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ClinicalTrials.gov public records Last synced Oct 23, 2026, 11:33 AM EDT

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Showing 1–24 of 1,707 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Multiple Abnormalities Clear all
Recruiting No phase listed Observational

A Natural History Study of Angelman Syndrome

NCT07417137
View directory record Official record
Conditions
Angelman Syndrome, Neurodevelopmental Conditions
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
1 Year and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 11:33 AM EDT
Active, not recruiting Not applicable Interventional

Home Based Tele-exercise for People With Chronic Neurological Impairments

NCT04564495
View directory record Official record
Conditions
Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy, Hemiplegia, Hemiparesis, Multiple Sclerosis, Parkinson Disease, Trauma, Brain, Transverse Myelitis, Friedreich Ataxia
Interventions
Seated exercise
Behavioral
Lead sponsor
Burke Medical Research Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
White Plains, New York
Source: ClinicalTrials.gov public record
Updated Feb 5, 2025 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

NCT01736475
View directory record Official record
Conditions
Hemophilia A
Interventions
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
17
States / cities
Aurora, Colorado • Gainesville, Florida • Atlanta, Georgia + 14 more
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Not applicable Interventional

A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS

NCT00002257
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
12
States / cities
Berkeley, California • San Francisco, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 11:33 AM EDT
Completed No phase listed Observational

Total Temporomandibular Joint Replacement System Post Approval Study

NCT00762944
View directory record Official record
Conditions
Arthroplasty
Interventions
Biomet TMJ Replacement System
Device
Lead sponsor
Zimmer Biomet
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
4
States / cities
Indianapolis, Indiana • West Bloomfield, Michigan • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2024 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 3 Interventional Results available

Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT00456365
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
pravastatin, Placebo
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
8 Years to 22 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 9, 2018 · Synced Oct 23, 2026, 11:33 AM EDT
Recruiting No phase listed Observational

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

NCT06193928
View directory record Official record
Conditions
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Interventions
Livmarli
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 23, 2026, 11:33 AM EDT
Completed No phase listed Observational

A Genetic Analysis of Usher Syndrome in Ashkenazi Jews

NCT00016471
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa, Congenital Hearing Impairment
Interventions
Audiogram, Electroretinogram
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2002
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10

NCT03294707
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
AG10 oral tablet, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 55 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 3 Interventional Results available

Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency

NCT02604810
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
IGIV-C 10%, IGSC 20%
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
2 Years to 75 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
20
States / cities
Los Angeles, California • Santa Monica, California • Denver, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2019 · Synced Oct 23, 2026, 11:33 AM EDT
Completed No phase listed Observational

A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

NCT03875534
View directory record Official record
Conditions
Achondroplasia
Interventions
Not listed
Lead sponsor
Ascendis Pharma A/S
Industry
Eligibility
0 Years to 8 Years
Enrollment
260 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
10
States / cities
Little Rock, Arkansas • Oakland, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 19, 2024 · Synced Oct 23, 2026, 11:33 AM EDT
Active, not recruiting Not applicable Interventional

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT06139172
View directory record Official record
Conditions
Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions
Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 2 Interventional Results available

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

NCT00865904
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-809, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
18
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2015 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 4 Interventional Results available

A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)

NCT04320316
View directory record Official record
Conditions
Epidermal Nevus Syndrome
Interventions
Crysvita (burosumab-twza) Treatment
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
6 Months and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 7, 2022 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

NCT04722887
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Miami, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B

NCT01386528
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
nonacog beta pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
13 Years to 70 Years · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
7
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 23, 2017 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Not applicable Interventional

Donor Stem Cell Transplant in Treating Older or Frail Patients With Hematologic Cancer

NCT00296023
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, filgrastim, therapeutic allogeneic lymphocytes, busulfan, fludarabine phosphate, methotrexate, tacrolimus, nonmyeloablative allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 75 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2008
U.S. locations
2
States / cities
Berkeley, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2012 · Synced Oct 23, 2026, 11:33 AM EDT
Enrolling by invitation Phase 1 Interventional

Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

NCT05301361
View directory record Official record
Conditions
Intellectual Disability, Fragile X Syndrome, Down Syndrome, Attention Deficit Hyperactivity Disorder
Interventions
Methylphenidate Oral Solution
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
6 Years to 24 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

NCT00891202
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
6
States / cities
San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2017 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 3 Interventional

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

NCT00006145
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 2 Interventional Results available

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

NCT01396239
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AVI-4658 (Eteplirsen), Placebo
Drug · Other
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

NCT02273596
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
4
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2021 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of a Decision Assisting Tool for Prenatal Testing

NCT00686062
View directory record Official record
Conditions
Pregnancy
Interventions
Control - computerized age-appropriate prenatal testing educational brochure, PT Tool - interactive, computerized, prenatal testing decision tool
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
16 Years to 50 Years · Female only
Enrollment
494 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2004
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 29, 2008 · Synced Oct 23, 2026, 11:33 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin

NCT02592954
View directory record Official record
Conditions
Epidermolysis Bullosa Simplex, Pachyonychia Congenita
Interventions
Jojoba oil with broccoli sprout extract, Jojoba oil (placebo)
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 6, 2017 · Synced Oct 23, 2026, 11:33 AM EDT