UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oli… (NCT02254863) | Clinical Trial Compass
RecruitingPhase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
United States40 participantsStarted 2014-09
Plain-language summary
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
Who can participate
Age range1 Week – 22 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Patients must be age ≥1 week to ≤21 years.
✓. Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample:
✓. Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:
✓. Patients must have adequate organ function as measured by:
✓. Patients must have an available, suitably matched, banked UCB unit for transplant.
✓. Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%
✓. Patients must have a life expectancy of ≥ 6 months.
Exclusion criteria
✕. Prior organ, tissue, or stem cell transplant within 3 years of study entry.