Clinical trials: Cerebellar Diseases
Childhood Burkitt Lymphoma, Childhood Central Nervous System Choriocarcinoma
Vorinostat and Bortezomib in Treating Young Patients With Refractory or Recurrent Solid Tumors, Including Central Nervous System Tumors and Lymphoma
This phase I trial is studying the side effects and best dose of vorinostat when given together with bortezomib in treating young patients with refractory or recurrent solid tumors, including CNS tumors and lymphoma.
- Conditions
- Childhood Burkitt Lymphoma, Childhood Central Nervous System Choriocarcinoma, Childhood Central Nervous System Germ Cell Tumor, Childhood Central Nervous System Germinoma
- Trial name
- NCT00994500
- Testing
- vorinostat, bortezomib, pharmacological study
- Where
- 6 sites in United States
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Brain and Central Nervous System Tumors
Lobradimil and Carboplatin in Treating Children With Brain Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Lobradimil may increase the effectiveness of a chemotherapy drug by making tumor cells more sensitive to the…
- Trial name
- NCT00019422
- Testing
- carboplatin, lobradimil
- Where
- 236 sites in United States, Australia and 5 more
- Sponsor
- National Cancer Institute (NCI)
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Temozolomide and O6-benzylguanine in Treating Children With Solid 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.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00020150
- Testing
- O6-benzylguanine, temozolomide
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Friedreich Ataxia
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy.
- Trial name
- NCT01965327
- Testing
- Interferon Gamma-1b
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Micronised Resveratrol as a Treatment for Friedreich Ataxia
The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial.
- Trial name
- NCT03933163
- Testing
- Resveratrol
- Where
- 4 sites in Australia
- Volunteers
- 25 people
- Sponsor
- Murdoch Childrens Research Institute
FXN Gene, FRDA
Biomarker for Friedreich's Ataxia (BioFridA)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Friedreich's Ataxia and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Conditions
- FXN Gene, FRDA, Hereditary Diseases
- Trial name
- BioFridA (NCT04548921)
- Where
- Lebanon
- Volunteers
- 1,000 people
- Sponsor
- CENTOGENE GmbH Rostock
Friedreich Ataxia
Open Trail of γIFN for Friedreich Ataxia
The investigator proposes an open label pilot study to investigate the safety and efficacy of gamma interferon (γIFN) in patients with Friedreich's Ataxia (FRDA).
- Trial name
- NCT03888664
- Testing
- gamma interferon
- Volunteers
- 12 people
- Sponsor
- IRCCS Eugenio Medea
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children
MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Ataxia Telangiectasia (A-T) is an autosomal recessively inherited neurodegenerative disorder that also has dramatic effects on the immune and endocrine systems.
- Conditions
- Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
- Trial name
- NCT04887311
- Testing
- MBM-01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Matrix Biomed, Inc.
Autism Spectrum Disorder
Cerebellar Transcranial Direct Current Stimulation (tDCS) in Children With Autism Spectrum Disorder: Raynor Cerebellum Project
The purpose of this research study is to investigate the effects of transcranial direct current stimulation (tDCS) on some of the challenges faced by children with Autism Spectrum Disorder (ASD).
- Trial name
- NCT05691283
- Testing
- trans cranial direct current stimulation (tDCS)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center
Brain and Central Nervous System Tumors, Neuroblastoma
Temozolomide Followed by Radiation Therapy in Treating Children With Newly Diagnosed Malignant CNS Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells.
- Trial name
- NCT00005955
- Testing
- temozolomide
- Where
- United States
- Sponsor
- Duke University
Friedreich Ataxia
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA).
- Trial name
- PROFA (NCT05943002)
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 200 people
- Sponsor
- German Center for Neurodegenerative Diseases (DZNE)
Friedreich's Ataxia
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).
- Trial name
- STEADFAST (NCT02797080)
- Testing
- interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 38 people
- Sponsor
- Amgen
Spinocerebellar Degenerations
High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration
This study will examine whether high-dose intravenous immunoglobulin (IVIG) is safe and effective for treating cerebellar ataxia-degeneration of the cerebellum, the part of the brain responsible for coordinating muscle movements…
- Trial name
- NCT00034242
- Testing
- high-dose intravenous immunoglobulin (IVIG)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Brain and Central Nervous System Tumors
Combination Chemotherapy With or Without Etoposide Followed By an Autologous Stem Cell Transplant in Treating Young Patients With Previously Untreated Malignant Brain Tumors
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00392886
- Testing
- carboplatin, cisplatin, cyclophosphamide
- Where
- 37 sites in United States, Australia and 3 more
- Volunteers
- 120 people
- Sponsor
- Children's Hospital Los Angeles
Developmental Dyslexia, Developmental Coordination Disorder
Functional MRI Study in Children With a Developmental Coordination Disorder (DCD) and/or Developmental Dyslexia (DD)
All the studies underlined the high frequency of co-morbid associations in specific learning disorders. Understanding the reasons for these associations could enable us to determine the cerebral bases that underlie each disorder.
- Trial name
- FMRI-DCD-DD (NCT02393729)
- Testing
- Neuropsychological assessment, MRI Study
- Where
- France
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Spinocerebellar Ataxia Type 3
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
DESIGN: Pilot, Phase II, double-blind, placebo-controlled study JUSTIFICATION: In the literature one does not find a pharmacological treatment that changes the natural history of Spinocerebellar ataxtia type 3 (SCA3).
- Trial name
- NCT01096095
- Testing
- Sodium Phenylbutyrate
- Where
- Brazil
- Sponsor
- Hospital de Clinicas de Porto Alegre
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Study design: Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.
- Trial name
- A-TC7 (NCT04513002)
- Testing
- Triheptanoin
- Where
- Australia
- Volunteers
- 30 people
- Sponsor
- The University of Queensland
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