A PHASE III TRIAL OF ALD-101 ADUVANT THERAPY of UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN PATIENTS WITH INBORN ERRORS OF METABOLISM

Aldagen, Inc0 sites40 target enrollmentOctober 29, 2009

Overview

No summary available.

Registry

who.int

Start Date

October 29, 2009

End Date

TBD

Last Updated

14 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

Aldagen, Inc

•1\. Patients must have a confirmed diagnosis of inborn error of metabolism. Diagnosis to be confirmed by two appropriate tests (enzyme or mutation analysis) before study entry.
•Inborn errors of metabolism include the following:
•Hurler Syndrome (MPS I)
•Hurler\-Scheie Syndrome
•Hunter Syndrome (MPS II)
•Sanfilippo Syndrome (MPS III)
•Maroteau\-Lamy Syndrome (MPS VI)
•Krabbe Disease (Globoid Leukodystrophy)
•Metachromatic Leukodystrophy (MLD)
•Adrenoleukodystrophy ALD and AMN)

•1\. Patients that are HIV, Hepatitis B and/or Hepatitis C positive.
•2\. Patients that are concurrently involved in any other clinical study that affects engraftment or immune reconstitution (e.g., other hematopoietic growth factors).
•3\. Patients with uncontrolled seizures, apnea, evidence of aspiration pneumonia, or evidence of brain stem involvement on MRI scans.
•4\. Patients with uncontrolled infections.
•5\. Patients with prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of enrollment.
•6\. Patients that have any comorbid condition which, in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.