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Withdrawn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
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Inherited Metabolic Disorders IMD | Drug: Umbilical cord blood transplantation with HSC835 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
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 |
Estimated Study Start Date : | October 10, 2017 |
Estimated Primary Completion Date : | May 18, 2020 |
Estimated Study Completion Date : | May 18, 2020 |

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 |
- Incidence of infusional toxicities [ Time Frame: 48 hours ]
- Incidence of neutrophil recovery [ Time Frame: 42 days ]
- Incidence of graft failure [ Time Frame: 42 days ]
- Time to neutrophil recovery [ Time Frame: 42 days ]
- Time to platelet recovery [ Time Frame: 180 days ]
- Number of patients with grade II-IV acute graft versus host disease (aGVHD) [ Time Frame: 100 days ]
- Number of patients with chronic graft versus host disease (cGVHD) [ Time Frame: 1 and 2 years ]
- Incidence of death [ Time Frame: 100 days, 1 year and 2 years ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Months to 25 Years (Child, Adult) |
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
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT) |
Metabolic Diseases |