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ClinicalTrials.gov public records Last synced Oct 23, 2026, 5:29 PM EDT

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Showing 409–432 of 10,333 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Completed Phase 2Phase 3 Interventional

Efficacy and Safety Study of Protein C Concentrate in Subjects With Severe Congenital Protein C Deficiency

NCT00157118
View directory record Official record
Conditions
Protein C Deficiency
Interventions
Protein C Concentrate (Human) Vapor Heated
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Not listed
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
12
States / cities
Los Angeles, California • Denver, Colorado • Atlanta, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated May 5, 2021 · Synced Oct 23, 2026, 5:29 PM EDT
Terminated Phase 1 Interventional

Single Agent and Combined Inhibition After Allogeneic Stem Cell Transplant

NCT02846376
View directory record Official record
Conditions
Acute Myeloid Leukemia and Myelodysplastic Syndrome
Interventions
Nivolumab, Ipilimumab
Drug
Lead sponsor
Hackensack Meridian Health
Other
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Hackensack, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 18, 2024 · Synced Oct 23, 2026, 5:29 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia

NCT01558076
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
8 Years to 21 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 1Phase 2 Interventional

Cholesterol in ASD: Characterization and Treatment

NCT00965068
View directory record Official record
Conditions
Autism, Asperger Disorder, PDD-NOS
Interventions
Cholesterol
Other
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
4 Years to 12 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced Oct 23, 2026, 5:29 PM EDT
Terminated Phase 1 Interventional

Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)

NCT05231668
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
SAR439459, Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
7
States / cities
Los Angeles, California • New Haven, Connecticut • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 23, 2026, 5:29 PM EDT
Recruiting Not applicable Interventional

Starlight Cardiovascular Lifeline Ductus Arteriosus Stent IDE Study

NCT07114718
View directory record Official record
Conditions
Congenital Heart Disease, Congenital Heart Disease (CHD)
Interventions
Ductus Arteriosus Stent
Device
Lead sponsor
Starlight Cardiovascular Inc
Industry
Eligibility
1 Minute to 6 Months
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
10
States / cities
Los Angeles, California • San Diego, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 23, 2026, 5:29 PM EDT
Completed No phase listed Observational

The Relationship Between Having a Positive Mindset and Exercise Capacity in Patients With Congenital Heart Disease

NCT03912025
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Relationship between positive mindset and exercise capacity
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
8 Years to 17 Years
Enrollment
90 participants
Timeline
2020 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Oct 23, 2026, 5:29 PM EDT
Completed No phase listed Observational

Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)

NCT04774289
View directory record Official record
Conditions
Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
296 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

NCT00975689
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl Cysteine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Months and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2013 · Synced Oct 23, 2026, 5:29 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors

NCT04657068
View directory record Official record
Conditions
Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Endometrial Cancer, Metastatic Colorectal Cancer, Pancreatic Ductal Adenocarcinoma, Acinar Cell Carcinoma
Interventions
ART0380, Gemcitabine, Irinotecan
Drug
Lead sponsor
Artios Pharma Ltd
Industry
Eligibility
18 Years and older
Enrollment
542 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
46
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2026 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 1 Interventional Results available

Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome

NCT02738450
View directory record Official record
Conditions
Down Syndrome
Interventions
ACI-24 low dose, ACI-24 high dose, Placebo
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
25 Years to 45 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
4
States / cities
Phoenix, Arizona • La Jolla, California • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2021 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 1 Interventional

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

NCT04176991
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 12, 2020 · Synced Oct 23, 2026, 5:29 PM EDT
Completed No phase listed Observational

Retrospective Chart Review of Subcutaneous IgG Use in Infants

NCT01166074
View directory record Official record
Conditions
Primary Immune Deficiency
Interventions
Subcutaneous IgG (SCIG)
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Plymouth, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2011 · Synced Oct 23, 2026, 5:29 PM EDT
Withdrawn Phase 2 Interventional

Trial to Evaluate Safety and Efficacy of GM-CSF /Sargramostim in Down Syndrome

NCT05482334
View directory record Official record
Conditions
Down Syndrome
Interventions
Sargramostim for Injection, Saline Placebo
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 2, 2025 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 3 Interventional Results available

Vincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms' Tumor

NCT00352534
View directory record Official record
Conditions
Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
Interventions
3-Dimensional Conformal Radiation Therapy, Biospecimen Collection, Chest Radiography, Computed Tomography, Dactinomycin, Doxorubicin Hydrochloride, Echocardiography Test, Magnetic Resonance Imaging, Therapeutic Conventional Surgery, Ultrasound Imaging, Vincristine Sulfate
Radiation · Procedure · Biological + 1 more
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 29 Years
Enrollment
808 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2026
U.S. locations
210
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 150 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 23, 2026, 5:29 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Breath Condensate Study in Patients With Cystic Fibrosis.

NCT02056132
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Exhaled Breath Condensate
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 25, 2023 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 2 Interventional

The Role of Topically Dissolved Oxygen (TDO) to Ameliorate Signs of Photodamage

NCT00991198
View directory record Official record
Conditions
Rosacea, Acne Vulgaris, Atopic Dermatitis, Seborrheic Dermatitis
Interventions
Topical oxygen, placebo
Drug
Lead sponsor
Aria Aesthetics Inc.
Industry
Eligibility
25 Years to 60 Years · Female only
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
High Point, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2011 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 2 Interventional

Campath-1H + FK506 and Methylprednisolone for GVHD

NCT00109993
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, tacrolimus
Biological · Drug
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
6
States / cities
New York, New York • Cleveland, Ohio • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2010 · Synced Oct 23, 2026, 5:29 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Inhaled Sodium Nitrite as an Antimicrobial for Cystic Fibrosis

NCT02694393
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
sodium nitrite
Drug
Lead sponsor
Schmidhofer, Mark, MD
Individual
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2024 · Synced Oct 23, 2026, 5:29 PM EDT
Terminated No phase listed Observational

Percutaneous Interventions in Adults With CHD

NCT00266916
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2006
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 23, 2007 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Phase 2 Interventional Results available

Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT01707004
View directory record Official record
Conditions
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, 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), de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia
Interventions
decitabine, fludarabine phosphate, busulfan, cyclophosphamide, tacrolimus, mycophenolate mofetil, filgrastim, total-body irradiation, allogeneic bone marrow transplantation, laboratory biomarker analysis
Drug · Biological · Radiation + 2 more
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 21, 2019 · Synced Oct 23, 2026, 5:29 PM EDT
Completed Not applicable Interventional

CTICU Pacifier Activated Music Player and Mother's Voice

NCT03035552
View directory record Official record
Conditions
Heart Defects, Congenital
Interventions
Pacifier activated music player
Behavioral
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
0 Months to 6 Months
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 23, 2026, 5:29 PM EDT
Terminated Phase 4 Interventional

Post-approval Study of the DURALOC® Option Ceramic-on-Ceramic Hip Prosthesis System

NCT00546598
View directory record Official record
Conditions
Non-inflammatory Joint Disease, Osteoarthritis, Avascular Necrosis, Congenital Hip Dysplasia, Post-traumatic Arthritis
Interventions
Total hip replacement/arthroplasty
Device
Lead sponsor
DePuy Orthopaedics
Industry
Eligibility
21 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
6
States / cities
Atlanta, Georgia • Springfield, Illinois • Cleveland, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2021 · Synced Oct 23, 2026, 5:29 PM EDT