Clinical trials: Pelizaeus-Merzbacher Disease
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to…
- Trial name
- NCT05659901
- Where
- 10 sites in United States, France and 5 more
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Pelizaeus-Merzbacher Disease
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01005004
- Testing
- HuCNS-SC cells
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.