Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

NCT01003912 | Withdrawn Phase 1 DMC

• 1 other identifier: Pro00010188
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to determine if it is safe to administer unrelated umbilical cord blood to pregnant women in their first trimester of pregnancy with a fetus that has a known diagnosis of certain lysosomal storage diseases. These diseases are known to cause severe and irreversible neurological disability in early infancy and which are lethal in childhood.

Conditions

Lysosomal Storage Diseases; Inborn Errors of Metabolism

Keywords

Lysosomal Storage Diseases; Inborn Errors of Metabolism; Krabbe; Infantile metachromatic leukodystrophy disease; MLD; Tay Sachs; Sandhoff; GM2 gangliosidoses; Pelizaeus-Merzbacher disease; PMD

Outcome Measures

Primary Outcomes (2)

• To determine whether immune tolerance and donor cell engraftment can be achieved through first trimester injection of donor cells to fetus's diagnosed with lethal LSDs.

  after 3 patients

• Safety and feasibility of fetal intrap.

  after 3 patients

Secondary Outcomes (3)

• Donor chimerism for neonate at birth and 7 days post delivery.

  after 3 patients

• Establishment of threshold enzyme levels in neonate at birth and 7 days post delivery.

  after 3 patients

• Donor chimerism for mother post delivery and 1 year post date of birth.

  after 3 patients

Interventions

ALD-601 BIOLOGICAL

ALD-601 is manufactured by Aldagen from the 20% compartment of the selected umbilical cord blood unit within 24 hrs of planned injection. A dose of ALD-601 that delivers 1 x 105 - 2 x 106 cells/kg of estimated fetal weight at day - 1 is suspended in 300 microliters of dialysis media.

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Fetus with known enzymatic and, if possible, mutation diagnosis of selected lysosomal storage diseases, and history of enzymatic or mutation diagnosis of a previously affected family member.
• Krabbe disease
• Infantile metachromatic leukodystrophy disease (MLD)
• Tay Sachs or Sandhoff (GM2 gangliosidoses) disease
• Pelizaeus-Merzbacher disease (PMD).
• Both parents have the "at risk" genotype
• Both parents agree to psychological counseling prior to CVS
• Both parents agree to delivery of subject at Duke University
• Gestational age of \< 9 weeks by sure menstrual dating or ultrasound of a natural pregnancy.
• Singleton fetus
• No evidence of abnormal nuchal thickening on preliminary ultrasound prior to chorion villus biopsy

You may not qualify if:

• Pregnancy by in vitro fertilization
• Evidence of a nuchal translucency of \> 3.0 mm at the time of the ultrasound for the chorion villus biopsy.
• HIV, hepatitis C or hepatitis B positive maternal status
• Active infection in the mother at the time of scheduled CVS or cell injection as determined by clinical practice

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead
• Aldagen: collaborator

Study Sites (2)

Duke University Medical Center Pediatric Blood and Marrow Transplant

Durham, North Carolina, 27705, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Lysosomal Storage Diseases; Metabolism, Inborn Errors; Gangliosidoses, GM2; Pelizaeus-Merzbacher Disease

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Gangliosidoses; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; Genetic Diseases, X-Linked

Study Officials

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: October 20, 2009
• First Posted: October 29, 2009
• Study Start: October 1, 2009
• Primary Completion: March 1, 2017
• Study Completion: March 1, 2017
• Last Updated: June 29, 2017

Record last verified: 2017-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)