Clinical trials: Cree Leukoencephalopathy
Cree Leukoencephalopathy
Single Patient Investigational Treatment for Cree Leukoencephalopathy
Cree Leukoencephalopathy (CLE) is a very rare and severe brain disease that mainly affects members of the Cree communities in Northern Quebec.
- Trial name
- NCT07300397
- Testing
- Fosigotifator
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Vanishing White Matter Disease
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.
- Trial name
- NCT05757141
- Testing
- Fosigotifator
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Calico Life Sciences LLC
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Vanishing White Matter, Cree Leukoencephalopathy
Expanded Access to Fosigotifator
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to Fosigotifator prior to approval by the local regulatory agency. Availability will depend on territory eligibility.
- Trial name
- NCT06594016
- Testing
- Fosigotifator
- Sponsor
- AbbVie
Cree Leukoencephalopathy, Vanishing White Matter
Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease
Cree Leukoencephalopathy (CLE) is a rare and fatal neurodegenerative disorder predominantly affecting the Cree population in Northern Quebec.
- Trial name
- NCT07272525
- Testing
- Fosigotifator (FGT/ABBV-CLS-7262)
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Leukodystrophy, White Matter Disease
The Myelin Disorders Biorepository Project
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects.
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome
- Trial name
- MDBP (NCT03047369)
- Where
- 23 sites in United States
- Volunteers
- 12,000 people
- Sponsor
- Children's Hospital of Philadelphia