Back to resultsSafety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
Primary Purpose
Inherited Metabolic Disorders IMD
Status
Withdrawn
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Umbilical cord blood transplantation with HSC835
Sponsored by
About this trial
This is an interventional treatment trial for Inherited Metabolic Disorders IMD focused on measuring Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
Eligibility Criteria
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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HSC835
Arm Description
HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation
Outcomes
Primary Outcome Measures
Incidence of infusional toxicities
Incidence of neutrophil recovery
Incidence of graft failure
Secondary Outcome Measures
Time to neutrophil recovery
Time to platelet recovery
Number of patients with grade II-IV acute graft versus host disease (aGVHD)
Number of patients with chronic graft versus host disease (cGVHD)
Incidence of death
Full Information
NCT ID
NCT02715505
First Posted
March 16, 2016
Last Updated
November 26, 2018
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02715505
Brief Title
Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2018
Overall Recruitment Status
Withdrawn
Why Stopped
This study was cancelled with no patients
Study Start Date
October 10, 2017 (Anticipated)
Primary Completion Date
May 18, 2020 (Anticipated)
Study Completion Date
May 18, 2020 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inherited Metabolic Disorders IMD
Keywords
Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
HSC835
Arm Type
Experimental
Arm Description
HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation
Intervention Type
Drug
Intervention Name(s)
Umbilical cord blood transplantation with HSC835
Other Intervention Name(s)
Spanlecortemlocel
Intervention Description
Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835
Primary Outcome Measure Information:
Title
Incidence of infusional toxicities
Time Frame
48 hours
Title
Incidence of neutrophil recovery
Time Frame
42 days
Title
Incidence of graft failure
Time Frame
42 days
Secondary Outcome Measure Information:
Title
Time to neutrophil recovery
Time Frame
42 days
Title
Time to platelet recovery
Time Frame
180 days
Title
Number of patients with grade II-IV acute graft versus host disease (aGVHD)
Time Frame
100 days
Title
Number of patients with chronic graft versus host disease (cGVHD)
Time Frame
1 and 2 years
Title
Incidence of death
Time Frame
100 days, 1 year and 2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
12. IPD Sharing Statement
Plan to Share IPD
Undecided
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Learn more about this trial
Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
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