Hematologic MalignanciesPhase 2NCT01627314
The PUMA Trial is a Trial of a Single ProHema Modulated-Cord Blood (CB) Unit as Part of a Double CB Transplant in Patients With Hematologic Malignancies.
A Phase 2 study testing ProHema-CB and Untreated CB in people with Hematologic Malignancies, run by Fate Therapeutics at 11 sites in United States.
Trial summary
This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. Read more about this study
- Who is it for?
- Ages 15 to 65 years
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- ProHema-CB and Untreated CB
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 11 sitesUnited States
- Study dates
- Jul 2012to May 2017
- Phase
- Phase 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 15 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:
- Acute lymphoblastic leukemia (ALL) (including T lymphoblastic lymphoma) in complete remission (CR).
- Remission is defined as < 5% blasts with no morphological characteristics of acute leukemia in a bone marrow with > 5% cellularity.
- Myelodysplastic disease, International Prognostic Scoring System (IPSS) Intermediate-2 or High risk (e.g., refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Patients have to have received leukemia type induction chemotherapy within ≤ 3 months and with ≤ 10% blasts by a bone marrow aspirate; a single hypomethylating agent is not considered adequate cytotoxic chemotherapy; all subtypes except chronic myelomonocytic leukemia (CMML).
- Acute myelogenous leukemia (AML) in high risk first CR or second or subsequent CR.
- High risk first CR is defined by but is not limited to at least one of the following factors: greater than one cycle of induction chemotherapy to achieve CR, prior myelodysplastic syndrome (MDS), presence of fms-like tyrosine kinase 3 (FLT3) abnormalities, French-American-British (FAB) M6 or M7 subtypes of leukemia, or adverse cytogenetics.
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- Remission is defined as < 5% blasts with no morphological characteristics of acute leukemia (e.g. Auer Rods) in a bone marrow with > 5% cellularity.
- AML arising from myelofibrosis is not permitted.
- Biphenotypic/undifferentiated leukemia in first or subsequent CR (same definition of CR as for ALL/AML).
- Chronic myelogenous leukemia (CML) with prior exposure to cytotoxic chemotherapy for the treatment of blast phase or with demonstrated intolerance to at least 2 tyrosine kinase inhibitors.
- Non Hodgkin's lymphoma (T cell, large cell or mantle cell) or Hodgkin's lymphoma in second or subsequent CR or in partial remission (PR) with documented chemosensitivity. In addition, marginal zone lymphoma or follicular lymphoma that has progressed after ≥ 2 therapies (excluding single agent rituximab).
- If the myeloablative conditioning regimen is selected, a history of prior myeloablative procedure is not allowed.
- If the reduced intensity conditioning regimen is selected, and the subject has had a prior autologous transplant, it must have taken place > 3 months from anticipated Day 0 visit.
- Lack of suitable 5 6/6 HLA matched related or (if institutional guidelines dictate) suitable 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.
- Both cord blood units (CBUs) are qualified by Fate Therapeutics
- Age 15 to 55 years (myeloablative regimen) or 15 to 65 years (reduced intensity regimen)
- Body weight > 45 kg
- Investigator selection of conditioning regimen (myeloablative or reduced intensity)
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 2.
- Signed Institutional Review Board (IRB) approved Informed Consent Form (ICF).
Who cannot join
- History of prior allogeneic transplantation
- Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction (Ejection fraction < 50%) as measured by gated radionuclide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of myocardial infarction with depressed ejection fraction.
- Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected diffusing capacity for carbon monoxide (DLCO) of < 50% of predicted, corrected for hemoglobin.
- Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min.
- Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or ongoing hemolytic anemia), aspartate aminotransferase (SGOT) or alanine aminotransferase (SGPT) > 5 × upper limit of normal.
- Neurologic disease: symptomatic leukoencephalopathy, active central nervous system (CNS) malignancy or other neuropsychiatric abnormalities believed to preclude transplantation.
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- HIV antibody.
- Uncontrolled infection.
- Pregnancy or breast feeding mother.
- Inability to comply with the requirements for care after allogeneic stem cell transplantation.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University-Winship Cancer Institute
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute- Hematopoietic Stem Cell Transplant Program
Boston, Massachusetts, 02215
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210
City of Hope
Duarte, California, 91010
Loyola University Medical Center
Maywood, Illinois, 60153
Mount Sinai Hospital
New York, New York, 10029
Oregon Health Sciences
Portland, Oregon, 97239
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase 2 Controlled Trial of a Single ProHema®-CB Unit (Ex Vivo Modulated Human Cord Blood) As Part of a Double Umbilical Cord Blood Transplant Following Myeloablative or Reduced Intensity Conditioning For Patients Age 15-65 Years With Hematologic Malignancies.
This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.
Read the detailed description
All subjects will receive a myeloablative or reduced intensity conditioning regimen, after which they will receive 2 Human Leukocyte Antigen (HLA)-matched or partially matched umbilical cord blood (UCB) units. A total of 40 subjects will receive one ProHema-CB as part of a double CB transplant and an additional 20 subjects will be enrolled as concurrent controls. The determination of which CB unit will be the ProHema-CB unit will be made based primarily upon the degree of HLA match.
