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A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders | Clinical Trials | Clareo Health

TERMINATEDPHASE1

A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders

A Phase 1 Trial of a Single ProHema® CB Product as Part of Single Cord Blood Unit Transplant After Busulfan/Cyclophosphamide/ATG Conditioning for Pediatric Patients With Inherited Metabolic Disorders

NCT02354443•Fate Therapeutics

View on ClinicalTrials.gov

Summary

The purpose of this study is to describe the safety profile of ProHema-CB as part of a single cord blood unit transplant after a myeloablative conditioning regimen in pediatric patients with inherited metabolic disorders. The safety profile will primarily be assessed by neutrophil engraftment.

Detailed Description

This study is an open-label trial of the safety of a single cord blood transplant using ProHema-CB following busulfan/cyclophosphamide/ATG conditioning for pediatric patients with inherited metabolic disorders. A maximum of 12 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 1 to 3 centers within the U.S. All subjects will be admitted to the hospital, per institutional practice and will receive a conditioning regimen, after which they will receive a HLA-matched or partially matched ProHema -CB unit on Day 0. They will receive study follow up assessments weekly following Day 0 through Day 100 and study visit Days 180, 270, 365 and 730.

Eligibility

Age

1 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Patients must have a confirmed diagnosis of an inherited metabolic disorder (IMD) and be amenable to treatment by hematopoietic cell transplantation:
•* Mucopolysaccharidoses: Hurler Syndrome (MPS IH), MPS I-HS (Hurler-Scheie Syndrome), Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), or MPS VI (Maroteaux-Lamy syndrome) with early neurologic involvement and/or sensitization to enzyme replacement therapy (ERT); or
•* Leukodystrophies: Krabbe disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN); or
•* Other IMD with lysosomal storage disorder including glycoproteinoses (Alpha-Mannosidosis, Mucolipidosis II or I-Cell disease), sphingo- and other lipidoses (Sandhoff disease, Tay Sachs disease, Pelizaeus Merzbacher (PMD), Niemann-Pick disease, GM1 gangliosidosis, Wolman's disease.
•2. Male and female subjects aged 1 to 18 years, inclusive.
•3. Lack of 4 6/6 HLA matched non-carrier related UCB or 8/8 HLA A, B, C, DRß1 matched non-carrier related or 8/8 unrelated bone marrow donor; or donor not available within appropriate timeframe, as determined by the transplant physician.
•4. Availability of suitable primary and secondary umbilical cord blood (UCB) units.
•5. Adequate performance status, defined as:
•* Subjects ≥ 16 years: Karnofsky score ≥ 70%.
•* Subjects \< 16 years: Lansky score ≥ 70%.
+7 more criteria

Exclusion Criteria

•1. Evidence of HIV infection or HIV positive serology.
•2. Current uncontrolled bacterial, viral or fungal infection (progression of clinical symptoms despite therapy).
•3. Requirement for continuous respiratory supportive therapy (e.g. ventilator). Patients on intermittent respiratory support should be discussed with the Sponsor.
•4. Active problems related to chronic aspiration.
•5. Uncontrolled seizures.
•6. Any active malignancy or myelodysplastic syndrome or any history of malignancy.
•7. Inability to give informed consent/assent or to comply with the requirements for care after allogeneic stem cell transplantation.
•8. Female subjects that are breastfeeding or with a positive pregnancy (HCG) test at Screening.
•9. Use of an investigational drug within 30 days prior to screening for the primary IMD.

Locations (2)

Boston Children's Hospital

Boston, Massachusetts, United States

Duke University Medical Center

Durham, North Carolina, United States

Key Dates

Start Date

June 1, 2015

Primary Completion Date

December 1, 2016

Completion Date

February 1, 2017

Last Updated

October 10, 2018

Enrollment

ACTUAL participants

Conditions

Metabolic Disorders

Interventions

ProHema-CB Transplant

BIOLOGICAL

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996

Severe Sickle Cell DiseaseBone Marrow Failure Syndromes+4 more

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RECRUITING

A Natural History Study of Metabolic Sizing in Health and Disease

NCT05398783

Metabolic DisordersCancer+3 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 10, 2018Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders

Inclusion Criteria

Exclusion Criteria

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