Clinical Trials on X-linked Lymphoproliferative Disease

Total 14257 results

Edimer Pharmaceuticals

Completed

Phenotypic and Genetic Properties in Males at Risk for X-linked Hypohidrotic Ectodermal Dysplasia: Evaluation of an Early Diagnosis Technology and Tests to Assess Nutritional Status (ECP-013)

X-linked Hypohidrotic Ectodermal Dysplasia | Hypohidrotic Ectodermal Dysplasia

United States
Edimer Pharmaceuticals

Completed

Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia (ECP-012) (ECP-012)

X-linked Hypohidrotic Ectodermal Dysplasia | Hypohidrotic Ectodermal Dysplasia

Spain
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
Atsena Therapeutics Inc.

Recruiting

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)

X-linked Retinoschisis

United States
Viking Therapeutics, Inc.

Recruiting

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

United States, France, Germany, Italy, United Kingdom
Novartis Pharmaceuticals

Terminated

EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes (EXPLAIN)

Fragile-X Syndrome

Germany
Sheba Medical Center
ElMindA Ltd

Recruiting

Brain Network Activation and Gait and Posture in FXTAS (FXTAS-BNA)

Fragile X Associated Tremor-ataxia Syndrome | FXTAS

Israel
Marinus Pharmaceuticals
University of California, Davis; U.S. Army Medical Research and Development...

Completed

Ganaxolone Treatment in Children With Fragile X Syndrome

Fragile x Syndrome

Belgium, United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Decisional Capacity and Informed Consent in Fragile X Syndrome

Fragile X Syndrome (FXS)

United States
NeuroVia, Inc.

Unknown

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

X-Linked Adrenoleukodystrophy

United States
Children's Hospital Medical Center, Cincinnati
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Neurophysiological and Acute Pharmacological Studies in FXS Patients

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Completed

Biomarker and DNA Collection in Subjects Participating in Protocol 22003

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.
National Institutes of Health (NIH)

Completed

A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107

Fragile X Syndrome

United States
Applied Genetic Technologies Corp
Foundation Fighting Blindness

Completed

Clinical Evaluation of Patients With X-linked Retinoschisis

X-linked Retinoschisis | XLRS

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

Chronic Granulomatous Disease (CGD) | X-Linked Chronic Granulomatous Disease

United States
University of California, Davis

Completed

Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome

Fragile X Syndrome | Genetic Diseases

United States
IRCCS Fondazione Stella Maris

Recruiting

Optical Imaging as a Tool for Monitoring Brain Function in Fragile X Syndrome

Fragile X Syndrome

Italy
Specila hospital for cerebral palsy and developmental...
FRAXA Research Foundation

Recruiting

Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome

Fragile X Syndrome

Serbia
Children's Hospital Medical Center, Cincinnati
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

Fragile X Syndrome

United States
Tetra Discovery Partners

Recruiting

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Fragile X Syndrome

United States
Zynerba Pharmaceuticals, Inc.

Recruiting

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome (RECONNECT)

Fragile X Syndrome

United States, Australia, United Kingdom, Ireland
National Institute of Mental Health (NIMH)

Completed

Protein Synthesis in the Brain of Patients With Fragile X Syndrome

Healthy Volunteers | Fragile X Syndrome

United States
Zynerba Pharmaceuticals, Inc.

Enrolling by invitation

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

Fragile X Syndrome

United States, United Kingdom, New Zealand, Australia
Elizabeth Berry-Kravis
National Institute of Neurological Disorders and Stroke (NINDS); Novartis Pharmaceuticals

Completed

AFQ056 for Language Learning in Children With FXS (FX-LEARN)

Fragile X Syndrome

United States
Zynerba Pharmaceuticals, Inc.

Completed

Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) (CONNECT-FX)

Fragile X Syndrome

United States, New Zealand, Australia
Children's Hospital Medical Center, Cincinnati
Rush University Medical Center

Completed

Study of Acamprosate in Fragile x Syndrome

Fragile X Syndrome

United States
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
Stanford University

Completed

Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome

Fragile X Syndrome

United States
Parc de Salut Mar

Completed

"Using Epigallocatechin Gallate (EGCG) and Cognitive Training to Modulate Cognitive Performance in Patients With Fragile X Syndrome" (TESFX)

Fragile X Syndrome

Spain
University of California, Davis

Completed

Cognitive Training for Fragile X Syndrome

Fragile X Syndrome

United States
Université de Sherbrooke
FRAXA Research Foundation

Completed

Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome (LovaMiX)

Fragile X Syndrome

Canada
University of California, Davis
The National Fragile X Foundation

Completed

Trial of Minocycline to Treat Children With Fragile X Syndrome

Fragile X Syndrome

United States
Alcobra Ltd.

Completed

A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome

Fragile X Syndrome

United States, Israel
Novartis Pharmaceuticals

Terminated

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

Germany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Fragile X Syndrome

United States
Stanford University

Completed

An Open-Label Trial of Donepezil in Fragile X Syndrome

Fragile X Syndrome

United States
Craig Erickson
Healx Limited

Recruiting

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Fragile X Syndrome

United States
Elizabeth Berry-Kravis

Completed

Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome

Fragile X Syndrome

United States
Novartis Pharmaceuticals

Completed

Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

Fragile X Syndrome

United States, Spain
Rowan University
University of Pennsylvania; FRAXA Research Foundation

Unknown

Metformin in Patients With Fragile X

Fragile X Syndrome

United States
Hoffmann-La Roche

Completed

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Fragile X Syndrome

United States
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
Yolanda de Diego Otero

Completed

Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome (SXF-TRA152)

Fragile X Syndrome

Spain
Seaside Therapeutics, Inc.

Completed

Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Children's Hospital Medical Center, Cincinnati

Recruiting

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG

Fragile X Syndrome

United States
Esoterix Genetic Laboratories, LLC

Unknown

Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay

Fragile X Syndrome

United States
Rett Syndrome Research Trust

Not yet recruiting

Validation of Innovative Biosensors for Rett Autonomic Symptom Tracking (VIBRANT)

Rett Syndrome

United States
AB2 Bio Ltd.

Completed

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

NLRC4-MAS | XIAP Deficiency

United States, Canada, Germany
AB2 Bio Ltd.

Active, not recruiting

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension

NLRC4-MAS | XIAP Deficiency

United States, Canada, Germany

Clinical Trials on X-linked Lymphoproliferative Disease

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