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Showing 1–24 of 55 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Skeletal Malformation Clear all
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03197766
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years to 18 Years
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
11
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2022 · Synced Jul 22, 2026, 2:08 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Skeletal Muscle Effects of GH in Boys

NCT04020913
View directory record Official record
Conditions
Growth Hormone Deficiency, Idiopathic Short Stature
Interventions
Somatropin injection
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
6 Years to 11 Years · Male only
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Jul 22, 2026, 2:08 PM EDT
Recruiting Phase 2Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

NCT07441876
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 333, Vosoritide Injection [Voxzogo]
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
2 Years to 17 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
9
States / cities
Oakland, California • Wilmington, Delaware • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Active, not recruiting Phase 2 Interventional

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03989947
View directory record Official record
Conditions
Achondroplasia
Interventions
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Months and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2038
U.S. locations
9
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 1, 2018 · Synced Jul 22, 2026, 2:08 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

NCT02972580
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Genetic characterization
Genetic
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
250 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 26, 2024 · Synced Jul 22, 2026, 2:08 PM EDT
Terminated Phase 2 Interventional Results available

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

NCT04638153
View directory record Official record
Conditions
Achondroplasia
Interventions
Recifercept
Biological
Lead sponsor
Pfizer
Industry
Eligibility
3 Months to 10 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
6
States / cities
Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 14, 2024 · Synced Jul 22, 2026, 2:08 PM EDT
Completed Phase 4 Interventional Results available

Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome

NCT00266656
View directory record Official record
Conditions
Turner Syndrome
Interventions
Humatrope
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
4 Years to 20 Years · Female only
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2015
U.S. locations
9
States / cities
Los Angeles, California • Aurora, Colorado • Hartford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2017 · Synced Jul 22, 2026, 2:08 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

NCT07362875
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Completed No phase listed Observational

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

NCT04035811
View directory record Official record
Conditions
Achondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 17 Years
Enrollment
271 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Completed Phase 2 Interventional

Bone Response to Enzyme Replacement in Gaucher's Disease

NCT00001416
View directory record Official record
Conditions
Gaucher's Disease
Interventions
CEREDASE™
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2016 · Synced Jul 22, 2026, 2:08 PM EDT
Completed No phase listed Observational

Genetic Analysis of the Chiari I Malformation

NCT00004738
View directory record Official record
Conditions
Syringomyelia, Type I Arnold Chiari Malformation
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Active, not recruiting Phase 3 Interventional

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03424018
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
6 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2031
U.S. locations
11
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced Jul 22, 2026, 2:08 PM EDT
Terminated Phase 2 Interventional Results available

Pharmacologically-based Strategies for Opioid Substitution Therapy During Pregnancy

NCT03831113
View directory record Official record
Conditions
Opiate Addiction, Pregnancy
Interventions
Magnitude Group, Frequency Group
Drug
Lead sponsor
Steve N. Caritis, MD
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 28, 2024 · Synced Jul 22, 2026, 2:08 PM EDT
Recruiting No phase listed Observational

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 16 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Recruiting Phase 2 Interventional

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
3
States / cities
La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Completed Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

NCT06164951
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 17 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jul 22, 2026, 2:08 PM EDT
Enrolling by invitation Phase 2 Interventional

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05145010
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Jul 22, 2026, 2:08 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Heart and Muscle Metabolism in Barth Syndrome

NCT01625663
View directory record Official record
Conditions
Barth Syndrome
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
8 Years to 35 Years · Male only
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 1, 2020 · Synced Jul 22, 2026, 2:08 PM EDT
Completed Not applicable Interventional Results available

Inflammatory Abnormalities in Muscle After Stroke: Effects of Exercise

NCT00387712
View directory record Official record
Conditions
Stroke, Inflammation
Interventions
Velocity based treadmill training, Duration based treadmill training
Other
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
40 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 13, 2017 · Synced Jul 22, 2026, 2:08 PM EDT
Completed No phase listed Observational

Study of Skeletal Disorders and Short Stature

NCT00001754
View directory record Official record
Conditions
Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 2:08 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

NCT06508021
View directory record Official record
Conditions
Fibrodysplasia Ossificans Progressiva
Interventions
Andecaliximab, Placebo
Drug
Lead sponsor
Ashibio Inc
Industry
Eligibility
2 Years and older
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
3
States / cities
San Francisco, California • Rochester, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Jul 22, 2026, 2:08 PM EDT
Active, not recruiting No phase listed Observational

Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy

NCT01851447
View directory record Official record
Conditions
Genetic Disorder
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 2:08 PM EDT