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

A Single-arm, Open-label, Study to Evaluate the Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Stem Cell Transplantation After Reduced Intensity Conditioning

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.

Study Overview

• Status: Withdrawn
• Conditions: Inherited Metabolic Disorders IMD
• Intervention / Treatment: Drug: Umbilical cord blood transplantation with HSC835

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 Type: Interventional
• Phase: Phase 2; Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HSC835; HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation	Drug: Umbilical cord blood transplantation with HSC835; Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835; Other Names: Spanlecortemlocel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of infusional toxicities	48 hours
Incidence of neutrophil recovery	42 days
Incidence of graft failure	42 days

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Anticipated): October 10, 2017
• Primary Completion (Anticipated): May 18, 2020
• Study Completion (Anticipated): May 18, 2020

Study Registration Dates

• First Submitted: March 16, 2016
• First Submitted That Met QC Criteria: March 16, 2016
• First Posted (Estimate): March 22, 2016

Study Record Updates

• Last Update Posted (Actual): November 28, 2018
• Last Update Submitted That Met QC Criteria: November 26, 2018
• Last Verified: November 1, 2018

More Information

Terms related to this study

• Keywords: Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
• Additional Relevant MeSH Terms: Metabolic Diseases
• Other Study ID Numbers: CHSC835X2203

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No