Related Donors Donating PBSC to a Family MemberPhase 2NCT04762875
MGTA-145 + Plerixafor in the Mobilization of HSCs for Allogeneic Transplant in Hematologic Malignancies
A Phase 2 study testing MGTA-145 and Plerixafor in people with Related Donors Donating PBSC to a Family Member and Healthy Donors, run by Ensoma at 8 sites in United States.
Trial summary
This research study tests a new medicine for mobilizing stem cells so they can be collected and used for allogeneic stem cell transplant for treatment of hematological malignancies. MGTA-145, the new medicine, will be given with plerixafor. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Related Donors Donating PBSC to a Family Member, Healthy Donors, Acute Myelogenous Leukemia
- What is being tested?
- MGTA-145 and Plerixafor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 8 sitesUnited States
- Study dates
- 16 Jun 2021to 14 Mar 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Donor Inclusion Criteria:
- Donor medical suitability and eligibility will be determined following Institution or NMDP/Be The Match standards
- Age 18-65 years old at the time of signing informed consent
- 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor
- Fulfill Institution or NMDP/Be The Match criteria to serve as a mobilized blood cell donor
- Serum creatinine < 1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CRCL) > 50 mL/min using the Modification of Diet in Renal Disease Study (MDRD) equation or similar method
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- Recipient Inclusion Criteria:
- At least 18 years old at the time of signing informed consent
- Has an available 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor willing to donate peripheral blood stem cells (PBSC) for transplant
- Fulfill additional individual Transplant Center Criteria for transplant beyond NMDP/Be The Match criteria
- One of the following diagnoses:
- Acute myelogenous leukemia (AML) in 1st remission or beyond with ≤ 5% marrow blasts and no circulating blasts. Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
- Acute lymphoblastic leukemia (ALL) in 1st remission or beyond with ≤ 5% marrow blasts and no circulating blasts. Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
- Patients with myelodysplasia (MDS) with no circulating blasts and with less than 10% blasts in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in outcomes with < 5% or 5-10% blasts in MDS). Documentation of bone marrow assessment will be accepted within 45 days prior to the date of consent.
- Cardiac function: Left ventricular ejection fraction at least 45% based on most recent echocardiogram or MUGA results obtained via standard of care
- Estimated creatinine clearance acceptable per local institutional guidelines
- Pulmonary function: diffusing capacity of the lungs for carbon monoxide (DLCO) corrected for hemoglobin at least 50% and forced expiratory volume in first second (FEV1) predicted at least 50% based on most recent DLCO results obtained via standard of care
- Liver function acceptable per local institutional guidelines
- Karnofsky performance status (KPS) of 70% or greater
- Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score of 4 or less
Who cannot join
- Donor Exclusion Criteria:
- Donor unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
- Donor already enrolled on another investigational agent study
- Pregnant or breastfeeding females, sexually active female and male donors not willing or able to use adequate contraception, or males who do not agree to refrain from donating sperm, from the time of consent through 3 months after treatment with MGTA-145 + plerixafor
- Recipient Exclusion Criteria:
- Subject unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
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- Subject whose donor does not meet the eligibility criteria and is a screen fail
- Subjects with a prior allogeneic transplant
- Subjects with active, uncontrolled infection at the time of the transplant preparative regimen
- Pregnant or breastfeeding females, sexually active female or male subjects not willing or able to use adequate contraception, or males who do not agree to refrain from donating sperm, from the time of consent through 3 months after PBSC infusion
- Subjects with clinical evidence of active Central Nervous System (CNS) tumor involvement as evidenced by documented disease on examination of spinal fluid or MRI within 45 days of start of conditioning
- A condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of primary and secondary endpoints
- Planned treatment with a new investigational agent from the time of transplant through 30 days post-transplant
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Emory University Hospital
Atlanta, Georgia, 30322
Roswell Park Cancer Institute
Buffalo, New York, 14203
Ohio State Medical Center, James Cancer Center
Columbus, Ohio, 43210
City of Hope National Medical Center
Duarte, California, 91010
M.D. Anderson Cancer Center
Houston, Texas, 77030
Mayo Clinic Rochester
Rochester, Minnesota, 55902
Be The Match Collection Center Seattle
Seattle, Washington, 98101
Stanford Health Care
Stanford, California, 94305
About this study
Official title: A Phase II Study Evaluating the Safety and Efficacy of MGTA-145 in Combination With Plerixafor for the Mobilization and Transplantation of HLA-Matched Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies
This research study tests a new medicine for mobilizing stem cells so they can be collected and used for allogeneic stem cell transplant for treatment of hematological malignancies. MGTA-145, the new medicine, will be given with plerixafor.
Read the detailed description
This is a Phase II, open-label, multicenter, prospective study of MGTA-145 + plerixafor mobilized HLA-matched sibling and matched unrelated donor allografts for myeloablative hematopoietic stem cell transplantation (HSCT) in recipients with hematological malignancies. Donors will undergo 1 or 2 days of mobilization and apheresis.
- Conditions
- Related Donors Donating PBSC to a Family Member, Healthy Donors, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
- Being tested
- MGTA-145, Plerixafor
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MGTA-145Biological
MGTA-145 will be be administered as an IV infusion
PlerixaforBiological
Also called Mozobil
240 µg/kg subcutaneously
| Study group | Role | What they receive |
|---|---|---|
| Single dose MGTA-145 plus plerixafor followed by apheresis | Experimental | MGTA-145 in combination with plerixafor followed by apheresis on one or two consecutive days Receives: Biological: MGTA-145; Biological: Plerixafor |
What is being measured
The primary outcome is the main question the study is designed to answer.
