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Results for “Genetic Skeletal Diseases”

86 trials

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Showing 20 of 86 results

Testing effectiveness (Phase 2)Enrolling By InvitationNCT07393373
What this trial is testing

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Who this might be right for
Hypochondroplasia
QED Therapeutics, a BridgeBio company 135
Testing effectiveness (Phase 2)Ended earlyNCT05116046
What this trial is testing

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Who this might be right for
Achondroplasia
Pfizer 35
Not applicableUnknownNCT03310502
What this trial is testing

Variation of Genes Controlling Carbohydrate and Lipid Metabolism

Who this might be right for
Diabetes Mellitus, Type 2Cardiovascular DiseasesObesity+3 more
Turku University Hospital 2,050
Not applicableStudy completedNCT01451281
What this trial is testing

Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

Who this might be right for
Muscular DystrophyMuscular Disease
National Institute of Neurological Disorders and Stroke (NINDS) 35
Very early researchLooking for participantsNCT07188012
What this trial is testing

Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Shanghai Siponuoyin Biotechnology Co Ltd 9
Not applicableStudy completedNCT04035811
What this trial is testing

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 271
Not applicableStudy completedNCT05181839
What this trial is testing

Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Who this might be right for
X-Linked Hypophosphatemia
Kyowa Kirin Pharmaceutical Development Ltd 25
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Not applicableLooking for participantsNCT07378553
What this trial is testing

Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies

Who this might be right for
Duchene Muscular DystrophyBecker Muscular Dystrophy
Nantes University Hospital 60
Not applicableLooking for participantsNCT05876416
What this trial is testing

Decoding the Genetic Landscape of Skeletal Diseases

Who this might be right for
Genetic Skeletal DiseasesSkeletal DysplasiaMolecular Causes+1 more
Karolinska Institutet 450
Not applicableActive Not RecruitingNCT05991609
What this trial is testing

Extreme Morphology and Metabolic Health

Who this might be right for
Skeletal Dysplasia
University of Bath 102
Not applicableLooking for participantsNCT05247645
What this trial is testing

Data Collection of Patients With Rare Bone Diseases

Who this might be right for
Skeletal Dysplasia
Luca Sangiorgi 1,000
Large-scale testing (Phase 3)Study completedNCT06164951
What this trial is testing

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

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 114
Not applicableStudy completedNCT00004738
What this trial is testing

Genetic Analysis of the Chiari I Malformation

Who this might be right for
SyringomyeliaType I Arnold Chiari Malformation
National Institute of Neurological Disorders and Stroke (NINDS) 152
Testing effectiveness (Phase 2)Looking for participantsNCT06092034
What this trial is testing

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

Who this might be right for
Danon Disease
Rocket Pharmaceuticals Inc. 14
Testing effectiveness (Phase 2)Study completedNCT03583697
What this trial is testing

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Who this might be right for
Achondroplasia
BioMarin Pharmaceutical 75
Testing effectiveness (Phase 2)Active Not RecruitingNCT03989947
What this trial is testing

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

Who this might be right for
Achondroplasia
BioMarin Pharmaceutical 73
Testing effectiveness (Phase 2)Study completedNCT01856868
What this trial is testing

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

Who this might be right for
Becker Muscular Dystrophy
Craig McDonald, MD 7
Not applicableStudy completedNCT00001754
What this trial is testing

Study of Skeletal Disorders and Short Stature

Who this might be right for
Developmental Bone DiseaseDwarfismSkeletal Dysplasias
National Human Genome Research Institute (NHGRI) 600
Not applicableUnknownNCT04332302
What this trial is testing

Power Strength Training in Type 2 Diabetic Mellitus Older Adults

Who this might be right for
Diabetes Mellitus, Type 2Frailty
Technical University of Madrid 41
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