Clinical Trials on X-linked Adrenoleukodystrophy in South Africa

Total 127 results

Catalyst Biosciences

Completed

Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

Hemophilia B

South Africa
bluebird bio

Completed

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD)

United States, Germany, Argentina, Australia, France, United Kingdom
Murdoch Childrens Research Institute
University of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaborators

Active, not recruiting

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

Autosomal Recessive Disorder | X-Linked Genetic Diseases

Australia
Bayer

Completed

Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors (MATCHBOX)

Hemophilia A | Hemophilia B

South Africa, Poland, United Kingdom
Anavex Life Sciences Corp.

Completed

ANAVEX2-73 Study in Patients With Rett Syndrome (AVATAR)

Rett Syndrome

Australia, United Kingdom
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
Novo Nordisk A/S

Completed

Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries (HAEMOcare)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B

India, Morocco, South Africa, Oman
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
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
Anavex Life Sciences Corp.
International Rett Syndrome Foundation Rettsyndrome.org

Completed

Study of ANAVEX2-73 in Patients With Rett Syndrome

Rett 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
Catalyst Biosciences

Completed

Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

Armenia, Georgia, South Africa, Poland, Russian Federation
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
Anavex Life Sciences Corp.
Anavex Australia Pty Ltd.; Anavex Germany GmbH

Completed

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome (EXCELLENCE)

Rett Syndrome

Canada, Australia, United Kingdom
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
Tetra Discovery Partners

Recruiting

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

Fragile X Syndrome

United States
Tetra Discovery Partners

Enrolling by invitation

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

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
Jazz Pharmaceuticals
GW Pharmaceuticals

Terminated

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Rett Syndrome | RTT

United States, Canada, Italy, Spain, United Kingdom, Australia
Neuren Pharmaceuticals Limited

Completed

A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Fragile X Syndrome

United States
Neuren Pharmaceuticals Limited
rettsyndrome.org

Completed

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Rett Syndrome

United States
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
Laboratoire français de Fractionnement et de Biotechnologies
LFB USA, Inc.

Completed

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)

Hemophilia A With Inhibitors | Hemophilia B With Inhibitors

Bulgaria, Ukraine, Czechia, United States, Georgia, South Africa
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
University of Alabama at Birmingham
University of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaborators

Completed

Genetic and Physical Characteristics of Rett Syndrome

Rett Syndrome | MECP2 Duplication Disorder | Rett-related Disorder

United States
ACADIA Pharmaceuticals Inc.

Completed

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LILAC™)

Rett Syndrome

United States
ACADIA Pharmaceuticals Inc.

Completed

Open-Label Extension Study of Trofinetide for Rett Syndrome

Rett Syndrome

United States
University of Alabama at Birmingham
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Biobanking of Rett Syndrome and Related Disorders

CDKL5 | Rett Syndrome | MECP2 Duplication | FOXG1 Disorders

United States
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
ACADIA Pharmaceuticals Inc.

Completed

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

Rett Syndrome

United States
Novartis Pharmaceuticals

Completed

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

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
University of Alabama at Birmingham
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Natural History of Rett Syndrome & Related Disorders

Rett Syndrome | CDKL5 Disorder | MECP2 Duplication dIsorder | FOXG1 Syndrome

United States
Jazz Pharmaceuticals

Terminated

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome (ARCH)

Rett Syndrome | RTT

United States, Spain, Italy, United Kingdom
CSL Behring

Terminated

Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors

Hemophilia A With Inhibitors | Hemophilia B With Inhibitors

Georgia, Italy, Malaysia, Russian Federation, South Africa, Spain, Thailand, Ukraine, United Kingdom
Kiora Pharmaceuticals, Inc.

Recruiting

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS) (ABACUS)

Retinitis Pigmentosa | Choroideremia

Australia
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

Hemophilia B

United States, United Kingdom, South Africa, Netherlands, Ireland, Australia
Bionano Genomics
Columbia University; University of Iowa; Medical College of Wisconsin; Augusta... and other collaborators

Recruiting

Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort

Intellectual Disability | Autism Spectrum Disorder | Fragile X Syndrome | Developmental Disability | Congenital Anomaly | Facioscapulohumeral Muscular Dystrophy 1

United States
Denali Therapeutics Inc.

Completed

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Mucopolysaccharidosis II

United Kingdom, United States, Italy, Netherlands
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
Sheffield Children's NHS Foundation Trust
Sheffield Hallam University; National Institute for Health Research, United... and other collaborators

Active, not recruiting

DMD- Interactive Virtual Reality Study (DMD-IVR)

Duchenne Muscular Dystrophy

United Kingdom
International Rett Syndrome Foundation
Baylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaborators

Recruiting

Rett Syndrome Registry (RSR)

Nervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, Atypical

United States
Freeline Therapeutics

Terminated

A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Hemophilia B

United Kingdom
Baylor College of Medicine
University of California, San Diego; University of Alabama at Birmingham; Boston... and other collaborators

Completed

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW (RDCRN)

Prader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep Problems

United States
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Spark Therapeutics
Children's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaborators

Terminated

Hemophilia B Gene Therapy With AAV8 Vector

Hemophilia B

United States, Australia
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Hemophilia A | Hemophilia B

United States, Italy, Spain, Taiwan, Australia, Belgium, United Kingdom, France, Germany, India, South Africa, Poland, Canada, Armenia, Turkey
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

Hemophilia A | Hemophilia B

Spain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Egypt, Poland, Armenia, France, India, Canada, Brazil, Turkey
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
Pfizer

Completed

A Gene Therapy Study for Hemophilia B

Hemophilia B

United States, Australia
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

Severe Hemophilia B

Sweden, United States, France, Italy, Russian Federation, United Kingdom, Germany, China, Poland, Japan, Australia, Brazil, Canada, India, South Africa, Hong Kong, Belgium

Clinical Trials on X-linked Adrenoleukodystrophy in South Africa

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