Clinical Trials on Hereditary Ataxia

Total 3051 results

Massachusetts General Hospital
United States Department of Defense; Recordati Rare Diseases

Recruiting

Screening Trial for Pain Relief in Schwannomatosis (STARFISH)

Pain, Chronic | Schwannomatosis | Schwannomas

United States
Vanderbilt-Ingram Cancer Center
National Cancer Institute (NCI); University of South Florida

Recruiting

Improving Care After Inherited Cancer Testing (IMPACT)

Breast Cancer | Colorectal Cancer | Prostate Cancer | Endometrial Cancer | Inherited Cancer Syndrome

United States
National Human Genome Research Institute (NHGRI)
Merck Sharp & Dohme LLC; Duke University; University of Massachusetts, Worcester

Recruiting

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

Fever | Genetic Diseases | Familial Mediterranean Fever (FMF) | Autoinflammation | Periodic Fever | ROSAH | ALPK1

United States
National Cancer Institute (NCI)

Active, not recruiting

MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

Neurofibromatosis 1 (NF1) | Plexiform Neurofibromas (PN)

United States
AstraZeneca
Merck Sharp & Dohme LLC

Active, not recruiting

A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

Neurofibromatosis 1 | Neurofibroma Plexiform

China
Pharnext S.C.A.
Premier Research Group plc; Eurofins Optimed; Synteract HCR (Syneos Health); Gr... and other collaborators

Active, not recruiting

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PLEO-CMT-FU)

Charcot-Marie-Tooth Disease, Type IA

United States, Belgium, Canada, France, Netherlands, Spain, United Kingdom
University of Alabama at Birmingham
Pacific Pediatric Neuro-Oncology Consortium; Array BioPharma

Active, not recruiting

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas (NF108-BINI)

Neurofibromatosis Type 1 | Plexiform Neurofibroma

United States
Sol-Gel Technologies, Ltd.

Terminated

Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

Basal Cell Nevus Syndrome

United States, France, Belgium, Denmark, Germany, Italy, Netherlands, Spain, United Kingdom
ProQR Therapeutics

Terminated

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (HELIA)

Retinitis Pigmentosa | Usher Syndrome Type 2

United States, Canada, France
Sanofi

Active, not recruiting

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Usher's Syndrome

United States, France
Yuzuncu Yıl University

Completed

The Effect of Ulnar Nerve Injury Localization on Sleep Quality in Patients With Ulnar Nerve Entrapment Neuropathy

Ulnar Neuropathies

Turkey
Novartis Pharmaceuticals

Completed

Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations

Cancer

France, Australia, United States, Canada, United Kingdom
NYU Langone Health

Completed

A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)

Neurofibromatosis 1 and 2 (NF1 and NF2)

United States
UCSF Benioff Children's Hospital Oakland
National Cancer Institute (NCI)

Completed

Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Face

Neoplastic Syndrome

United States
Edward Maytin, MD, PhD
DUSA Pharmaceuticals, Inc.

Completed

Blue vs Red Light During Levulan Based Photodynamic Therapy in Patients With Basal Cell Nevus Syndrome

Basal Cell Nevus Syndrome

United States
Pharnext SA

Completed

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PLEO-CMT)

Charcot-Marie-Tooth Disease Type 1A

United States, United Kingdom, Belgium, Canada, France, Netherlands, Spain, Germany
Nobelpharma

Completed

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Plaque | Tuberous Sclerosis | Angiofibroma | Hypomelanotic Macule

Japan
Christian Kern

Unknown

Severe and Transient Hypoxemia During Selective Intra-arterial Chemotherapy for Retinoblastoma in Children: Evaluation of the Right-sided Heart Function.

Retinoblastoma

Switzerland
Burzynski Research Institute

Terminated

Antineoplaston Therapy in Treating Patients With Neurofibroma and Schwannoma

CNS Tumor

United States
Assistance Publique - Hôpitaux de Paris
Novartis

Completed

Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas (NFitor)

Neurofibromatoses | Neurofibromatosis Type 1 | Plexiform Neurofibroma

France
National Institute of Arthritis and Musculoskeletal...

Completed

Interleukin-1 Trap to Treat Autoinflammatory Diseases

Inflammation | Familial Mediterranean Fever | Still's Disease, Adult-Onset

United States
Guy Hains Chiropractor

Completed

The Treatment of the Median Nerve for the Elimination of the Symptoms Associated With the Carpal Tunnel Syndrome

Compression Neuropathy, Carpal Tunnel

Canada
National Cancer Institute (NCI)

Completed

A Phase II Trial of All-Trans-Retinoic Acid in Combination With Interferon-Alpha 2a in Children With Recurrent Neuroblastoma or Wilms' Tumor

Neuroblastoma | Nephroblastoma

United States
Foundation Fighting Blindness

Recruiting

Inherited Retinal Degenerative Disease Registry (MRTR)

