Hematologic MalignanciesPhase 1NCT00677859
Safety Study of MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia
A Phase 1 study testing MultiStem® in people with Hematologic Malignancies, run by Healios K.K. at 6 sites in 2 countries.
Trial summary
The purpose of this study is to determine if MultiStem® can safely be given to patients with acute leukemia, chronic myeloid leukemia, or myelodysplasia after they have received hematopoietic stem cell transplantation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- MultiStem®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 6 sitesin 2 countries
- Study dates
- Jul 2008to Nov 2011
- 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 of either sex aged 18-65 years of age
- Diagnosis of acute myeloid or lymphoblastic leukemia (second or subsequent remission, if not in remission, then <20% bone marrow blasts), chronic myelogenous leukemia resistant to or intolerant of tyrosine kinase inhibitor therapy (accelerated phase, first chronic phase with TKI resistance, or second chronic phase), or myelodysplastic syndrome (intermediate/high or high risk by International Prognostic Scoring System (IPSS), lower risk by IPSS with patient having progressed after prior therapy. Complete remission is defined as the absence of blasts in the peripheral circulation at the time of enrollment and <5% blasts in the marrow within 28 days of enrollment.
- Life expectancy of at least 100 days
- Patients scheduled for allogeneic bone marrow transplant or peripheral blood stem cell transplant (PBST) procedure
- Family-related or unrelated donors
- HLA matching should either be matched related or matched unrelated donors, 6/6 match or 5/6 single allelic mismatch, with provision that the DRB1 is molecularly matched
- Performance status (ECOG ≤2)
- Signed informed consent
Who cannot join
- Active infection
- Known allergies to bovine or porcine products
- Renal function: Serum creatinine >2 mg/dL or creatinine clearance ≤50 mL/min
- Hepatic function: Screening ALT or AST ≥3x than the upper limit of normal for the laboratory OR total bilirubin ≥2.0 mg/dL (Exception: acceptable if patient is identified with pre-existing condition e.g., Gilbert's disease that will contribute to baseline elevations of bilirubin)
- Pulmonary function: FEV1, FVC, DLCO ≤50% predicted
- Cardiac function: left ventricular ejection fraction ≤50%
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- Patient received an investigational agent within 30 days prior to transplant
- The patient is pregnant, has a positive serum BhCG, or is lactating
- Patient on corticosteroids at a dose >0.25 mg/kg/day
- Planned non-myeloablative transplant
- Planned cord blood transplant
- Prior allogeneic myeloablative HSCT
- HIV seropositive, HTLV seropositive, hepatitis B or C seropositive, varicella virus active infection, or syphilis active infection
- Other serious medical or psychiatric illness that, in the investigator's opinion, would not permit the patient to be managed according to the protocol
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic Hospital
Phoenix, Arizona, 85054
Oregon State University Medical Center
Portland, Oregon, 97239
Texas Transplant Institute
San Antonio, Texas, 78229
About this study
Official title: A Phase I, Multicenter, Dose-Escalation Trial Evaluating Maximum-Tolerated Dose of Single and Repeated Administration of Allogeneic MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia
The purpose of this study is to determine if MultiStem® can safely be given to patients with acute leukemia, chronic myeloid leukemia, or myelodysplasia after they have received hematopoietic stem cell transplantation.
Read the detailed description
Graft-vs.-Host Disease (GVHD) is one of the major limitations of allogeneic hematopoietic stem cell transplants (HSCT). This complication is major cause of morbidity and mortality and is thought to be initiated by activation of donor T-cells through recognition of "foreign" cells resident in the transplant recipient. Acute GVHD is associated with damage to the liver, skin, gastrointestinal tract and mucosa. Moderate to severe GVHD Grades II-IV occurs in 30-50% of matched related HSCTs and 50-70% of unrelated donor recipients. Severe GHVD requires intense immunosuppression involving steroids and additional agents to get it under control, and patients may develop severe infections as a result of such immunosuppression. An agent or cell therapy that could reduce the incidence and/or severity of GVHD without increasing relapse or infectious risk in HSCT patients would provide substantial benefits.
- Conditions
- Hematologic Malignancies
- Being tested
- MultiStem®
- Keywords
- Leukemia, Graft vs. Host Disease, Hematopoietic Stem Cell Transplant, Bone Marrow Transplant, Myelodysplasia
- Medical subject headings
- Hematologic Neoplasms, Leukemia, Graft vs Host Disease, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MultiStem®Biological
Patients will receive a single IV infusion of MultiStem® 2 days after HSCT.