- Conditions
- Hematologic Malignancies
- Being tested
- ProHema-CB, Untreated CB
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProHema-CBBiological
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Untreated CBBiological
Cord Blood
| Study group | Role | What they receive |
|---|---|---|
| ProHema-CB with MAC Preparative Regimen | Experimental | ProHema-CB and Wash-Only CB Unit with myeloablative conditioning regimen (MAC) Receives: Biological: ProHema-CB |
| ProHema-CB with RIC Preparative Regimen | Experimental | ProHema-CB and Wash-Only CB Unit with reduced intensity conditioning regimen (RIC) Receives: Biological: ProHema-CB |
| Control Arm with MAC Preparative Regimen | Placebo comparator | Two Wash-Only CB Units (Untreated CB) with myeloablative conditioning regimen Receives: Biological: Untreated CB |
| Control Arm with RIC Preparative Regimen | Placebo comparator | Two Wash-Only CB Units (Untreated CB) with reduced intensity conditioning regimen Receives: Biological: Untreated CB |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of early neutrophil engraftment using Myeloablative Conditioning
To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following myeloablative conditioning for subjects with hematologic malignancies.
Measured: Neutrophil engraftment < 26 days
Rate of early neutrophil engraftment using Reduced Intensity Conditioning
To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following reduced intensity conditioning for subjects with hematologic malignancies.
Measured: Neutrophil engraftment < 21 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2012 (estimated)
- Study startJul 2012
- Primary completionMar 2016
- Study completionMay 2017
Why it stopped: Business decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fate Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chris Storgard, M.D., Study director, Fate Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01627314
- Sponsor's protocol ID
- FT1050-03
- Status last verified
- Nov 2021
- First submitted
- 21 Jun 2012
- Last updated
- 30 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Controlled Trial of a Single ProHema®-CB Unit (Ex Vivo Modulated Human Cord Blood) As Part of a Double Umbilical Cord Blood Transplant Following Myeloablative or Reduced Intensity Conditioning For Patients Age 15-65 Years With Hematologic Malignancies.
- ClinicalTrials.gov ID
- NCT01627314
- Acronym / sponsor ID
- FT1050-03
- Conditions
- Hematologic Malignancies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business decision
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Fate TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Jul 2012
- Primary completion
- Mar 2016 (actual)
- Study completion
- May 2017 (actual)
- First posted
- 25 Jun 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.
This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.
Detailed description
All subjects will receive a myeloablative or reduced intensity conditioning regimen, after which they will receive 2 Human Leukocyte Antigen (HLA)-matched or partially matched umbilical cord blood (UCB) units. A total of 40 subjects will receive one ProHema-CB as part of a double CB transplant and an additional 20 subjects will be enrolled as concurrent controls. The determination of which CB unit will be the ProHema-CB unit will be made based primarily upon the degree of HLA match.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| ProHema-CB with MAC Preparative Regimen | Experimental | Biological: ProHema-CBProHema-CB and Wash-Only CB Unit with myeloablative conditioning regimen (MAC) |
| ProHema-CB with RIC Preparative Regimen | Experimental | Biological: ProHema-CBProHema-CB and Wash-Only CB Unit with reduced intensity conditioning regimen (RIC) |
| Control Arm with MAC Preparative Regimen | Placebo comparator | Biological: Untreated CBTwo Wash-Only CB Units (Untreated CB) with myeloablative conditioning regimen |
| Control Arm with RIC Preparative Regimen | Placebo comparator | Biological: Untreated CBTwo Wash-Only CB Units (Untreated CB) with reduced intensity conditioning regimen |
Who may qualify
- Ages
- 15 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:
- Lack of suitable 5 6/6 HLA matched related or (if institutional guidelines dictate) suitable 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.
- Both cord blood units (CBUs) are qualified by Fate Therapeutics
- Age 15 to 55 years (myeloablative regimen) or 15 to 65 years (reduced intensity regimen)
- Body weight > 45 kg
Key exclusion criteria (10 listed)
- History of prior allogeneic transplantation
- Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction (Ejection fraction < 50%) as measured by gated radionuclide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of myocardial infarction with depressed ejection fraction.
- Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected diffusing capacity for carbon monoxide (DLCO) of < 50% of predicted, corrected for hemoglobin.
- Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min.
- Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or ongoing hemolytic anemia), aspartate aminotransferase (SGOT) or alanine aminotransferase (SGPT) > 5 × upper limit of normal.
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 |
|---|---|---|
| ProHema-CBEx-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood | Biological | ProHema-CB with MAC Preparative Regimen, ProHema-CB with RIC Preparative Regimen |
| Untreated CBCord Blood | Biological | Control Arm with MAC Preparative Regimen, Control Arm with RIC Preparative Regimen |
Primary outcome
Rate of early neutrophil engraftment using Myeloablative Conditioning
Time frame: Neutrophil engraftment < 26 days
To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following myeloablative conditioning for subjects with hematologic malignancies.
Rate of early neutrophil engraftment using Reduced Intensity Conditioning
Time frame: Neutrophil engraftment < 21 days
To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant following reduced intensity conditioning for subjects with hematologic malignancies.
Results and safety
The study was stopped early (reason given: Business decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2021.
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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.
Chris Storgard, M.D. study director
Fate Therapeutics
11 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 ProHema-CB and Untreated CB in people with Hematologic Malignancies, run by Fate Therapeutics at 11 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 10 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 30 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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. Some participants in this study may receive a placebo. 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?
The study was stopped early (reason given: Business decision). Results are not available in the source record.
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?
Rate of early neutrophil engraftment using Myeloablative Conditioning (time frame: Neutrophil engraftment < 26 days). The study lists 2 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 30 Nov 2021.