NCT02715505

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• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Months – 25 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Inherited Metabolic Disorders IMD

Interventions

Type	Name	Description
DRUG	Umbilical cord blood transplantation with HSC835	Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835

Timeline

• Start date: 2017-10-10
• Primary completion: 2020-05-18
• Completion: 2020-05-18
• First posted: 2016-03-22
• Last updated: 2018-11-28

Regulatory

• FDA-regulated drug study