MultiStem®Biological
Patients will receive either 3 weekly IV infusions or 5 weekly infusions of MultiStem®
| Study group | Role | What they receive |
|---|---|---|
| Single dose Arm | Experimental | There will be six cohorts of three patients each. Three escalating doses of MultiStem will be evaluated. Receives: Biological: MultiStem® |
| Repeat Dose Arm | Experimental | There will be six cohorts of three patients each. Four dosing regimens will be evaluated,varying doses at three times weekly or five times weekly. Receives: Biological: MultiStem® |
What is being measured
The primary outcome is the main question the study is designed to answer.
maximum tolerated dose
Measured: 30 days
Secondary outcomes (1)
incidence of grade III/IV GVHD
Measured: 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2008 (estimated)
- Study startJul 2008
- Primary completionOct 2011
- Study completionNov 2011
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 1
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Healios K.K.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cato Research
- Responsible party
- Sponsor
- Investigators
- Richard Maziarz, MD, Principal investigator, Oregon Health and Science UniversitySteven Devine, MD, Principal investigator, Ohio State UniversityHillard Lazarus, MD, Principal investigator, Case Western Reserve University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00677859
- Sponsor's protocol ID
- GVHD-2007-001
- Status last verified
- Jan 2012
- First submitted
- 14 May 2008
- Last updated
- 5 Jan 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multicenter, Dose-Escalation Trial Evaluating Maximum-Tolerated Dose of Single and Repeated Administration of Allogeneic MultiStem® in Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplasia
- ClinicalTrials.gov ID
- NCT00677859
- Acronym / sponsor ID
- GVHD-2007-001
- Conditions
- Hematologic Malignancies
- Keywords
- Leukemia, Graft vs. Host Disease, Hematopoietic Stem Cell Transplant, Bone Marrow Transplant, Myelodysplasia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Healios K.K.Industry (pharma, biotech, devices)
- Collaborators
- Cato Research
- Study start
- Jul 2008
- Primary completion
- Oct 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 15 May 2008 (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 study is to determine if MultiStem® can safely be given to patients with acute leukemia, chronic myeloid leukemia, or myelodysplasia after they have received hematopoietic stem cell transplantation.
Detailed description
Graft-vs.-Host Disease (GVHD) is one of the major limitations of allogeneic hematopoietic stem cell transplants (HSCT). This complication is major cause of morbidity and mortality and is thought to be initiated by activation of donor T-cells through recognition of "foreign" cells resident in the transplant recipient. Acute GVHD is associated with damage to the liver, skin, gastrointestinal tract and mucosa. Moderate to severe GVHD Grades II-IV occurs in 30-50% of matched related HSCTs and 50-70% of unrelated donor recipients. Severe GHVD requires intense immunosuppression involving steroids and additional agents to get it under control, and patients may develop severe infections as a result of such immunosuppression. An agent or cell therapy that could reduce the incidence and/or severity of GVHD without increasing relapse or infectious risk in HSCT patients would provide substantial benefits.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Single dose Arm | Experimental | Biological: MultiStem®There will be six cohorts of three patients each. Three escalating doses of MultiStem will be evaluated. |
| Repeat Dose Arm | Experimental | Biological: MultiStem®There will be six cohorts of three patients each. Four dosing regimens will be evaluated,varying doses at three times weekly or five times weekly. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patients of either sex aged 18-65 years of age
- Diagnosis of acute myeloid or lymphoblastic leukemia (second or subsequent remission, if not in remission, then <20% bone marrow blasts), chronic myelogenous leukemia resistant to or intolerant of tyrosine kinase inhibitor therapy (accelerated phase, first chronic phase with TKI resistance, or second chronic phase), or myelodysplastic syndrome (intermediate/high or high risk by International Prognostic Scoring System (IPSS), lower risk by IPSS with patient having progressed after prior therapy. Complete remission is defined as the absence of blasts in the peripheral circulation at the time of enrollment and <5% blasts in the marrow within 28 days of enrollment.
- Life expectancy of at least 100 days
- Patients scheduled for allogeneic bone marrow transplant or peripheral blood stem cell transplant (PBST) procedure
- Family-related or unrelated donors
Key exclusion criteria (14 listed)
- Active infection
- Known allergies to bovine or porcine products
- Renal function: Serum creatinine >2 mg/dL or creatinine clearance ≤50 mL/min
- Hepatic function: Screening ALT or AST ≥3x than the upper limit of normal for the laboratory OR total bilirubin ≥2.0 mg/dL (Exception: acceptable if patient is identified with pre-existing condition e.g., Gilbert's disease that will contribute to baseline elevations of bilirubin)
- Pulmonary function: FEV1, FVC, DLCO ≤50% predicted
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 |
|---|---|---|
| MultiStem®Patients will receive a single IV infusion of MultiStem® 2 days after HSCT. | Biological | Single dose Arm |
| MultiStem®Patients will receive either 3 weekly IV infusions or 5 weekly infusions of MultiStem® | Biological | Repeat Dose Arm |
Primary outcome
maximum tolerated dose
Time frame: 30 days
1 secondary outcome
incidence of grade III/IV GVHD
Time frame: 100 days
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 5 Jan 2012.
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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.
Richard Maziarz, MD principal investigator
Oregon Health and Science University
Steven Devine, MD principal investigator
Ohio State University
Hillard Lazarus, MD principal investigator
Case Western Reserve University
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MultiStem® in people with Hematologic Malignancies, run by Healios K.K. at 6 sites in 2 countries.
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 8 inclusion and 14 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 5 Jan 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Belgium. 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?
maximum tolerated dose (time frame: 30 days). 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 5 Jan 2012.