Hematologic MalignanciesPhase 1NCT00890500
Safety and Efficacy of ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematologic Malignancies.
A Phase 1 study testing Fludarabine, Melphalan and Antithymocyte Globulin in people with Hematologic Malignancies and Allogeneic Stem Cell Transplantation, run by Fate Therapeutics at 2 sites in United States.
Trial summary
The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hematologic Malignancies, Allogeneic Stem Cell Transplantation
- What is being tested?
- Fludarabine, Melphalan and Antithymocyte Globulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2011to Oct 2013
- Phase
- Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
- Patient must be ineligible for traditional myeloablative transplantation according to treating physician
- Lack of 6/6 or 5/6 HLA-matched related, 8/8 HLA-matched unrelated donor, or unrelated donor not available within a time frame necessary to perform a potentially curative stem cell transplant
- 18-65 years of age
- ECOG Performance Status 0-2
Who cannot join
- The following hematologic malignancies are excluded:
- Myelofibrosis unless there has been exposure to cytotoxic chemotherapy for the treatment of progression to acute myeloid leukemia
- Chronic Myelogenous Leukemia, unless there has been exposure to cytotoxic chemotherapy for the treatment of blast phase, 3) Aplastic anemia, in the absence of transformation to Myelodysplastic disorder
- Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction as measured by gated radionucleotide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension
- Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected hemoglobin
- Renal disease: serum creatinine > 2.0mg/dl
Show all 12Show fewer
- Hepatic disease: serum bilirubin > 2.0mg/dl (expect in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT > 3 x upper limit of normal
- Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
- 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
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
Official title: A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies.
The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.
Read the detailed description
The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.
-As part of this research study cord units will be modulated in the laboratory with ProHema before it is given to the participant. Two different treatment groups will be tested. Group 1: will have the second cord blood unit modulated with ProHema; Group 2: will have the first cord blood unit modulated with ProHema.
- Being tested
- Fludarabine, Melphalan, Antithymocyte Globulin, Sirolimus, Tacrolimus
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
30mg/m2/day IV x 6 days
MelphalanDrug
100 mg/m2/day IV x 1 day
Antithymocyte GlobulinDrug
1mg/kg/day x 4 days
SirolimusDrug
GVHD Prophylaxis: Target range 3-12 ng/ml
TacrolimusDrug
GVHD Prophylaxis: Target range 5-10 ng/ml
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Active comparator | 2 umbilical cord units: Second cord blood unit modulated with ProHema Receives: Drug: Fludarabine; Drug: Melphalan; Drug: Antithymocyte Globulin; Drug: Sirolimus; Drug: Tacrolimus |
| Group 2 | Active comparator | 2 umbilical cord units: First cord blood unit modulated with ProHema Receives: Drug: Fludarabine; Drug: Melphalan; Drug: Antithymocyte Globulin; Drug: Sirolimus; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.
Measured: 2 years
Secondary outcomes (5)
Time to engraftment of umbilical cord blood units
Measured: 2 years
Fractional chimerism of transplanted cord blood units
Measured: 2 years
Rates of acute and chronic GVHD
Measured: 2 years
30- and 100-day treatment related mortality
Measured: 2 years
Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2009 (estimated)
- Study startJan 2011
- Primary completionMay 2013
- Study completionOct 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fate Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Dana-Farber Cancer Institute
- Responsible party
- Sponsor
- Investigators
- Corey Cutler, MD, MPH, FRCP(C), Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00890500
- Sponsor's protocol ID
- FT1050-01
- Status last verified
- Oct 2013
- First submitted
- 28 Apr 2009
- Last updated
- 8 Oct 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Cutler C, Multani P, Robbins D, Kim HT, Le T, Hoggatt J, Pelus LM, Desponts C, Chen YB, Rezner B, Armand P, Koreth J, Glotzbecker B, Ho VT, Alyea E, Isom M, Kao G, Armant M, Silberstein L, Hu P, Soiffer RJ, Scadden DT, Ritz J, Goessling W, North TE, Mendlein J, Ballen K, Zon LI, Antin JH, Shoemaker DD. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood. 2013 Oct 24;122(17):3074-81. doi: 10.1182/blood-2013-05-503177. Epub 2013 Aug 30.
