Inherited Metabolic Disorders IMDPhase 1/2NCT02715505
Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
A Phase 1/2 study testing Umbilical cord blood transplantation with HSC835 in people with Inherited Metabolic Disorders IMD, run by Novartis Pharmaceuticals.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 months to 25 years
- Conditions the study is for
- Inherited Metabolic Disorders IMD
- What is being tested?
- Umbilical cord blood transplantation with HSC835
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 10 Oct 2017to 18 May 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material
Who cannot join
- 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
About this study
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
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.
Read the 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.
- Conditions
- Inherited Metabolic Disorders IMD
- Being tested
- Umbilical cord blood transplantation with HSC835
- Keywords
- Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
- Medical subject headings
- Mucopolysaccharidosis I, Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical cord blood transplantation with HSC835Drug
Also called Spanlecortemlocel
Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835
| Study group | Role | What they receive |
|---|---|---|
| HSC835 | Experimental | HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation Receives: Drug: Umbilical cord blood transplantation with HSC835 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of infusional toxicities
Measured: 48 hours
Incidence of neutrophil recovery
Measured: 42 days
Incidence of graft failure
Measured: 42 days
Secondary outcomes (5)
Time to neutrophil recovery
Measured: 42 days
Time to platelet recovery
Measured: 180 days
Number of patients with grade II-IV acute graft versus host disease (aGVHD)
Measured: 100 days
Number of patients with chronic graft versus host disease (cGVHD)
Measured: 1 and 2 years
Incidence of death
Measured: 100 days, 1 year and 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2016 (estimated)
- Study start10 Oct 2017 (estimated)
- Primary completion18 May 2020 (estimated)
- Study completion18 May 2020 (estimated)
Why it stopped: This study was cancelled with no patients
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inherited Metabolic Disorders IMDStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02715505
- Sponsor's protocol ID
- CHSC835X2203
- Status last verified
- Nov 2018
- First submitted
- 16 Mar 2016
- Last updated
- 28 Nov 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02715505
- Acronym / sponsor ID
- CHSC835X2203
- Conditions
- Inherited Metabolic Disorders IMD
- Keywords
- Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: This study was cancelled with no patients
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2017 (estimated)
- Primary completion
- 18 May 2020 (estimated)
- Study completion
- 18 May 2020 (estimated)
- First posted
- 22 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSC835 | Experimental | Drug: Umbilical cord blood transplantation with HSC835HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation |
Who may qualify
- Ages
- 12 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material
Key exclusion criteria (1 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical cord blood transplantation with HSC835Also: SpanlecortemlocelHematopoietic Stem cell transplantation will be done with the cell therapy product HSC835 | Drug | HSC835 |
Primary outcome
Incidence of infusional toxicities
Time frame: 48 hours
Incidence of neutrophil recovery
Time frame: 42 days
Incidence of graft failure
Time frame: 42 days
5 secondary outcomes
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2018.
- Individual participant dataSharing planned: Undecided. 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
Related trials for Inherited Metabolic Disorders IMD
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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RecruitingObservational study
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Umbilical cord blood transplantation with HSC835 in people with Inherited Metabolic Disorders IMD, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 28 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of infusional toxicities (time frame: 48 hours). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2018.