Clinical Trials Logo

Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT00654433
Other study ID # UCBT-002
Secondary ID BB-IND#13502
Status Terminated
Phase Phase 3
First received April 3, 2008
Last updated July 7, 2014
Start date March 2008
Est. completion date November 2011

Study information

Verified date July 2014
Source Aldagen
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Terminated
Enrollment 40
Est. completion date November 2011
Est. primary completion date July 2011
Accepts healthy volunteers No
Gender Both
Age group N/A to 16 Years
Eligibility 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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

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

Locations

Country Name City State
United States Duke University Durham North Carolina
United States Mattel Children's Hospital at UCLA Los Angeles California
United States Mt. Sinai Medical Center New York New York

Sponsors (1)

Lead Sponsor Collaborator
Aldagen

Country where clinical trial is conducted

United States, 

References & Publications (4)

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. — View 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. — View 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. — View 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. Review. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary 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 180 Days No
Secondary To assess the efficacy of ALD-101 in accelerating neutrophil engraftment 180 Days No
Secondary To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. 180 Days Yes
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03548779 - North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 N/A
Completed NCT01049854 - CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant Phase 2
Completed NCT00001596 - Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome Phase 2
Withdrawn NCT01003912 - Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases Phase 1
Completed NCT00744692 - Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders Phase 1
Completed NCT00692926 - Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells Phase 1
Recruiting NCT05818566 - Orphan Drugs for Inherited Metabolic Diseases
Completed NCT05330039 - Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
Withdrawn NCT03866954 - Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy Phase 2
Completed NCT03911089 - A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD N/A
Completed NCT03058848 - Evaluation of PKU Start N/A
Suspended NCT04399694 - Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
Completed NCT03168399 - Evaluation of PKU Explore N/A
Recruiting NCT00078078 - Clinical and Laboratory Study of Methylmalonic Acidemia
Completed NCT00328159 - Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids N/A
Completed NCT04309331 - Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute N/A
Completed NCT04709965 - Evaluating Face-Recognition Technology in Syndrome Diagnosis N/A
Recruiting NCT06360913 - Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases N/A
Completed NCT00309400 - The Early History of Universal Screening for Metabolic Disorders N/A
Completed NCT00004378 - Stem Cell Transplantation (SCT) for Genetic Diseases N/A