PubMed 23996087
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies.
- ClinicalTrials.gov ID
- NCT00890500
- Acronym / sponsor ID
- FT1050-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Fate TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Dana-Farber Cancer Institute
- Study start
- Jan 2011
- Primary completion
- May 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 29 Apr 2009 (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.
The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.
Detailed description
The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.
-As part of this research study cord units will be modulated in the laboratory with ProHema before it is given to the participant. Two different treatment groups will be tested. Group 1: will have the second cord blood unit modulated with ProHema; Group 2: will have the first cord blood unit modulated with ProHema.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Active comparator | Drug: Fludarabine, Drug: Melphalan, Drug: Antithymocyte Globulin, Drug: Sirolimus, Drug: Tacrolimus2 umbilical cord units: Second cord blood unit modulated with ProHema |
| Group 2 | Active comparator | Drug: Fludarabine, Drug: Melphalan, Drug: Antithymocyte Globulin, Drug: Sirolimus, Drug: Tacrolimus2 umbilical cord units: First cord blood unit modulated with ProHema |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
- Patient must be ineligible for traditional myeloablative transplantation according to treating physician
- Lack of 6/6 or 5/6 HLA-matched related, 8/8 HLA-matched unrelated donor, or unrelated donor not available within a time frame necessary to perform a potentially curative stem cell transplant
- 18-65 years of age
- ECOG Performance Status 0-2
Key exclusion criteria (12 listed)
- The following hematologic malignancies are excluded:
- Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction as measured by gated radionucleotide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension
- Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected hemoglobin
- Renal disease: serum creatinine > 2.0mg/dl
- Hepatic disease: serum bilirubin > 2.0mg/dl (expect in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT > 3 x 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 |
|---|---|---|
| Fludarabine30mg/m2/day IV x 6 days | Drug | Group 1, Group 2 |
| Melphalan100 mg/m2/day IV x 1 day | Drug | Group 1, Group 2 |
| Antithymocyte Globulin1mg/kg/day x 4 days | Drug | Group 1, Group 2 |
| SirolimusGVHD Prophylaxis: Target range 3-12 ng/ml | Drug | Group 1, Group 2 |
| TacrolimusGVHD Prophylaxis: Target range 5-10 ng/ml | Drug | Group 1, Group 2 |
Primary outcome
Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.
Time frame: 2 years
5 secondary outcomes
Time to engraftment of umbilical cord blood units
Time frame: 2 years
Fractional chimerism of transplanted cord blood units
Time frame: 2 years
Rates of acute and chronic GVHD
Time frame: 2 years
30- and 100-day treatment related mortality
Time frame: 2 years
Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2013.
- Derived from this study: PubMed 23996087Cutler C, Multani P, Robbins D, Kim HT, Le T, Hoggatt J, Pelus LM, Desponts C, Chen YB, Rezner B, Armand P, Koreth J, Glotzbecker B, Ho VT, Alyea E, Isom M, Kao G, Armant M, Silberstein L, Hu P, Soiffer RJ, Scadden DT, Ritz J, Goessling W, North TE, Mendlein J, Ballen K, Zon LI, Antin JH, Shoemaker DD. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood. 2013 Oct 24;122(17):3074-81. doi: 10.1182/blood-2013-05-503177. Epub 2013 Aug 30.
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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.
Corey Cutler, MD, MPH, FRCP(C) principal investigator
Dana-Farber Cancer Institute
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 Fludarabine, Melphalan and Antithymocyte Globulin in people with Hematologic Malignancies and Allogeneic Stem Cell Transplantation, 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 5 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: finished, as of the sponsor's last update on 8 Oct 2013. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model. (time frame: 2 years). 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 NCT00890500, which also lists 1 linked publication.
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 Oct 2013.