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

Inherited Metabolic Disorders IMD Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02715505
• Other study ID #: CHSC835X2203
• Status: Withdrawn
• Phase: Phase 1/Phase 2
• Start date: October 10, 2017
• Est. completion date: May 18, 2020

Study information

• Verified date: November 2018
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: May 18, 2020
• Est. primary completion date: May 18, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 25 Years

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Inherited Metabolic Disorders IMD
• Metabolic Diseases

Intervention

Drug:
• Umbilical cord blood transplantation with HSC835
Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of infusional toxicities		48 hours
Primary	Incidence of neutrophil recovery		42 days
Primary	Incidence of graft failure		42 days
Secondary	Time to neutrophil recovery		42 days
Secondary	Time to platelet recovery		180 days
Secondary	Number of patients with grade II-IV acute graft versus host disease (aGVHD)		100 days
Secondary	Number of patients with chronic graft versus host disease (cGVHD)		1 and 2 years
Secondary	Incidence of death		100 days, 1 year and 2 years