Clinical Trials on X-Linked Hypophosphatemia

Total 2572 results

JCR Pharmaceuticals Co., Ltd.

Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Mucopolysaccharidosis II

United States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
Denali Therapeutics Inc.

Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

United States, Netherlands, Canada, United Kingdom
Denali Therapeutics Inc.

Enrolling by invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Mucopolysaccharidosis II

United States, Canada, Belgium, Czechia, Netherlands
JCR Pharmaceuticals Co., Ltd.

Enrolling by invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Mucopolysaccharidosis II

United States
Rocket Pharmaceuticals Inc.

Active, not recruiting

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Danon Disease

United States
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Japan
REGENXBIO Inc.

Completed

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Brazil, United States, United Kingdom
REGENXBIO Inc.

Enrolling by invitation

Long-term Follow-Up for RGX-121

Mucopolysaccharidosis II

United States
National Human Genome Research Institute (NHGRI)

Completed

Predictors of Caregiver Adaptation to Pervasive Developmental Disorders

Autism | Asperger's Syndrome | Rett's Disorder

United States
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Mucopolysaccharidosis II

United States, Germany, Philippines
Soma Jyonouchi
Baylor College of Medicine

Completed

Interleukin-2 Treatment for Wiskott-Aldrich Syndrome (WAS)

X-linked Thrombocytopenia | Wiskott-Aldrich Syndrome (WAS)

United States
Ramathibodi Hospital
AstraZeneca

Completed

Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy

Spinobulbar Muscular Atrophy | Kennedy's Disease

Thailand
National Institute of Neurological Disorders and...

Completed

Dutasteride to Treat Spinal and Bulbar Muscular Atrophy (SBMA)

Spinal and Bulbar Muscular Atrophy | Kennedy's Disease

United States
University of Mississippi Medical Center

Completed

Advanced Grandparental Age as a Risk Factor for Autism

Autistic Disorder | Rett Syndrome | Asperger Syndrome | Pervasive Developmental Disorder | Childhood Disintegrative Disorder

United States
Janssen Pharmaceutical K.K.

Recruiting

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

Japan
Frontera Therapeutics
Peking Union Medical College Hospital; Eye & ENT Hospital of Fudan University; Henan Provincial People's Hospital

Recruiting

Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

China
Janssen Pharmaceutical K.K.

Recruiting

An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

Japan
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
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
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
Rocket Pharmaceuticals Inc.

Recruiting

A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Danon Disease

United States
University of Manchester
Manchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaborators

Recruiting

Autologous Ex-vivo Gene Modified HSCT in MPSII

Mucopolysaccharidosis II

United Kingdom
GC Biopharma Corp

Recruiting

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ

Mucopolysaccharidosis II | Hunter Syndrome

Korea, Republic of
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia
Homology Medicines, Inc

Withdrawn

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Mucopolysaccharidosis II

United States, Canada
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
AO GENERIUM

Completed

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Metabolic Diseases | Mucopolysaccharidosis Type II

Russian Federation
Hacettepe University

Completed

Chewing and Swallowing Training Program in Coffin-Lowry Syndrome

Chewing Problem | Swallowing Disorder

Turkey
GlaxoSmithKline

Completed

A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

Mucopolysaccharidosis II

Japan
Green Cross Corporation

Unknown

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Mucopolysaccharidosis II
National Center for Research Resources (NCRR)
University of California, San Diego

Completed

Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

Lesch-Nyhan Syndrome
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
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
Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Shire

Active, not recruiting

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
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
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

Clinical Trials on X-Linked Hypophosphatemia

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