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

Phase 3

Completed

Conditions: genetic metabolic diseases
inborn errors of metabolism
10027424

Registration Number: NL-OMON32439

Lead Sponsor: Aldagen, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 12

Inclusion Criteria

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.
Patients must be < 16 years of age at the time of study enrollment.
Patients must be >= 5 kg in weight.
Patients must have a good performance status (Lansky >= 80%).
Patients must have adequate function of other organ systems.
Patients must have a minimum life expectancy of at least 6 months.
Patient must be determined to be a good candidate for a standard UCBT according to the investigator and standard eligibility work-up at the site.

Exclusion Criteria

Patients that are HIV, Hepatitis B and/or Hepatitis C positive.
Patients that are concurrently involved in any other clinical study that affects engraftment or immune reconstitution (e.g., other hematopoietic growth factors).
Patients with uncontrolled infections.
Patients with prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of enrollment.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Platelet Engraftment (50k): Platelet engraftment will be defined as the first<br /><br>day that the patients has achieved a platelet count > 50,000/mm3 and is<br /><br>platelet transfusion independent for a minimum of seven days and will be<br /><br>required to have >90% donor chimerism.<br /><br><br /><br>Neutrophil Engraftment: Neutrophil engraftment is defined as achieving ANC ><br /><br>500/mm3 for three consecutive measurements over three or more days by Day 42<br /><br>and will be required to have >90% donor chimerism.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Infusional toxicity<br /><br>Adverse events<br /><br>Primary graft failure<br /><br>Overall survival<br /><br>Incidence of infection<br /><br>Immune reconstitution<br /><br>Acute graft-versus-host disease</p><br>