Clinical Trials on X-linked Adrenoleukodystrophy

Total 2359 results

Beacon Therapeutics

Active, not recruiting

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (HORIZON)

X-Linked Retinitis Pigmentosa

United States
Beacon Therapeutics

Active, not recruiting

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

X-Linked Retinitis Pigmentosa

United States
Beacon Therapeutics

Enrolling by invitation

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

X-Linked Retinitis Pigmentosa

United States
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

United States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
Biogen

Completed

A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 (XIRIUS)

X-Linked Retinitis Pigmentosa

United States, United Kingdom
4D Molecular Therapeutics

Active, not recruiting

4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

X-Linked Retinitis Pigmentosa

United States
NightstaRx Ltd, a Biogen Company

Completed

Natural History of the Progression of Choroideremia Study (NIGHT)

Choroideremia

Finland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
Applied Genetic Technologies Corp

Completed

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

X-Linked Retinitis Pigmentosa

United States
Takeda

Recruiting

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Hunter Syndrome

India
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Takeda

Completed

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

Brazil
Green Cross Corporation

Completed

Safety and Efficacy of Hunterase (GC1111)

Hunter Syndrome

Korea, Republic of
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

Belgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
University Hospital, Montpellier
Association Xtraordinaire sub-group DDX3X; Genida

Unknown

Better Delineation of DDX3X Related Phenotype and Epigenetic Signature.

Intellectual Developmental Disorder | X-LINKED

France
Beacon Therapeutics

Recruiting

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

X-Linked Retinitis Pigmentosa

United States
Spark Therapeutics

Completed

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Choroideremia | CHM (Choroideremia) Gene Mutations

United States
Uskudar University

Recruiting

Physiotherapy Assessment Based on the ICF Model in The Lesch-Nyhan Syndrome: Case Report

Lesch-Nyhan Syndrome

Turkey
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Lundquist Institute for Biomedical Innovation at...

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI

United States
Shire

Completed

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Green Cross Corporation

Unknown

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Hunter Syndrome

Korea, Republic of
Janssen Research & Development, LLC

Active, not recruiting

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

X-Linked Retinitis Pigmentosa

United States, United Kingdom
Eunice Kennedy Shriver National Institute of Child...

Terminated

The Natural History of Reproductive and Overall Health in Girls and Women With a Pre-Mutation in the FMR1 Gene; Creation of a Patient Registry

Fragile X Syndrome | Premature Ovarian Failure | Premature Menopause | FMR1 Premutation | Primary Ovairan Insufficiency

United States
Duke University

Recruiting

MPS II Immunophenotyping

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

United States
Shire

Completed

Hunter Outcome Survey (HOS) (HOS)

Hunter Syndrome

United States
National Institute of Arthritis and Musculoskeletal...

Completed

National Registry for Ichthyosis and Related Disorders

Ichthyosis | Ichthyosis, X-Linked | Ichthyosis, Lamellar | Darier Disease | Hailey-Hailey Disease | Hyperkeratosis, Epidermolytic | Keratoderma

United States
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
National Taiwan University Hospital

Unknown

The Role of Probiotics PS128 in Movement Disorders

Rett Syndrome | Tourette Syndrome | Tic Disorders

Taiwan
University Hospital, Montpellier
Agence de La Biomédecine

Active, not recruiting

NIPD on cffDNA for Triplet Repeat Diseases

Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy 1

France
REGENXBIO Inc.

Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Brazil
Kyowa Kirin Co., Ltd.

Active, not recruiting

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

X-linked Hypophosphatemia (XLH)

China
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

X-Linked Hypophosphatemia

Spain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
Shire

Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Hunter Syndrome | Mucopolysaccharidosis (MPS)

United States, Argentina, Mexico, Spain, United Kingdom
National Institute of Neurological Disorders and...

Completed

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

Motor Neuron Disease

United States
Psyadon Pharma

Completed

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

Lesch-Nyhan Disease

United States
NightstaRx Ltd, a Biogen Company

Completed

Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS)

X-Linked Retinitis Pigmentosa

United States, France, United Kingdom, Finland, Netherlands, Germany, Canada
University of Oxford
University College, London; University of Southampton; Moorfields Eye Hospital... and other collaborators

Completed

Gene Therapy for Blindness Caused by Choroideremia

Choroideremia

United Kingdom
University of British Columbia
Vancouver General Hospital

Withdrawn

Cannabinoids in Bipolar Affective Disorder

Bipolar Affective Disorder

Canada
Takeda
Takeda Development Center Americas, Inc.

Recruiting

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Hunter Syndrome | Mucopolysaccharidosis (MPS)

United States
Kyowa Kirin Pharmaceutical Development Ltd

Recruiting

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

X-Linked Hypophosphatemia

France, Netherlands, United Kingdom
National Institute of Neurological Disorders and...

Recruiting

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

Motor Neuron Disease | Spinal and Bulbar Muscular Atrophy | Kennedys Disease

United States
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

Korea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
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
Medical University of Graz

Completed

Evaluation of an App for Smartphones for People With a Bipolar Affective Disorder

Bipolar Affective Disorder

Austria
REGENXBIO Inc.

Active, not recruiting

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Canada
Kyowa Kirin Co., Ltd.

Active, not recruiting

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

X-linked Hypophosphatemia (XLH)

China

Clinical Trials on X-linked Adrenoleukodystrophy

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