Clinical trials: Cerebellar Disorders
Brain and Central Nervous System Tumors, Metastatic Cancer
Radiolabeled Monoclonal Antibody Therapy in Treating Patients With Primary or Metastatic Brain Cancers
RATIONALE: Radiolabeled monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Trial name
- NCT00002752
- Testing
- iodine I 131 monoclonal antibody 81C6
- Where
- United States
- Sponsor
- Duke University
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Spinocerebellar Ataxia Type 3, Machado Joseph Disease
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Design: Phase II-III, double-blind, parallel, placebo controlled randomized Clinical trial Background: Spinocerebellar ataxia type 3 (SCA-3) is an autosomal dominant adult-onset neurodegenerative disorder for which there is no…
- Trial name
- NCT01096082
- Testing
- Lithium Carbonate
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Brain and Central Nervous System Tumors, Lymphoma
Chemotherapy and Stem Cell Transplantation in Treating Children With Central Nervous System Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of…
- Conditions
- Brain and Central Nervous System Tumors, Lymphoma, Neuroblastoma, Retinoblastoma
- Trial name
- NCT00053118
- Testing
- filgrastim, carboplatin, etoposide
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Roswell Park Cancer Institute
Brain and Central Nervous System Tumors, Chordoma
Polyvinylpyrrolidone-Sodium Hyaluronate Gel in Reducing Pain From Oral Mucositis in Young Patients With Cancer
RATIONALE: Polyvinylpyrrolidone-sodium hyaluronate gel may lessen the pain of oral mucositis, or mouth sores, in patients undergoing treatment for cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chordoma, Kidney Cancer, Leukemia
- Trial name
- NCT00349024
- Testing
- polyvinylpyrrolidone-sodium hyaluronate gel
- Where
- 21 sites in Ireland, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Children's Cancer and Leukaemia Group
Brain Tumors, Central Nervous System Tumors
Combination Chemotherapy in Treating Children With Progressive Brain Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Trial name
- NCT00002944
- Testing
- carboplatin, lomustine, procarbazine hydrochloride
- Where
- 204 sites in United States, Australia and 4 more
- Volunteers
- 428 people
- Sponsor
- Children's Oncology Group
Brain and Central Nervous System Tumors, Unspecified Childhood Solid Tumor, Protocol Specific
Temozolomide, Vincristine, and Irinotecan in Treating Young Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy, such as temozolomide, vincristine, and irinotecan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00138216
- Testing
- irinotecan hydrochloride, temozolomide, vincristine sulfate
- Where
- 18 sites in United States, Canada
- Volunteers
- 42 people
- Sponsor
- Children's Oncology Group
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
Brain and Central Nervous System Tumors
Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of phenylacetate in treating children with recurrent or…
- Trial name
- NCT00003241
- Testing
- phenylacetate
- Where
- 6 sites in United States
- Sponsor
- Texas Children's Cancer Center
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Choriocarcinoma
Vorinostat and Temozolomide in Treating Young Patients With Relapsed or Refractory Primary Brain Tumors or Spinal Cord Tumors
This phase I trial is studying the side effects and best dose of vorinostat when given together with temozolomide in treating young patients with relapsed or refractory primary brain tumors or spinal cord tumors.
- Conditions
- Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Choriocarcinoma, Childhood Central Nervous System Embryonal Tumor, Childhood Central Nervous System Germinoma
- Trial name
- NCT01076530
- Testing
- vorinostat, temozolomide, diagnostic laboratory biomarker analysis
- Where
- 9 sites in United States, Canada
- Volunteers
- 27 people
- Sponsor
- National Cancer Institute (NCI)
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Friedreich Ataxia
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Ataxia Telangiectasia (AT)
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Ataxia Telangiectasia (AT) is an autosomal recessive inherited condition caused by mutations in the ATM gene1. Patients suffer from neuro-degenerative problems, usually commencing in the second year of life, and affecting…
- Trial name
- NCT00656409
- Testing
- Conjugated pneumococcal vaccine (Prevenar)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institute of Child Health
Childhood High-grade Cerebral Astrocytoma, Childhood Oligodendroglioma
Tipifarnib in Treating Young Patients With Recurrent or Progressive High-Grade Glioma, Medulloblastoma, Primitive Neuroectodermal Tumor, or Brain Stem Glioma
This phase II trial is studying how well tipifarnib works in treating young patients with recurrent or progressive high-grade glioma, medulloblastoma, primitive neuroectodermal tumor, or brain stem glioma.
- Conditions
- Childhood High-grade Cerebral Astrocytoma, Childhood Oligodendroglioma, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Cerebellar Astrocytoma
- Trial name
- NCT00070525
- Testing
- tipifarnib
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Cancer Institute (NCI)
Ataxia Telangiectasia, Growth Failure
Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)
This study will evaluate the status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and composition and neuroprotection in children with Ataxia telangiectasia (AT).
- Trial name
- GHAT (NCT01052623)
- Testing
- Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
Friedreich Ataxia, Cardiomyopathy
Clinical Course Of Disease In Participants With FA-CM
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics