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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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.

City, postcode or hospital

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

Other trials in United States

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

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 groups in NCT02743351
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.

20 Dec 20165 Nov 2021

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)

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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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.

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

Results posted to the registry on 8 Feb 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase I ProTmune	7	7
Phase 2 ProTmune	45	35
Phase 2 Control	44	36

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. Number (95% Confidence Interval). Unit: percentage of participants

Measure	Phase I ProTmune n = 7	Phase 2 ProTmune n = 40	Phase 2 Control n = 41
Value	42.9 (7.6 to 75.7)	44.4 (28.1 to 59.5)	24.9 (12.7 to 39.1)

Population analysed: Modified intent-to-treat (mITT) population * Phase 1: All subjects who received ProTmune * Phase 2: All randomized subjects who received Investigational Product (ProTmune or Control mPB units)

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of investigational product up to 730 days post-HCT. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase I ProTmune	3 of 7 (42.9%)	7 of 7 (100%)	2 of 7
Phase 2 ProTmune	24 of 40 (60%)	40 of 40 (100%)	12 of 40
Control Arm	20 of 41 (48.8%)	41 of 41 (100%)	6 of 41

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?

On the ClinicalTrials.gov record for NCT02743351.

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.