Hematologic MalignanciesPhase 1/2NCT02743351
Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies
A Phase 1/2 study testing ProTmune in people with Hematologic Malignancies and Acute Myeloid Leukemia, run by Fate Therapeutics at 15 sites in United States.
Trial summary
This study is a Phase 1, non-randomized, open-label/Phase 2 randomized, blinded study of ProTmune (ex vivo programmed mobilized peripheral blood cells) versus non-programmed mobilized peripheral blood cells for allogeneic hematopoietic cell transplantation (HCT) in adult subjects aged 18 years and older with… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancies, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
- What is being tested?
- ProTmune
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 96 participants
- Where
- 15 sitesUnited States
- Study dates
- 20 Dec 2016to 5 Nov 2021
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female patients aged 18 years and older, inclusive;
- Patients must have a hematologic malignancy for which allogeneic hematopoietic peripheral blood cell transplantation is deemed clinically appropriate.
- Eligible diseases and stages include the following:
- Acute myeloid leukemia
- Acute lymphoblastic leukemia, including T lymphoblastic lymphoma with a history of marrow involvement
- Myelodysplastic Syndrome
Show all 17Show fewer
- Chronic myelogenous leukemia
- Availability of a suitable 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated mPB donor;
- mBP donor collection that meets protocol specifications;
- Adequate performance status, defined as Karnofsky score greater than or equal to 70%;
- For female patients of childbearing potential, all of the following criteria must be met:
- They are not pregnant (i.e., female patients must have a negative serum pregnancy test at screening);
- They are not breastfeeding;
- They do not plan to become pregnant during the study; and
- They are using an effective method of contraception from screening to the end of the study, unless their sexual partner is surgically sterile.
- For male patients, agreement to use condoms with spermicide during sexual intercourse from screening to the end of study; and
- Willingness and ability to sign an IRB/IEC-approved ICF before performance of any study-specific procedures or tests and to comply with protocol visits, and study procedures.
Who cannot join
- Phase 1 only: Known bone marrow fibrosis; Phase 2 only: Bone marrow fibrosis grade 3 (severe) or greater;
- Positive serology for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV) at any time prior to enrollment;
- Currently uncontrolled bacterial, viral, or fungal infection (progression of clinical symptoms despite therapy);
- Prior autologous or allogeneic HCT;
- Active malignancy, other than the one for which the allogeneic mPB transplant is being performed, within 12 months of enrollment, excluding superficial basal cell and carcinoma in situ cervical cancer;
- Pulmonary disease, renal dysfunction, hepatic disease, cardiac disease, neurologic disease;
- Participation in another clinical trial involving an investigational product within 30 days prior to screening; or
- Any condition or therapy, which, in the opinion of the Investigator, might pose a risk to the patient or make participation in the study not in the best interest of the patient.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
University of Alabama
Birmingham, Alabama
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
University of Chicago
Chicago, Illinois
Jewish Hospital
Cincinnati, Ohio
The Ohio State University
Columbus, Ohio, 43210
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201
City of Hope
Duarte, California, 91010
Indiana Blood and Marrow Transplant
Indianapolis, Indiana, 46237
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
Weill Cornell Medicine
New York, New York
Oregon Health & Science University
Portland, Oregon
Virginia Commonwealth University
Richmond, Virginia, 23298
Huntsman Cancer Institute (University of Utah)
Salt Lake City, Utah, 84103
Texas Transplant Institute
San Antonio, Texas
University of California, San Diego (UCSD) Moores Cancer Center
San Diego, California
About this study
Official title: A Phase 1, Non-Randomized, Open-Label/Phase 2, Randomized, Blinded Study of ProTmune™ (ex Vivo Programmed Mobilized Peripheral Blood Cells) Versus Non-Programmed Mobilized Peripheral Blood Cells for Allogeneic Hematopoietic Cell Transplantation in Adult Subjects With Hematologic Malignancies
This study is a Phase 1, non-randomized, open-label/Phase 2 randomized, blinded study of ProTmune (ex vivo programmed mobilized peripheral blood cells) versus non-programmed mobilized peripheral blood cells for allogeneic hematopoietic cell transplantation (HCT) in adult subjects aged 18 years and older with hematologic malignancies. A total of 88 study subjects were treated in the trial at approximately 15 centers in the US.
