NCT00654433

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2008

Typical duration for phase_3

Geographic Reach
1 country

3 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2008

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

April 3, 2008

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 8, 2008

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2011

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2011

Completed
Last Updated

July 8, 2014

Status Verified

July 1, 2014

Enrollment Period

3.3 years

First QC Date

April 3, 2008

Last Update Submit

July 7, 2014

Conditions

Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage DiseasesInborn Errors of MetabolismMucopolysaccharidosis

Keywords

Hurler SyndromeMPS IHurler-Scheie SyndromeHunter SyndromeMPS IISanfilippo SyndromeMPS IIIMaroteaux-Lamy SyndromeMPS VIKrabbe DiseaseGloboid LeukodystrophyMetachromatic LeukodystrophyMLDAdrenoleukodystrophyALDAdrenomyeloneuropathyAMNSandhoff DiseaseTay Sachs DiseasePelizaeus MerzbacherPMDNiemann-Pick Disease ANiemann-Pick Disease BAlpha-mannosidosisInherited metabolic diseasesInborn errors of metabolismLysosomal storage diseasesPeroxisomal storage diseasesHematopoietic stem cell transplantationUmbilical cord blood transplantationMucopolysaccharidosisMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IIIMucopolysaccharidosis VII Cell diseaseFucosidosisGM1 GangliosidosisCanavan Disease

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (2)

  • To assess the efficacy of ALD-101 in accelerating neutrophil engraftment

    180 Days

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

    180 Days

Study Arms (1)

I

EXPERIMENTAL
Biological: ALD-101

Interventions

ALD-101BIOLOGICAL

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

I

Eligibility Criteria

AgeUp to 16 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)
  • +10 more criteria

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Mattel Children's Hospital at UCLA

Los Angeles, California, 90095, United States

Location

Mt. Sinai Medical Center

New York, New York, 27705, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Related Publications (4)

  • 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.

    PMID: 18176609BACKGROUND

  • 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.

    PMID: 16443516BACKGROUND

  • 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.

    PMID: 16923928BACKGROUND

  • 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.

    PMID: 17882722BACKGROUND

Related Links

MeSH Terms

Conditions

Lysosomal Storage DiseasesMetabolism, Inborn ErrorsMucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis IISudden Infant DeathMucopolysaccharidosis IIIMucopolysaccharidosis VILeukodystrophy, Globoid CellLeukodystrophy, MetachromaticAdrenoleukodystrophySandhoff DiseaseTay-Sachs Diseasealpha-MannosidosisMucolipidosesFucosidosisGangliosidosis, GM1Canavan Disease

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesCarbohydrate Metabolism, Inborn ErrorsMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesGenetic Diseases, X-LinkedHeredodegenerative Disorders, Nervous SystemDeath, SuddenDeathPathologic ProcessesPathological Conditions, Signs and SymptomsInfant DeathHereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesSphingolipidosesLysosomal Storage Diseases, Nervous SystemLeukoencephalopathiesDemyelinating DiseasesLipidosesLipid Metabolism, Inborn ErrorsLipid Metabolism DisordersSulfatidosisPeroxisomal DisordersAdrenal InsufficiencyAdrenal Gland DiseasesEndocrine System DiseasesGangliosidoses, GM2GangliosidosesMannosidase Deficiency DiseasesBone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesNeurodegenerative Diseases

Study Officials

  • James Hinson, MD

    Aldagen

    STUDY DIRECTOR

  • Joanne Kurtzberg, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 3, 2008

First Posted

April 8, 2008

Study Start

March 1, 2008

Primary Completion

July 1, 2011

Study Completion

November 1, 2011

Last Updated

July 8, 2014

Record last verified: 2014-07

Locations

US(3)