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

Study Overview
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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.

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.

Official Title
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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

Conditions
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Inherited Metabolic Disorders IMD

Intervention / Treatment
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* Drug: Umbilical cord blood transplantation with HSC835


Participation Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Months
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)