- Conditions
- Hematologic Malignancies, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Chronic Myelogenous Leukemia, Acute Graft-versus-host Disease
- Being tested
- ProTmune, Control Arm
- Keywords
- Cell Transplants, Hematopoietic Cell Transplant, HCT, Hematologic Malignancies, Hematologic Malignancy, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, ALL, AML, Myelodysplastic Syndrome, MDS, Allogeneic, CML, Stem Cell Transplant, Transplant, aGvHD, Acute graft-versus-host Disease, cGvHD, Chronic graft-versus-host Disease
- Medical subject headings
- Hematologic Neoplasms, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProTmuneBiological
Ex-vivo, programmed mobilized peripheral blood (mPB) cells
Control ArmBiological
Untreated mobilized peripheral blood (mPB) cells
| Study group | Role | What they receive |
|---|---|---|
| ProTmune | Experimental | Conditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140).
Subjects will receive mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor that were programmed ex vivo with ProTmune. Receives: Biological: ProTmune |
| Control Arm | Active comparator | Conditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140).
Subjects will receive unmanipulated mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor. Receives: Biological: Control Arm |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment
Acute GvHD (aGvHD) is assessed by assigning the clinical stage for the target organs (skin, liver, and gut) along with assigning an overall grade based on the minimum degree of organ involvement required to confer that grade. Grade II is defined as Stage 1 skin, Stage 1 liver, or Stage 1 GI; Grade III is defined as any Stage 1-3 skin, and Stage 2-3 liver, or Stage 2-4 GI; Grade IV is defined as Stage 4 skin, or Stage 4 liver and any Stage GI. A higher overall grade indicates a more severe outcome. The cumulative incidence of CIBMTR Grade II to IV aGVHD through approximately 100 days following HCT is measured by the percentage of participants who experienced grade II to IV aGVHD. The cumulative incidence and the associated 95% confidence interval are estimated using a competing risk analysis with death and relapse without grade II-IV aGvHD as a competing risk.
Measured: 100 days post-HCT
Secondary outcomes (2)
1-year GvHD-free, Relapse-free Survival (GRFS)
1-year GvHD-free, relapse-free survival (GRFS) is a composite endpoint in which events included Grade III to IV aGvHD, cGvHD requiring systemic immunosuppressive therapy, relapse, or death from any cause. GRFS is defined as the time from HCT to the earlier of the dates of the first documented CIBMTR Grade III-IV aGvHD, first use of systemic immunosuppressive therapy for cGvHD, first documented relapse of the underlying malignancy, or death from any cause. Subjects who are alive with no documented events for Grade III-IV aGvHD, use of systemic immunosuppressive therapy for cGvHD, relapse, or death at the data cutoff will be censored at their last visit date or follow-up on or prior to the date of one-year study completion/early discontinuation. The Kaplan-Meier estimate of the median GRFS and the 95% CI are presented
Measured: 365 days post-HCT
Percentage of Subjects Alive Without Relapse and Without Moderate or Severe cGvHD at Day +365 - mITT Population
Percentage of subjects alive without relapse and without moderate or severe cGvHD per NIH Consensus Criteria at Day +365
Measured: 365 days post-HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2016 (estimated)
- Study start20 Dec 2016
- Primary completion4 Dec 2020
- Study completion5 Nov 2021
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 Hematologic MalignanciesStudies 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 1/2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fate Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sarah Cooley, MD, Study director, Fate Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02743351
- Sponsor's protocol ID
- PT-001
- Status last verified
- Dec 2022
- First submitted
- 9 Feb 2016
- Last updated
- 8 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Non-Randomized, Open-Label/Phase 2, Randomized, Blinded Study of ProTmune™ (ex Vivo Programmed Mobilized Peripheral Blood Cells) Versus Non-Programmed Mobilized Peripheral Blood Cells for Allogeneic Hematopoietic Cell Transplantation in Adult Subjects With Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT02743351
- Acronym / sponsor ID
- PT-001
- Conditions
- Hematologic Malignancies, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Chronic Myelogenous Leukemia, Acute Graft-versus-host Disease
- Keywords
- Cell Transplants, Hematopoietic Cell Transplant, HCT, Hematologic Malignancies, Hematologic Malignancy, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, ALL
