Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1/PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-10

Study Completion Date

2020-05-18

Brief Summary

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This study is designed to assess the safety and exploratory efficacy of using HSC835 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.

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Detailed Description

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This phase II study is designed to assess the safety of the Novartis product HSC835 and its ability to achieve donor blood stem cell engraftment in patients with certain Inherited Metabolic Disorders who undergo stem cell transplantation. A reduced intensity conditioning will be used prior to transplantation. Patients with Hurler syndrome, MLD, Krabbe or cALD could be eligible for this study.

Conditions

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Inherited Metabolic Disorders IMD

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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HSC835

HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation

Group Type EXPERIMENTAL

Umbilical cord blood transplantation with HSC835

Intervention Type DRUG

Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835

Interventions

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Umbilical cord blood transplantation with HSC835

Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835

Intervention Type DRUG

Other Intervention Names

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Spanlecortemlocel

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material

Exclusion Criteria

* Availability of a matched-related donor who is not a carrier of the same genetic defect -Active infection at screening -Prior myeloablative transplant -Pregnant or nursing women and women of child bearing potential unless using highly effective contraception methods. For the pediatric population, female patients of child bearing potential who do not agree to abstinence or agree to use highly effective contraception methods -Sexually active male patients unless using condoms as contraception -Human Immunodeficiency virus (HIV) infection

Minimum Eligible Age

12 Months

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No