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

Status

Withdrawn

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02715505

Enrollment

Registered

2016-03-22

Start date

2017-10-10

Completion date

2020-05-18

Last updated

2018-11-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Inherited Metabolic Disorders IMD

Keywords

Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)

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.

Interventions

DRUGUmbilical cord blood transplantation with HSC835

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

12 Months to 25 Years

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

Design outcomes

Primary

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

Secondary

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026