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Fate TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Dec 2016 (actual)
- Primary completion
- 4 Dec 2020 (actual)
- Study completion
- 5 Nov 2021 (actual)
- First posted
- 19 Apr 2016 (estimated)
- Results
- Posted 8 Feb 2023
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 a Phase 1, non-randomized, open-label/Phase 2 randomized, blinded study of ProTmune (ex vivo programmed mobilized peripheral blood cells) versus non-programmed mobilized peripheral blood cells for allogeneic hematopoietic cell transplantation (HCT) in adult subjects aged 18 years and older with hematologic malignancies. A total of 88 study subjects were treated in the trial at approximately 15 centers in the US.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ProTmune | Experimental | Biological: ProTmuneConditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140). Subjects will receive mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor that were programmed ex vivo with ProTmune. |
| Control Arm | Active comparator | Biological: Control ArmConditioning regimen consisting of one of the following five preparative regimens: fludarabine and busulfan (FluBu4); busulfan (Bu) and cyclophosphamide (Cy); Cy and >or=12 Gy total body irradiation (TBI); TBI and etoposide; or fludarabine and melphalan (FluMel 140). Subjects will receive unmanipulated mPB cells from an available 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated peripheral blood cell donor. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male and female patients aged 18 years and older, inclusive;
- Patients must have a hematologic malignancy for which allogeneic hematopoietic peripheral blood cell transplantation is deemed clinically appropriate.
- Availability of a suitable 8/8 HLA-A, -B, -C, and -DRB1-matched unrelated mPB donor;
- mBP donor collection that meets protocol specifications;
- Adequate performance status, defined as Karnofsky score greater than or equal to 70%;
Key exclusion criteria (8 listed)
- Phase 1 only: Known bone marrow fibrosis; Phase 2 only: Bone marrow fibrosis grade 3 (severe) or greater;
- Positive serology for human immunodeficiency virus (HIV) or human T-cell lymphotropic virus (HTLV) at any time prior to enrollment;
- Currently uncontrolled bacterial, viral, or fungal infection (progression of clinical symptoms despite therapy);
- Prior autologous or allogeneic HCT;
- Active malignancy, other than the one for which the allogeneic mPB transplant is being performed, within 12 months of enrollment, excluding superficial basal cell and carcinoma in situ cervical cancer;
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 |
|---|---|---|
| ProTmuneEx-vivo, programmed mobilized peripheral blood (mPB) cells | Biological | ProTmune |
| Control ArmUntreated mobilized peripheral blood (mPB) cells | Biological | Control Arm |
Primary outcome
Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment
Time frame: 100 days post-HCT
Acute GvHD (aGvHD) is assessed by assigning the clinical stage for the target organs (skin, liver, and gut) along with assigning an overall grade based on the minimum degree of organ involvement required to confer that grade. Grade II is defined as Stage 1 skin, Stage 1 liver, or Stage 1 GI; Grade III is defined as any Stage 1-3 skin, and Stage 2-3 liver, or Stage 2-4 GI; Grade IV is defined as Stage 4 skin, or Stage 4 liver and any Stage GI. A higher overall grade indicates a more severe outcome. The cumulative incidence of CIBMTR Grade II to IV aGVHD through approximately 100 days following HCT is measured by the percentage of participants who experienced grade II to IV aGVHD. The cumulative incidence and the associated 95% confidence interval are estimated using a competing risk analysis with death and relapse without grade II-IV aGvHD as a competing risk.
2 secondary outcomes
1-year GvHD-free, Relapse-free Survival (GRFS)
Time frame: 365 days post-HCT
Percentage of Subjects Alive Without Relapse and Without Moderate or Severe cGvHD at Day +365 - mITT Population
Time frame: 365 days post-HCT
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Sarah Cooley, MD study director
Fate Therapeutics
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ProTmune in people with Hematologic Malignancies and Acute Myeloid Leukemia, run by Fate Therapeutics at 15 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 17 inclusion and 8 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 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 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 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?
Yes. Results are posted in the registry (first posted 8 Feb 2023). 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?
Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment (time frame: 100 days post-HCT). 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 8 Feb 2023.