Clinical trials: X-Linked
Duchenne Muscular Dystrophy
Bicycle Ergometer Training in Duchenne Muscular Dystrophy
The progressive muscle weakness and contractures of the patients adversely affect their gait and balance. It is known that the disorder of the patients' balance and gait affects their functional capacity.
- Trial name
- BETDMD (NCT05330195)
- Testing
- Bicycle Ergometer Training, Home-based exercise group
- Where
- Turkey (Türkiye)
- Volunteers
- 23 people
- Sponsor
- Hacettepe University
Hypohidrotic Ectodermal Dysplasia
Characterization of Sweat Gland Function in Patients With Recessively Inherited Hypohidrotic Ectodermal Dysplasia
Hypohidrotic ectodermal dysplasia (HED) is a complex genetic disorder characterized by lack of sweat glands, sparse hair, and missing or malformed teeth.
- Trial name
- NCT01109290
- Where
- Germany
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- University Hospital Erlangen
Rett Syndrome
Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)
The overall purpose of this study is to develop a broad-based (i.e., multiple domains) behavioral outcome measure for children between the ages of 3-18 years with Rett syndrome (RTT).
- Trial name
- NCT03196323
- Where
- United States
- Volunteers
- 146 people
- Sponsor
- University of South Florida
Duchenne Muscular Dystrophy
Strength Training in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a debilitating neuromuscular disease that causes muscle breakdown, weakness, and eventual death.
- Trial name
- NCT02421523
- Testing
- Aim 2 Exercise group, Aim 1 Exercise Dosing, Aim 2 Control group
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Florida
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
X-linked Chronic Granulomatous Disease
Gene Therapy for X-CGD
X-linked chronic granulomatous disease (X-CGD) is a rare inherited immune defect, which is caused by the inability of phagocytic cells to produce reactive oxygen species due to a defect in the gp91phox subunit of the NADPH…
- Trial name
- NCT01906541
- Testing
- ex-vivo gene-therapy
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- Hubert Serve, Prof., MD
Becker Muscular Dystrophy
Sodium Nitrate for Muscular Dystrophy
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation.
- Trial name
- NCT02434627
- Testing
- Sodium Nitrate
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Cedars-Sinai Medical Center
Androgenetic Alopecia
Association of Polymorphisms in the Androgen Receptor Gene and Finasteride Response in Women With Androgenetic Alopecia
Previous studies of finasteride treatment in women with hair loss have failed to show positive results, yet, some women have responded anecdotally.
- Trial name
- NCT01052870
- Testing
- Finasteride
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- HairDx, LLC
Dent Disease
Evaluation of the Release of Monomers From Composite Bonding Resins in Orthodontics
In recent decades, the field of public health has become increasingly interested in endocrine disruptors, and their effects on humans.
- Trial name
- MONORTHO (NCT04459013)
- Testing
- Salivary samples
- Where
- France
- Volunteers
- 41 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Rett Syndrome
Lamivudine as a Novel Clinical Effort for Rett Syndrome
Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder caused primarily by mutations in the MECP2 gene, leading to progressive impairments in motor function, communication, and behavior following an initial period of…
- Trial name
- LANCE (NCT07640503)
- Testing
- Lamivudine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- Maria Denise Fernandes Carvalho de Andrade
The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome.
- Trial name
- THERIPEX (NCT07697118)
- Testing
- FOXP3-T4 drug product, ILT-101
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Developmental Dysphasia, Epileptic Encephalopathy, Childhood-Onset
CNKSR2 Natural History Study
This prospective natural history study is being conducted to define the electroclinical, neurodevelopmental, and behavioral characteristics of CNKSR2 epilepsy aphasia syndrome (EAS) and intellectual disability (ID) in children…
- Conditions
- Developmental Dysphasia, Epileptic Encephalopathy, Childhood-Onset, X-Linked Intellectual Disability
- Trial name
- CNKSR2 (NCT06500260)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Francisco
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-44 (NCT07037862)
- Testing
- ENTR-601-44
- Where
- 14 sites in Belgium, Italy and 2 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Duchenne Muscular Dystrophy
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03039686
- Testing
- RO7239361
- Where
- 44 sites in United States, Argentina and 11 more
- Volunteers
- 166 people
- Sponsor
- Hoffmann-La Roche
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Triple Negative Breast Neoplasms
Bicalutamide and Abemaciclib in Inoperable or Metastatic Androgen Receptor-positive Triple-negative Breast Cancer
This study has as goal to evaluate the use of abemaciclib and bicalutamide in androgen receptor positive metastatic triple negative breast cancer.
- Trial name
- ABBICAR (NCT06365788)
- Testing
- Abemaciclib, Bicalutamide
- Where
- 6 sites in Belgium
- Volunteers
- 53 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Fabry Disease
A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)
This study is designed to describe the metabolism of AT1001 (migalastat HCl) and the contribution of metabolism and urinary excretion to its overall elimination as part of the continuing assessment of the safety and effectiveness…
- Trial name
- NCT01730482
- Testing
- [14C] AT1001
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Duchenne Muscular Dystrophy (DMD)
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration.
- Trial name
- Acti-nRoll (NCT07664124)
- Testing
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Clinical Trial of Growth Hormone in MPS I, II, and VI
The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT00748969
- Testing
- Somatropin (DNA origin)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT06641895
- Testing
- BBM-D101
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
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