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

Sponsor
Novartis Pharmaceuticals (Industry)
Overall Status
Withdrawn
CT.gov ID
NCT02715505
Collaborator
(none)
0
Enrollment
1
Arm
31.2
Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Umbilical cord blood transplantation with HSC835
 Phase 1/Phase 2

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
0 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Anticipated Study Start Date :
Oct 10, 2017
Anticipated Primary Completion Date :
May 18, 2020
Anticipated Study Completion Date :
May 18, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: HSC835

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

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of infusional toxicities [48 hours]

    2. Incidence of neutrophil recovery [42 days]

    3. Incidence of graft failure [42 days]

    Secondary Outcome Measures

    1. Time to neutrophil recovery [42 days]

    2. Time to platelet recovery [180 days]

    3. Number of patients with grade II-IV acute graft versus host disease (aGVHD) [100 days]

    4. Number of patients with chronic graft versus host disease (cGVHD) [1 and 2 years]

    5. Incidence of death [100 days, 1 year and 2 years]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Months to 25 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Novartis Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Novartis Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT02715505
    Other Study ID Numbers:
    • CHSC835X2203
    First Posted:
    Mar 22, 2016
    Last Update Posted:
    Nov 28, 2018
    Last Verified:
    Nov 1, 2018
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Novartis Pharmaceuticals
    Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
    Additional relevant MeSH terms:
    Metabolic Diseases

    Study Results

    No Results Posted as of Nov 28, 2018