High Risk Hematologic MalignanciesPhase 1NCT01818479
Phase I/II Study of Treg/Tcon Addback to Partially Matched Related Donor Stem Cells With Myeloablative Conditioning and Post-transplant Cyclophosphamide for High Risk Hematologic Malignancies
A Phase 1 study testing stem cell transplant in people with High Risk Hematologic Malignancies, run by University of Utah at 1 site in United States.
Trial summary
Open label, dose finding trial to assess the efficacy of Treg/Tcon addback to partially matched related donor stem cells. The maximum tolerated dose will be established using 3 subjects per dose level, with an expansion cohort at the maximum tolerated dose. Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- High Risk Hematologic Malignancies
- What is being tested?
- stem cell transplant
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jul 2013to 1 Apr 2019
- 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 Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Age 0-70 years 2. Karnofsky or Lansky Performance status >70% 3. High risk hematologic malignancy 4. Acute myeloid leukemia (AML) with one or more of the following criteria: 4a.Poor risk cytogenetics, including -5, 5q-, -7, 7q-, t(9;22); complex cytogenetics (>3 abnormalities); or normal cytogenetics with Flt3 internal tandem duplication (ITD), in first or subsequent complete remission (CR).
- 4b. Relapsed or primary refractory AML with <10% blasts in the peripheral blood.
- 4c. Subjects in first complete remission (CR1) who required two cycles of induction to achieve remission may be included at the discretion of the treating physician.
- 4d. Standard risk or intermediate risk cytogenetics in second or subsequent CR (enrolled at the discretion of the treating physician).
- 5. Acute lymphoblastic leukemia (ALL) with one of the following criteria: 5a. Second or subsequent CR 5b. Any partial remission (PR) (no circulating blasts) 5c. High-risk ALL in first CR including (Ph+, t(4:11), complex karyotype, hypodiploidy (<44 chromosomes), or positive minimal residual disease (MRD) after induction 6. Myelodysplasia, intermediate -2 (score 1.5-2.0) or high risk (score >2.5) by the International Prognostic Score System.
- 7. Myeloproliferative Disorders (include chronic myelomonocytic leukemia (CMML), agnogenic myeloid metaplasia (AMM) or Idiopathic Myelofibrosis, and JMML) with excess blasts (>5%) 8. Chronic myeloid leukemia (CML) with one of the following criteria: 8a. Second or subsequent chronic phase 8b. Accelerated phase 8c. blast crisis 9. Non-Hodgkin's lymphoma (NHL) meeting one of the following criteria: 9a. Relapse after autologous stem cell transplantation with evidence of responsive disease.
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- 9b. Subject with chemosensitive relapse who have no option for autologous stem cell transplantation due to blood or marrow involvement or failure to mobilize autologous stem cells or are not considered eligible for autologous transplant by their treating physician.
- 9c. Hodgkin's Lymphoma: relapse after autologous hematopoietic cell transplantation (HCT), chemo-refractory disease 9d. Multiple myeloma: per National Comprehensive Cancer Network (NCCN) guidelines. Updated annually at: www.nccn.org 10. No suitable human leukocyte antigen (HLA)-identical sibling donor. 11. No identified 8/8 (based upon A, B, C, DR beta 1 (DRB1) loci) allele matched unrelated donor, or unable to wait sufficient time to procure a 8/8 allele matched unrelated donor 12. Available HLA 3-5/6 matched genotypically haploidentical partially matched related donor 13. Female subjects must be surgically sterile, postmenopausal (minimum 1 year without menses), or agree to use approved form of contraception from the time of signing the informed consent form through Day +100. Male subjects must also agree to use an approved form of birth control for either themselves or their partner, as appropriate, from the time of signing the informed consent form through Day +100.
- 14. Able to provide informed consent and have signed an approved consent form that conforms to federal and institutional guidelines.
