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

Primary Purpose

Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases

Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
ALD-101
Sponsored by
Aldagen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Inherited Metabolic Diseases focused on measuring Hurler Syndrome, MPS I, Hurler-Scheie Syndrome, Hunter Syndrome, MPS II, Sanfilippo Syndrome, MPS III, Maroteaux-Lamy Syndrome, MPS VI, Krabbe Disease, Globoid Leukodystrophy, Metachromatic Leukodystrophy, MLD, Adrenoleukodystrophy, ALD, Adrenomyeloneuropathy, AMN, Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher, PMD, Niemann-Pick Disease A, Niemann-Pick Disease B, Alpha-mannosidosis, Inherited metabolic diseases, Inborn errors of metabolism, Lysosomal storage diseases, Peroxisomal storage diseases, Hematopoietic stem cell transplantation, Umbilical cord blood transplantation, Mucopolysaccharidosis, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis VI, I Cell disease, Fucosidosis, GM1 Gangliosidosis, Canavan Disease

Eligibility Criteria

undefined - 16 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Mattel Children's Hospital at UCLA
  • Mt. Sinai Medical Center
  • Duke University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

I

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.

Full Information

First Posted
April 3, 2008
Last Updated
July 7, 2014
Sponsor
Aldagen
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1. Study Identification

Unique Protocol Identification Number
NCT00654433
Brief Title
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Acronym
UCBT-002
Official Title
A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
Study Type
Interventional

2. Study Status

Record Verification Date
July 2014
Overall Recruitment Status
Terminated
Why Stopped
Terminated - Sponsor Decision
Study Start Date
March 2008 (undefined)
Primary Completion Date
July 2011 (Actual)
Study Completion Date
November 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aldagen

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Keywords
Hurler Syndrome, MPS I, Hurler-Scheie Syndrome, Hunter Syndrome, MPS II, Sanfilippo Syndrome, MPS III, Maroteaux-Lamy Syndrome, MPS VI, Krabbe Disease, Globoid Leukodystrophy, Metachromatic Leukodystrophy, MLD, Adrenoleukodystrophy, ALD, Adrenomyeloneuropathy, AMN, Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher, PMD, Niemann-Pick Disease A, Niemann-Pick Disease B, Alpha-mannosidosis, Inherited metabolic diseases, Inborn errors of metabolism, Lysosomal storage diseases, Peroxisomal storage diseases, Hematopoietic stem cell transplantation, Umbilical cord blood transplantation, Mucopolysaccharidosis, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis VI, I Cell disease, Fucosidosis, GM1 Gangliosidosis, Canavan Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
I
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
ALD-101
Intervention Description
A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
Time Frame
180 Days
Title
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
Time Frame
180 Days

10. Eligibility

Sex
All
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Hinson, MD
Organizational Affiliation
Aldagen
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Joanne Kurtzberg, MD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mattel Children's Hospital at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Mt. Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
27705
Country
United States
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
18176609
Citation
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99-108. doi: 10.1038/sj.bmt.1705970. Epub 2008 Jan 7.
Results Reference
background
PubMed Identifier
16443516
Citation
Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.
Results Reference
background
PubMed Identifier
16923928
Citation
Escolar ML, Poe MD, Martin HR, Kurtzberg J. A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics. 2006 Sep;118(3):e879-89. doi: 10.1542/peds.2006-0747. Epub 2006 Aug 21.
Results Reference
background
PubMed Identifier
17882722
Citation
Gentry T, Deibert E, Foster SJ, Haley R, Kurtzberg J, Balber AE. Isolation of early hematopoietic cells, including megakaryocyte progenitors, in the ALDH-bright cell population of cryopreserved, banked UC blood. Cytotherapy. 2007;9(6):569-76. doi: 10.1080/14653240701466347.
Results Reference
background
Links:
URL
http://www.aldagen.com
Description
Sponsor's Website

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

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