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

Novartis

Status and phase

Withdrawn

Phase 2

Phase 1

Conditions

Inherited Metabolic Disorders IMD

Treatments

Drug: Umbilical cord blood transplantation with HSC835

Study type

Interventional

Funder types

Industry

Identifiers

NCT02715505

CHSC835X2203

Details and patient eligibility

About

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.

Full description

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.

Sex

All

Ages

12 months to 25 years old

Volunteers

No Healthy Volunteers

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