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

NCT02715505 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: CHSC835X2203
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Inherited Metabolic Disorders IMD

Keywords

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

Outcome Measures

Primary Outcomes (3)

• Incidence of infusional toxicities

  48 hours

• Incidence of neutrophil recovery

  42 days

• Incidence of graft failure

  42 days

Secondary Outcomes (5)

• 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

Study Arms (1)

HSC835

EXPERIMENTAL

HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation

Drug: Umbilical cord blood transplantation with HSC835

Interventions

Umbilical cord blood transplantation with HSC835 DRUG

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

Also known as: Spanlecortemlocel

HSC835

Eligibility Criteria

• Age: 12 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Mucopolysaccharidosis I; Leukodystrophy, Metachromatic

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sulfatidosis; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Leukoencephalopathies; Demyelinating Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2016
• First Posted: March 22, 2016
• Study Start: October 10, 2017
• Primary Completion: May 18, 2020
• Study Completion: May 18, 2020
• Last Updated: November 28, 2018

Record last verified: 2018-11