Hematologic MalignanciesPhase 1NCT02354417
A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
A Phase 1 study testing Biological: ProHema-CB in people with Hematologic Malignancies, run by Fate Therapeutics at 2 sites in United States.
Trial summary
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- Biological: ProHema-CB
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2014to Feb 2017
- Phase
- Phase 1
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female subjects aged 1 to 18 years, inclusive.
- Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
- Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
- Acute Lymphoblastic Leukemia (ALL) in CR
- NK cell lymphoblastic leukemia in any CR
- Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
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- Myelodysplastic Syndrome (MDS) at any stage.
- Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
- Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe, as determined by the transplant physician.
- Availability of suitable primary and secondary umbilical cord blood (UCB) units.
- Adequate performance status, defined as:
- Subjects ≥ 16 years: Karnofsky score ≥ 70%.
- Subjects < 16 years: Lansky score ≥ 70%.
- Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%.
- Pulmonary:
- Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for hemoglobin)
- FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation > 92% on room air.
- Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70mL/min/1.73m2.
- Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
- Signed IRB approved Informed Consent Form (ICF).
Who cannot join
- Female subjects that are pregnant or breastfeeding.
- Evidence of HIV infection or HIV positive serology.
- Current uncontrolled bacterial, viral or fungal infection.
- Prior allogeneic hematopoietic stem cell transplant.
- Autologous transplant < 12 months prior to enrollment.
- Prior autologous transplant for the disease for which the UCB transplant is being performed.
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- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment.
- Inability to receive TBI.
- Requirement of supplemental oxygen.
- HLA-matched related donor able to donate.
- Use of an investigational drug within 30 days prior to screening.
- Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Boston Children's Hospital
Boston, Massachusetts, 02115-5450
City of Hope
Duarte, California, 91010
About this study
Official title: A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
Read the detailed description
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18 subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1 to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled simultaneously.
All subjects will be admitted to the hospital, per institutional practice, and will receive a myeloablative conditioning regimen, after which they will receive an HLA-matched or partially matched ProHema-CB unit on study Day 0.
- Conditions
- Hematologic Malignancies
- Being tested
- Biological: ProHema-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.
Biological: ProHema-CBBiological
Each subject will receive one administration of ProHema-CB unit transplant.
| Study group | Role | What they receive |
|---|---|---|
| ProHema-CB | Experimental | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant.
ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight:
For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min.
For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. Receives: Biological: Biological: ProHema-CB |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Profile, Primarily Assessed by Neutrophil Engraftment
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.
Measured: Neutrophil engraftment by Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2015 (estimated)
- Study startDec 2014
- Primary completionDec 2016
- Study completionFeb 2017
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fate Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chris Storgard, MD, Study director, Fate Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02354417
- Sponsor's protocol ID
- FT1050-04
- Status last verified
- Feb 2018
- First submitted
- 25 Sep 2014
- Last updated
- 10 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT02354417
- Acronym / sponsor ID
- FT1050-04
- Conditions
- Hematologic Malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Fate TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Dec 2016 (actual)
- Study completion
- Feb 2017 (actual)
- First posted
- 3 Feb 2015 (estimated)
- Results
- Posted 10 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
Detailed description
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18 subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1 to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled simultaneously.
All subjects will be admitted to the hospital, per institutional practice, and will receive a myeloablative conditioning regimen, after which they will receive an HLA-matched or partially matched ProHema-CB unit on study Day 0.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ProHema-CB | Experimental | Biological: Biological: ProHema-CBAll subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (20 listed)
- Male and female subjects aged 1 to 18 years, inclusive.
- Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
- Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe, as determined by the transplant physician.
- Availability of suitable primary and secondary umbilical cord blood (UCB) units.
- Adequate performance status, defined as:
Key exclusion criteria (12 listed)
- Female subjects that are pregnant or breastfeeding.
- Evidence of HIV infection or HIV positive serology.
- Current uncontrolled bacterial, viral or fungal infection.
- Prior allogeneic hematopoietic stem cell transplant.
- Autologous transplant < 12 months prior to enrollment.
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 |
|---|---|---|
| Biological: ProHema-CBEach subject will receive one administration of ProHema-CB unit transplant. | Biological | ProHema-CB |
Primary outcome
Safety Profile, Primarily Assessed by Neutrophil Engraftment
Time frame: Neutrophil engraftment by Day 42
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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, MD study director
Fate Therapeutics
2 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 Biological: ProHema-CB in people with Hematologic Malignancies, run by Fate Therapeutics at 2 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 12 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 10 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
Yes. Results are posted in the registry (first posted 10 Oct 2018). 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?
Safety Profile, Primarily Assessed by Neutrophil Engraftment (time frame: Neutrophil engraftment by Day 42). 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 10 Oct 2018.