Mucopolysaccharidosis IPhase 1HPDSCNCT01586455
Human Placental-Derived Stem Cell Transplantation
A Phase 1 study testing Human Placental Derived Stem Cell in people with Mucopolysaccharidosis I and Mucopolysaccharidosis VI, run by New York Medical College at 3 sites in United States.
Trial summary
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Read more about this study
- Who is it for?
- Ages 0 to 55 years
- Conditions the study is for
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy
- What is being tested?
- Human Placental Derived Stem Cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2013to Sep 2022
- Phase
- Phase 1
- 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 0 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- < 55 years of age
- Life expectancy greater than 3 months
- Lansky performance status ≥ 50% (children) or Karnofsky performance status ≥ 70% (adults) or ECOG performance status 0-2 (adults)
- DLCO > 50 percent predicted
- Left ventricular ejection fraction > 40% estimated
- Creatinine clearance or estimated GFR . 60 mL/min/1.73m2
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- Serum bilirubin < 1.5x upper limit of normal
- Transaminases < 3x upper limit of normal
- Absence of uncontrolled infection
- HIV negative
Who cannot join
- Fanconi Anemia
- Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities
- Uncontrolled infection
- Pregnant or breast-feeding females
- Received other investigational agents within 30 days prior to the start of the conditioning regimen
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Children's Hospital Colorado
Denver, Colorado
University of Utah
Salt Lake City, Utah
New York Medical College
Valhalla, New York, 10595
About this study
Official title: A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease
- Being tested
- Human Placental Derived Stem Cell
- Keywords
- umbilical cord blood, stem cell transplantation, placental stem cell, inborn errors of metabolism, marrow failure, Severe Combined Immunodeficiency Disease, AML, ALL, MDS
- Medical subject headings
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Diseases, Leukodystrophy, Metachromatic, Wolman Disease, Leukodystrophy, Globoid Cell, Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Placental Derived Stem CellDrug
Also called HPDSC
Infusions of thawed HPDSC to be given following UCB infusion.
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | related cord blood with ≥3/6 HLA match to the patient and related HPDSC Receives: Drug: Human Placental Derived Stem Cell |
| Group B | Experimental | unrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC Receives: Drug: Human Placental Derived Stem Cell |
| Group C | Experimental | unrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC Receives: Drug: Human Placental Derived Stem Cell |
| Group D | Experimental | double unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC Receives: Drug: Human Placental Derived Stem Cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
to evaluate the safety of human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells in patients with malignant and non-malignant diseases.
Measured: 100 days
Secondary outcomes (5)
donor chimerism
donor chimerism will be assessed at set timepoints
Measured: 1 year
engraftment
Measured: 1 year
Survival
Measured: 100 days and 180 days
Relapse
Measured: 100 days and 180 days
Mortality
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2012 (estimated)
- Study startApr 2013
- Primary completionJun 2020
- Study completionSep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mitchell S Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01586455
- Sponsor's protocol ID
- NYMC 550
- Other IDs
- NYMC IRB L-10,733
- Acronym
- HPDSC
- Status last verified
- Oct 2022
- First submitted
- 25 Apr 2012
- Last updated
- 25 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
- ClinicalTrials.gov ID
- NCT01586455
- Acronym / sponsor ID
- HPDSC / NYMC 550
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease
- Keywords
- umbilical cord blood, stem cell transplantation, placental stem cell, inborn errors of metabolism, marrow failure, Severe Combined Immunodeficiency Disease, AML, ALL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Study start
- Apr 2013 (actual)
- Primary completion
- Jun 2020 (actual)
- Study completion
- Sep 2022 (actual)
- First posted
- 26 Apr 2012 (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.
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Drug: Human Placental Derived Stem Cellrelated cord blood with ≥3/6 HLA match to the patient and related HPDSC |
| Group B | Experimental | Drug: Human Placental Derived Stem Cellunrelated cord blood with ≥ 4/6 HLA match to the patient and unrelated HPDSC |
| Group C | Experimental | Drug: Human Placental Derived Stem Cellunrelated cord blood with ≥4/6 HLA match to the patient but related to HPDSC |
| Group D | Experimental | Drug: Human Placental Derived Stem Celldouble unrelated cord blood units with ≥4/6 HLA match to patient and each other and unrelated HPDSC |
Who may qualify
- Ages
- 0 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- < 55 years of age
- Life expectancy greater than 3 months
- Lansky performance status ≥ 50% (children) or Karnofsky performance status ≥ 70% (adults) or ECOG performance status 0-2 (adults)
- DLCO > 50 percent predicted
- Left ventricular ejection fraction > 40% estimated
Key exclusion criteria (5 listed)
- Fanconi Anemia
- Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities
- Uncontrolled infection
- Pregnant or breast-feeding females
- Received other investigational agents within 30 days prior to the start of the conditioning regimen
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 |
|---|---|---|
| Human Placental Derived Stem CellAlso: HPDSCInfusions of thawed HPDSC to be given following UCB infusion. | Drug | Group A, Group B, Group C, Group D |
Primary outcome
Safety
Time frame: 100 days
to evaluate the safety of human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells in patients with malignant and non-malignant diseases.
5 secondary outcomes
donor chimerism
Time frame: 1 year
engraftment
Time frame: 1 year
Survival
Time frame: 100 days and 180 days
Relapse
Time frame: 100 days and 180 days
Mortality
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2022.
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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.
Mitchell S Cairo, MD principal investigator
New York Medical College
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Human Placental Derived Stem Cell in people with Mucopolysaccharidosis I and Mucopolysaccharidosis VI, run by New York Medical College at 3 sites in United States.
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 10 inclusion and 5 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: finished, as of the sponsor's last update on 25 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 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.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety (time frame: 100 days). 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 25 Oct 2022.