ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

July 7, 2014 updated by: Aldagen

A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism

Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease. A portion of cord blood cells (ALD-101) will be separated from the cord blood unit and given approximately 4 hours after the standard cord blood transfusion.

The study will test if the supplemental cells will increase the speed at which normal levels of circulating blood cells are re-established after transplant.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Subjects will be hospitalized and undergo high doses of chemotherapy which will destroy the child's normal cells including their bone marrow (which forms blood cells) in order to prepare their body for the umbilical cord blood transplant. The cord blood transplant is intended to rescue your child's bone marrow from the bad effects of the procedure. The child will receive 80% of a standard cord blood transplant followed by 20% supplemental stem cell called ALD-101.

The study will evaluate if these cells (ALD-101) will repopulate the bone marrow more rapidly after transplant. This would shorten the period of time that the transplanted subject would be at risk for infection and bleeding and would also decrease the number of red blood cell and platelet transfusions needed.

ALD-101 has been used as a supplement to cord blood transplant in twenty-eight children with genetic diseases and malignancy in one previous clinical study that is on-going.

The main purpose of this research study is to test whether a portion of cord blood cells that have been separated from a cord blood unit (ALD-101) will increase the speed at which normal levels of circulating blood cells are re-established after transplant. This is the experimental part of this study. ALD-101 is also being tested to see if it is safe.

Study Type

Interventional

Enrollment (Anticipated)

40

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Mattel Children's Hospital at UCLA
    • New York
      • New York, New York, United States, 27705
        • Mt. Sinai Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • confirmed diagnosis of inherited metabolic diseases; including the following:

    • Hurler Syndrome (MPS I)
    • Hurler-Scheie Syndrome
    • Hunter Syndrome (MPS II)
    • Sanfilippo Syndrome A and B(MPS III)
    • Maroteaux-Lamy Syndrome (MPS VI)
    • Krabbe Disease (Globoid Leukodystrophy)
    • Metachromatic Leukodystrophy (MLD)
    • Adrenoleukodystrophy (ALD and AMN)
    • Sandhoff Disease
    • Tay Sachs Disease
    • Pelizaeus Merzbacher (PMD)
    • Niemann-Pick Disease
    • Alpha-mannosidosis
    • I-Cell Disease (ML II)
    • Fucosidosis
    • GM I Gangliosidosis
    • Canavan Disease
  • must be <16 years of age at the time of study enrollment
  • must have a good performance status (Lansky ≥80%)
  • must have adequate function of other organ systems including: kidney, liver, heart and lungs
  • must have given valid written informed consent
  • must have a minimum life expectancy of at least 6 months
  • must be determined to be a good candidate for a standard umbilical cord blood transplant
  • must have an IQ >70 or if too young for IQ testing the potential to reach this endpoint by age 5

Exclusion Criteria:

  • HIV, Hepatitis B and/or Hepatitis C positive
  • concurrently involved in any other clinical study that affects engraftment or immune reconstitution
  • uncontrolled seizures, apnea, evidence of aspiration pneumonia, or evidence of brain stem involvement
  • uncontrolled infections
  • prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of enrollment

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: I
Biological: ALD-101
A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases
Time Frame: 180 Days
180 Days

Secondary Outcome Measures

Outcome Measure
Time Frame
To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
Time Frame: 180 Days
180 Days
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
Time Frame: 180 Days
180 Days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Aldagen

Investigators

  • Study Director: James Hinson, MD, Aldagen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2008

Primary Completion (Actual)

July 1, 2011

Study Completion (Actual)

November 1, 2011

Study Registration Dates

First Submitted

April 3, 2008

First Submitted That Met QC Criteria

April 7, 2008

First Posted (Estimate)

April 8, 2008

Study Record Updates

Last Update Posted (Estimate)

July 8, 2014

Last Update Submitted That Met QC Criteria

July 7, 2014

Last Verified

July 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UCBT-002
  • BB-IND#13502

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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