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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02715505
Recruitment Status : Withdrawn (This study was cancelled with no patients)
First Posted : March 22, 2016
Last Update Posted : November 28, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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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
Inherited Metabolic Disorders IMD 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
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
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
Estimated Study Start Date : October 10, 2017
Estimated Primary Completion Date : May 18, 2020
Estimated Study Completion Date : May 18, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: Spanlecortemlocel


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of infusional toxicities [ Time Frame: 48 hours ]
  2. Incidence of neutrophil recovery [ Time Frame: 42 days ]
  3. Incidence of graft failure [ Time Frame: 42 days ]

Secondary Outcome Measures :
  1. Time to neutrophil recovery [ Time Frame: 42 days ]
  2. Time to platelet recovery [ Time Frame: 180 days ]
  3. Number of patients with grade II-IV acute graft versus host disease (aGVHD) [ Time Frame: 100 days ]
  4. Number of patients with chronic graft versus host disease (cGVHD) [ Time Frame: 1 and 2 years ]
  5. Incidence of death [ Time Frame: 100 days, 1 year and 2 years ]

Eligibility Criteria
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Ages Eligible for Study:   12 Months to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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No Contacts or Locations Provided
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02715505    
Other Study ID Numbers: CHSC835X2203
First Posted: March 22, 2016    Key Record Dates
Last Update Posted: November 28, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
Additional relevant MeSH terms:
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Metabolic Diseases