Clinical trials: Cerebellar Disorders
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
Adult Anaplastic Ependymoma, Adult Anaplastic Oligodendroglioma
18F-FDOPA PET/CT or PET/MRI in Measuring Tumors in Patients With Newly-Diagnosed or Recurrent Gliomas
To evaluate 18F-FDOPA PET obtained from PET/CT or PET/MRI imaging in patients with newly diagnosed or recurrent gliomas.
- Conditions
- Adult Anaplastic Ependymoma, Adult Anaplastic Oligodendroglioma, Adult Brain Stem Glioma, Adult Diffuse Astrocytoma
- Trial name
- NCT02175745
- Testing
- 18F-fluoro-dihydroxyphenylalanine, Positron emission tomography (PET), Computed tomography (CT)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Erik Mittra
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside
Study design: Single arm open-label clinical trial in ataxia-telangiectasia to test the effects of nicotinamide riboside on ataxia scales, immune function, and neurofilament light chain.
- Trial name
- ATNAD (NCT06324877)
- Testing
- Nicotinamide riboside
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- The University of Queensland
Cerebellar Neoplasm
Impact of Cerebellar Mass Resection on Pain Processing
The purpose of the study is to evaluate how cerebellar resection surgery may affect pain sensation and the way the brain "reads" pain signals.
- Trial name
- NCT02261649
- Testing
- Medoc Advanced Medical Systems PATHWAY Model ATS, Cold water bath, MRI scanner
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Dana-Farber Cancer Institute
Friedreich Ataxia
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological…
- Trial name
- UNIFAI (NCT06016946)
- Where
- 34 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor
Temozolomide and O6-Benzylguanine in Treating Children With Recurrent Brain Tumors
Phase I trial to study the safety of combining O6-benzylguanine with temozolomide in treating children who have recurrent or refractory brain tumors.
- Conditions
- Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Ependymoblastoma
- Trial name
- NCT00052780
- Testing
- O6-benzylguanine, temozolomide, filgrastim
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- National Cancer Institute (NCI)
Friedreich Ataxia
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich's Ataxia (FA) Friedreich's Ataxia is a neurodegenerative disease caused by a homozygous expansion of the GAA triplet repeats of the frataxin gene (FXN). FA usually begins in childhood or adolescence.
- Trial name
- CPCAF (NCT05874388)
- Where
- France
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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
Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor
AZD2171 in Treating Young Patients With Recurrent, Progressive, or Refractory Primary CNS Tumors
This phase I trial is studying the side effects and best dose of AZD2171 in treating young patients with recurrent, progressive, or refractory primary CNS tumors.
- Conditions
- Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor, Childhood Cerebral Anaplastic Astrocytoma, Childhood Cerebral Astrocytoma
- Trial name
- NCT00326664
- Testing
- Cediranib Maleate
- Where
- 11 sites in United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Solid Tumor, Adult Central Nervous System Germ Cell Tumor
Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor
RATIONALE: Giving high-dose chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them.
- Conditions
- Solid Tumor, Adult Central Nervous System Germ Cell Tumor, Adult Rhabdomyosarcoma, Childhood Central Nervous System Germ Cell Tumor
- Trial name
- NCT00638898
- Testing
- busulfan, melphalan, topotecan hydrochloride
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- City of Hope Medical Center
Ataxia Telangiectasia in Children, Ataxia Telangiectasia
Home-based Complex Intervention for Children With Ataxia Telangiectasia
Ataxia telangiectasia is a rare, genetic and progressive condition with no known cure. Therapies present a mainstream management option and have the potential to offer optimisation of fitness and general health.
- Trial name
- NCT05692622
- Testing
- Whole-body exercise and respiratory exercise
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- University of Plymouth
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
ABT-751 in Treating Young Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy, such as ABT-751, work in different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00036959
- Testing
- ABT-751
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Brain and Central Nervous System Tumors, Kidney Cancer
Early Enteral Tube Feedings in Children Receiving Chemo for AML/MDS & High Risk Solid Tumors
RATIONALE: Tubefeeding may help maintain good nutrition and lessen weight loss in younger patients receiving chemotherapy for cancer.
- Conditions
- Brain and Central Nervous System Tumors, Kidney Cancer, Leukemia, Liver Cancer
- Trial name
- NCT00624962
- Testing
- nutritional intervention, therapeutic nutritional supplementation
- Sponsor
- Vanderbilt University
Friedreich Ataxia
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients
Friedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease.
- Trial name
- CalcitriolFA (NCT04801303)
- Testing
- Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Berta Alemany
Autism Spectrum Disorder
Cerebellar tDCS in Children With Autism Spectrum Disorder
The purpose of this research study is to investigate whether tDCS to the cerebellum (specifically, the right crus I/II area of the cerebellum) of children and young adults with autism spectrum disorders (ASD) is safe and to…
- Trial name
- NCT04446442
- Testing
- tDCS
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Childhood Burkitt Lymphoma, Childhood Central Nervous System Germ Cell Tumor
Oxaliplatin and Irinotecan in Treating Young Patients With Refractory Solid Tumors or Lymphomas
This phase I trial is studying the side effects and best dose of oxaliplatin when given together with irinotecan in treating young patients with refractory solid tumors or lymphomas.
- Conditions
- Childhood Burkitt Lymphoma, Childhood Central Nervous System Germ Cell Tumor, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis
- Trial name
- NCT00101270
- Testing
- irinotecan hydrochloride, oxaliplatin
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT06673056
- Testing
- N-Acetyl-L-Leucine
- Where
- 11 sites in United States, Germany and 4 more
- Volunteers
- 60 people
- Sponsor
- IntraBio Inc
Dystonia
Interactions Between Striatum and Cerebellum in ADCY5 and PRRT2 Dystonias
The investigators will study the relationship between the basal ganglia and the cerebellum in dystonia by associating cerebellar stimulations with functional magnetic resonance imaging analysis.
- Trial name
- AMEDYST (NCT03481491)
- Testing
- transcranial magnetic stimulation of the cerebellum
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France