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

Phase 1

Withdrawn

• Conditions: Inherited Metabolic Disorders IMD
• Interventions: Drug: Umbilical cord blood transplantation with HSC835

• Registration Number: NCT02715505
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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.

Detailed 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.

Study Timeline

• Study First Submitted: 2016-03-16
• Study First Submitted QC: 2016-03-16
• Study First Posted: 2016-03-22
• Study Start: 2017-10-10
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-26
• Last Update Posted: 2018-11-28
• Primary Completion: 2020-05-18
• Study Completion: 2020-05-18

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HSC835	Umbilical cord blood transplantation with HSC835	HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation

Primary Outcome Measures

Name	Time	Method
Incidence of graft failure	42 days
Incidence of infusional toxicities	48 hours
Incidence of neutrophil recovery	42 days

Secondary Outcome Measures

Name	Time	Method
Time to neutrophil recovery	42 days
Time to platelet recovery	180 days
Number of patients with grade II-IV acute graft versus host disease (aGVHD)	100 days
Number of patients with chronic graft versus host disease (cGVHD)	1 and 2 years
Incidence of death	100 days, 1 year and 2 years