Enrollment
401 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
27
States / cities
Alabaster, Alabama • Birmingham, Alabama • Los Angeles, California + 22 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 1 Interventional

Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)

NCT05618301
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Motixafortide, Natalizumab, Leukapheresis
Drug · Procedure
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Oct 6, 2026, 7:06 PM EDT
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional

Alemtuzumab and Glucocorticoids in Treating Newly Diagnosed Acute Graft-Versus-Host Disease in Patients Who Have Undergone a Donor Stem Cell Transplant

NCT00410657
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
alemtuzumab, methylprednisolone, prednisone, flow cytometry, immunologic technique, pharmacological study
Biological · Drug · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 14, 2010 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 2 Interventional Results available

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

NCT03061201
View directory record Official record
Conditions
Hemophilia A
Interventions
SB-525 (PF-07055480)
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
22
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Examining the Effects of a Mobilization With Movement and a Dynamic Stretch With Over Pressure on Hip Flexion.

NCT04762212
View directory record Official record
Conditions
Hip; Anomaly
Interventions
Mobilization with movement and stretching, stretching or control
Procedure
Lead sponsor
University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated Dec 2, 2021 · Synced Oct 6, 2026, 7:06 PM EDT
Completed Phase 1 Interventional Results available

Understanding Dexmedetomidine in Neonates After Open Heart Surgery

NCT00576381
View directory record Official record
Conditions
Tetrology of Fallot, Heart Ventricle, Hypoplastic Left Heart
Interventions
Dexmedetomidine
Drug
Lead sponsor
Athena Zuppa
Other
Eligibility
1 Hour to 30 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 6, 2026, 7:06 PM EDT