Retinitis Pigmentosa | Choroideremia | Usher Syndrome | Batten Disease | Leber Congenital Amaurosis | Goldmann-Favre Syndrome | Kearns-Sayre Syndrome | Retinal Disease | Bardet-Biedl Syndrome | Stargardt Disease | Cone Dystrophy | Retinoschisis | Achromatopsia | Gyrate Atrophy | Eye Diseases Hereditary | Bassen-Kornzweig... and other conditions

United States
Istanbul University - Cerrahpasa (IUC)

Not yet recruiting

Comparing Efficacies of Median Nerve Hydrodissection With Dexamethasone and Dextrose in Carpal Tunnel Syndrome

Carpal Tunnel Syndrome | Entrapment Neuropathy | Median Nerve Entrapment

Turkey
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
St. Jude Children's Research Hospital

Recruiting

Determination of the Sensitivity and Specificity of a Smartphone Application to Detect Retinoblastoma

Glaucoma, Congenital | Retinoblastoma | Cataracts Infantile | Leucocoria

United States
Assistance Publique Hopitaux De Marseille

Recruiting

Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers (CMT-TOOLS)

Charcot-Marie-Tooth Disease Type 1A

France
AstraZeneca

Completed

C-PATROL - Non-interventional Study (NIS) to Collect Clinical and Patient Reported Outcome Data in an Olaparib Treated BRCAm+ PSR Ovarian Cancer Population (C-PATROL)

Ovarian Cancer, Hereditary Ovarian Cancer Syndrome

Germany
Oslo University Hospital

Enrolling by invitation

Nevoid Basal Cell Carcinomas in Gorlin Syndrome

Basal Cell Carcinoma | Gorlin Syndrome

Norway
University of Aberdeen
NHS Grampian

Completed

Abdominal Cutaneous Nerve Entrapment Syndrome (ACNES)

Abdominal Pain | Abdominal Cutaneous Nerve Entrapment Syndrome

United Kingdom
Afyonkarahisar Health Sciences University

Completed

High Intensity Laser Therapy in Carpal Tunnel Syndrome

Musculoskeletal Diseases | Carpal Tunnel Syndrome | Median Neuropathy | Entrapment Neuropathy

Turkey
Sanofi

Terminated

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

Retinitis Pigmentosa | Usher Syndrome

United States, France
Promethera Therapeutics

Completed

Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) (HEP001)

Urea Cycle Disorders | Crigler Najjar Syndrome

Israel, United Kingdom, Belgium, France, Italy
PellePharm, Inc.

Completed

Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients

Basal Cell Nevus Syndrome

United Kingdom
Columbia University

Withdrawn

Nivolumab With Vismodegib in Patients With Basal Cell Nevus Syndrome

Basal Cell Nevus Syndrome
Washington University School of Medicine
St. Louis Children's Hospital

Terminated

Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma

Neurofibromatosis Type 1

United States
Bursa Yüksek İhtisas Education and Research Hospital

Completed

Evaluation of Fibromyalgia With Disease Activity and Clinical Findings in Women With Behçet's Disease

Fibromyalgia | Behcet's Disease

Turkey
NYU Langone Health

Completed

A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

Anxiety Disorders | Familial Dysautonomia | Dysthymia | Paroxysmal Hypertension | Autosomal Recessive Disease

United States
University of Alabama at Birmingham

Completed

MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1 (MEK Inhibitor)

Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN

United States
Children's Hospital of Philadelphia

Completed

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Neurofibromatosis Type 1 | Precancerous Condition

United States
Zymenex A/S

Completed

The Natural History of Infantile Globoid Cell Leukodystrophy

Infantile Globoid Cell Leukodystrophy

United States
University of Michigan Rogel Cancer Center

Completed

A Pilot Study of Tumor Cell Vaccine for High-risk Solid Tumor Patients Following Stem Cell Transplantation

Sarcoma | Neuroblastoma | Wilm's Tumor

United States
National Cancer Institute (NCI)

Terminated

AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine

Neurofibromatosis Type 1 | Plexiform Neurofibroma | Spinal Cord Neurofibroma

United States
University Health Network, Toronto

Completed

Study of BMS-833923 in Two Specific Patients With Basal Cell Nevus Syndrome

Basal Cell Nevus Syndrome

Canada
Massachusetts General Hospital

Completed

Relationship Between Psychosocial Factors, Health Literacy, Quality of Life and Satisfaction With Medical Visits in Adults With NF

Neurofibromatosis

United States
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Gene Analysis in Studying Susceptibility to Wilms Tumor

Recurrent Childhood Kidney Neoplasm | Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor

United States
NYU Langone Health

Withdrawn

Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Brain Tumor

Sarcoma | Brain and Central Nervous System Tumors | Neuroblastoma | Retinoblastoma

United States, Canada, Argentina
Children's Hospital of Philadelphia

Terminated

Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma

Neurofibromatosis Type 1 | Precancerous Condition

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Celecoxib in Preventing Basal Cell Carcinoma in Patients With Basal Cell Nevus Syndrome

Non-melanomatous Skin Cancer

United States

Clinical Trials on Hereditary Ataxia

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Rare Diseases

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