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

Sponsored by:	Aldagen
Information provided by:	Aldagen
ClinicalTrials.gov Identifier:	NCT00654433

Purpose

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.

Condition	Intervention	Phase
Inherited Metabolic Diseases Lysosomal Storage Disorders Peroxisomal Storage Diseases Hematopoietic Stem Cell Transplantation	Biological: ALD-101	Phase III

Genetics Home Reference related topics: alpha-mannosidosis Krabbe disease metachromatic leukodystrophy mucopolysaccharidosis type I mucopolysaccharidosis type II Sandhoff disease Tay-Sachs disease X-linked adrenoleukodystrophy

MedlinePlus related topics: Blood Transfusion and Donation Leukodystrophies Metabolic Disorders

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
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

Further study details as provided by Aldagen:

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 [ Time Frame: 180 Days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To assess the efficacy of ALD-101 in accelerating neutrophil engraftment [ Time Frame: 180 Days ] [ Designated as safety issue: No ]
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. [ Time Frame: 180 Days ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	40
Study Start Date:	March 2008
Estimated Study Completion Date:	March 2010
Estimated Primary Completion Date:	November 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
I: Experimental	Biological: ALD-101 A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).

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.

Eligibility

Ages Eligible for Study:	up to 16 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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 (MPS III)
- Maroteau-Lamy Syndrome (MPS VI)
- Krabbe Disease (Globoid Leukodystrophy)
- Metachromatic Leukodystrophy (MLD)
- Adrenoleukodystrophy ALD and AMN)
- Sandhoff Disease
- Tay Sachs Disease
- Pelizaeus Merzbacher (PMD)
- Neiman-Pick Disease
- Alpha-mannosidosis
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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00654433

Contacts

Contact: Rachael Crozier, MS, RN	919-484-2571 ext 260	rcrozier@aldagen.com
Contact: Becky Durham	919-484-2571 ext 242	bdurham@aldagen.com

Locations

United States, California
Mattel Children's Hospital at UCLA	Recruiting
Los Angeles, California, United States, 90095
Contact: Kristen C. Venick, RN, MSN, CPN 310-825-6708 kvenick@mednet.ucla.edu
Contact: Alison Bell, RN, NP 310-825-6708 abell@mednet.ucla.edu
Principal Investigator: Theodore Moore, MD
United States, New York
Mt. Sinai Medical Center	Recruiting
New York, New York, United States, 27705
Contact: Rosa Chu 212-241-7022 rosa.chu@mssm.edu
Contact: Nita Narayan 212-241-4142 nita.narayan@mssm.edu
Principal Investigator: Gustavo Del Toro, MD
United States, North Carolina
Duke University	Recruiting
Durham, North Carolina, United States, 27705
Contact: Jennifer Baker, RN 919-668-6536 baker133@mc.duke.edu
Contact: Caroline Elkins, RN 919-668-1280 caroline.elkins@duke.edu
Principal Investigator: Joanne Kurtzberg, MD

Sponsors and Collaborators

Aldagen

Investigators

Study Director:	Laurence Keller, MD	Aldagen
Principal Investigator:	Joanne Kurtzberg, MD	Duke University

More Information

Sponsor's Website This link exits the ClinicalTrials.gov site

Publications:

Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99-108. Epub 2008 Jan 7. Review.

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.

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. Epub 2006 Aug 21.

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.

Responsible Party:	Aldagen, Inc. ( Laurence Keller, MD Chief Medical Officer )
Study ID Numbers:	UCBT-002, BB-IND#13502
Study First Received:	April 3, 2008
Last Updated:	December 10, 2008
ClinicalTrials.gov Identifier:	NCT00654433
Health Authority:	United States: Food and Drug Administration

Keywords provided by Aldagen:

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
AMN
Sandhoff Disease
Tay Sachs Disease
Pelizaeus Merzbacher
PMD
Neiman-Pick Disease
Alpha-mannosidosis
Inherited metabolic diseases
Inborn errors of metabolism
Lysosomal storage diseases
Peroxisomal storage diseases
Hematopoietic stem cell transplantation
Umbilical cord blood transplantation

Study placed in the following topic categories:

Hunter-McAlpine syndrome
Mucopolysaccharidosis II
Pick Disease of the Brain
Gangliosidosis (GM2) Type1
Addison's disease
Leukodystrophy, Metachromatic
Frontotemporal dementia
Tay-Sachs disease
Aphasia, Primary Progressive
Leukodystrophy
Krabbe leukodystrophy
Alpha-L-iduronidase deficiency
Metabolism, Inborn Errors
Adrenoleukodystrophy
Addison Disease

Primary progressive aphasia
Metachromatic leukodystrophy
Tay-Sachs Disease
Sandhoff disease
Leukodystrophy, Globoid Cell
X-linked adrenoleukodystrophy
Alpha-Mannosidosis
Metabolic Diseases
Lysosomal Storage Diseases
Mannosidase Deficiency Diseases
Mucopolysaccharidosis I
Genetic Diseases, Inborn
Hunter syndrome
Lobar atrophy of brain
Metabolic disorder

ClinicalTrials.gov processed this record on January 16, 2009