Krabbe Clinical Trials

Identifier: NCT03406962

Description of Trial:  This is a Phase 2 Study designed to evaluate the safety and efficacy of a pharmacological agent to produce rapid and sustained engraftment for patients undergoing a Hematopoietic Stem Cell Transplant (HSCT) with an Inherited Metabolic Disorder (IMD).

Patient Inclusion Criteria:

1. Diagnosed with one of the following:
   1. Globoid Cell Leukodystrophy or Krabbe disease
   2. Hurler Syndrome
   3. Metachromatic Leukodystrophy (MLD)
   4. Cerebral Adrenoleukodystrophy (ALD)
2. Must have adequate organ function
3. Must have availability of eligible donor material

Patient Exclusion Criteria:

1. Availability of a matched-related donor who is not a carrier of the same genetic defect
2. Active infection at screening
3. Prior myeloablative conditioning
4. History of human immunodeficiency virus (HIV) infection

For More Information:
Contact:  Magenta Clinical Trials
Principal Investigator: Dr. Paul Orchard

Please Note:

1. Choosing to participate in a clinical trial is a personal decision and one that should be discussed thoroughly with your specific care team.
2. For more information on this specific clinical trial, please contact Magenta or Dr. Paul Orchard.  KrabbeConnect does not have any additional insight or information.
3. KrabbeConnect Clinical Trials webpage is strictly a resource to inform patients and caregivers of the current opportunities available.  The information should never be interpreted as medical advice.