HSC Yield in Apheresis Product
Number of subjects with adequate number of hematopoietic stem cells (≥ 2.0 x 10^6 CD34+ cells/kg) in one apheresis setting.
Measured: Up to 2 days
Secondary outcomes (6)
HSC Yield in Apheresis Product
To determine the proportion of donors whose cells can be successfully mobilized and collected with a target CD34+ cell dose of at least 4.0 x 10^6 CD34+ cells/kg actual recipient weight in one apheresis collection
Measured: Up to 2 days
Adverse Events Experienced by Donors
To ascertain the incidence of adverse events (AEs) before and during apheresis experienced by donors receiving MGTA-145 + plerixafor
Measured: Baseline though day 180
Graft Durability
The proportion of participants with primary and secondary graft failure after transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor
Measured: Day 28
Graft-versus Host Disease (GVHD)
To determine the incidence of acute and chronic graft versus host disease (GVHD) after transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor
Measured: Day 100
Treatment-related Mortality
To determine the proportion of treatment-related mortality and disease relapse/progression after transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor
Measured: Day 100
Overall Survival
To determine the probability of overall survival after transplantation of hematopoietic cells mobilized with MGTA-145 + plerixafor
Measured: Day 100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2021
- Study start16 Jun 2021
- Primary completion14 Sep 2021
- Study completion14 Mar 2022
Why it stopped: This study was voluntarily terminated due to a business decision not to proceed and not due to any safety issue
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Related Donors Donating PBSC to a Family MemberStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ensoma
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Marrow Donor Program
- Responsible party
- Sponsor
- Investigators
- Steven Devine, MD, Study chair, National Marrow Donor Program
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04762875
- Sponsor's protocol ID
- 145-ADS-202
- Status last verified
- Oct 2024
- First submitted
- 12 Feb 2021
- Last updated
- 6 Nov 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Evaluating the Safety and Efficacy of MGTA-145 in Combination With Plerixafor for the Mobilization and Transplantation of HLA-Matched Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT04762875
- Acronym / sponsor ID
- 145-ADS-202
- Conditions
- Related Donors Donating PBSC to a Family Member, Healthy Donors, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was voluntarily terminated due to a business decision not to proceed and not due to any safety issue
- Enrolment
- 7 participants (actual)
- Lead sponsor
- EnsomaIndustry (pharma, biotech, devices)
- Collaborators
- National Marrow Donor Program
- Study start
- 16 Jun 2021 (actual)
- Primary completion
- 14 Sep 2021 (actual)
- Study completion
- 14 Mar 2022 (actual)
- First posted
- 21 Feb 2021 (actual)
- Results
- Posted 29 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study tests a new medicine for mobilizing stem cells so they can be collected and used for allogeneic stem cell transplant for treatment of hematological malignancies. MGTA-145, the new medicine, will be given with plerixafor.
Detailed description
This is a Phase II, open-label, multicenter, prospective study of MGTA-145 + plerixafor mobilized HLA-matched sibling and matched unrelated donor allografts for myeloablative hematopoietic stem cell transplantation (HSCT) in recipients with hematological malignancies. Donors will undergo 1 or 2 days of mobilization and apheresis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single dose MGTA-145 plus plerixafor followed by apheresis | Experimental | Biological: MGTA-145, Biological: PlerixaforMGTA-145 in combination with plerixafor followed by apheresis on one or two consecutive days |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Donor medical suitability and eligibility will be determined following Institution or NMDP/Be The Match standards
- Age 18-65 years old at the time of signing informed consent
- 8/8 (HLA- A, B, C, and DRB1) HLA-matched sibling or volunteer unrelated donor
- Fulfill Institution or NMDP/Be The Match criteria to serve as a mobilized blood cell donor
- Serum creatinine < 1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CRCL) > 50 mL/min using the Modification of Diet in Renal Disease Study (MDRD) equation or similar method
Key exclusion criteria (13 listed)
- Donor unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
- Donor already enrolled on another investigational agent study
- Pregnant or breastfeeding females, sexually active female and male donors not willing or able to use adequate contraception, or males who do not agree to refrain from donating sperm, from the time of consent through 3 months after treatment with MGTA-145 + plerixafor
- Subject unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
- Subject whose donor does not meet the eligibility criteria and is a screen fail
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 |
|---|---|---|
| MGTA-145MGTA-145 will be be administered as an IV infusion | Biological | Single dose MGTA-145 plus plerixafor followed by apheresis |
| PlerixaforAlso: Mozobil240 µg/kg subcutaneously | Biological | Single dose MGTA-145 plus plerixafor followed by apheresis |
Primary outcome
HSC Yield in Apheresis Product
Time frame: Up to 2 days
Number of subjects with adequate number of hematopoietic stem cells (≥ 2.0 x 10^6 CD34+ cells/kg) in one apheresis setting.
6 secondary outcomes
HSC Yield in Apheresis Product
Time frame: Up to 2 days
Adverse Events Experienced by Donors
Time frame: Baseline though day 180
Graft Durability
Time frame: Day 28
Graft-versus Host Disease (GVHD)
Time frame: Day 100
Treatment-related Mortality
Time frame: Day 100
Overall Survival
Time frame: Day 100
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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.
Steven Devine, MD study chair
National Marrow Donor Program
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MGTA-145 and Plerixafor in people with Related Donors Donating PBSC to a Family Member and Healthy Donors, run by Ensoma at 8 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 20 inclusion and 13 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: stopped early, as of the sponsor's last update on 6 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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 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?
Yes. Results are posted in the registry (first posted 29 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
HSC Yield in Apheresis Product (time frame: Up to 2 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 6 Nov 2024.