Who cannot join
- Available HLA identical matched sibling donor (unless having failed a prior allogeneic transplant from an HLA identical matched sibling)
- Recipient HLA antibodies against donor HLA
- Any of the following organ dysfunctions:
- Cardiac- left ventricular ejection fraction <40%, symptomatic coronary artery disease, or uncontrolled arrhythmias
- Pulmonary- forced expiratory volume at one second (FEV1) or diffusion capacity of lung for carbon monoxide (DLco)<40% or need for use of supplemental oxygen
- Renal- calculated or measured glomerular filtration rate (GFR) <30 ml/min, dialysis requirement, or prior renal transplant
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- Hepatic- bilirubin > 2.0, alanine aminotransferase (ALT) > 2.5 X upper limit of normal (ULN), cirrhosis
- Subjects with active or uncontrolled bacterial, viral, or fungal infections requiring systemic therapy.
- Subjects who have tested positive for HIV.
- Pregnant women, nursing mothers or women of child-bearing potential who are unwilling to use medically accepted methods of contraception.
Trial locations and contacts
1 site in 1 country.
United States1 site
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
About this study
Open label, dose finding trial to assess the efficacy of Treg/Tcon addback to partially matched related donor stem cells. The maximum tolerated dose will be established using 3 subjects per dose level, with an expansion cohort at the maximum tolerated dose.
- Conditions
- High Risk Hematologic Malignancies
- Being tested
- stem cell transplant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
stem cell transplantBiological
Open label, dose finding trial with two treatment regimens, Busulfan and Total Body Irradiation (TBI) with four dose escalation cohorts, to assess the efficacy of Treg/Tcon addback to partially matched related donor stem cells.
Stem cell transplantProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| All participants | Experimental | Not described Receives: Biological: stem cell transplant; Procedure: Stem cell transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of acute GVHD
Rate of acute Graft Versus Host Disease (GVHD) grade III-IV at Day +100 post transplant
Measured: 36 months
Secondary outcomes (2)
Rate of Engraftment
Day +28 and +100 neutrophil engraftment
Measured: 36 months
Survival at Day 100
Day +100 and one year survival Day +100 and one year transplant related mortality Day +100 and one year relapse rates
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2013 (estimated)
- Study start12 Jul 2013
- Primary completion1 Apr 2019
- Study completion1 Apr 2019
Why it stopped: Trial discontinued prior to starting Phase II portion due to low accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High Risk Hematologic MalignanciesStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- 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
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Boyer, MD, Principal investigator, Huntsman Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01818479
- Sponsor's protocol ID
- HCI61077
- Status last verified
- Aug 2020
- First submitted
- 14 Mar 2013
- Last updated
- 11 Aug 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Treg/Tcon Addback to Partially Matched Related Donor Stem Cells With Myeloablative Conditioning and Post-transplant Cyclophosphamide for High Risk Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT01818479
- Acronym / sponsor ID
- HCI61077
- Conditions
- High Risk Hematologic Malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Trial discontinued prior to starting Phase II portion due to low accrual.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Study start
- 12 Jul 2013 (actual)
- Primary completion
- 1 Apr 2019 (actual)
- Study completion
- 1 Apr 2019 (actual)
- First posted
- 26 Mar 2013 (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.
Open label, dose finding trial to assess the efficacy of Treg/Tcon addback to partially matched related donor stem cells. The maximum tolerated dose will be established using 3 subjects per dose level, with an expansion cohort at the maximum tolerated dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All participants | Experimental | Biological: stem cell transplant, Procedure: Stem cell transplant |
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 1. Age 0-70 years 2. Karnofsky or Lansky Performance status >70% 3. High risk hematologic malignancy 4. Acute myeloid leukemia (AML) with one or more of the following criteria: 4a.Poor risk cytogenetics, including -5, 5q-, -7, 7q-, t(9;22); complex cytogenetics (>3 abnormalities); or normal cytogenetics with Flt3 internal tandem duplication (ITD), in first or subsequent complete remission (CR).
- 4b. Relapsed or primary refractory AML with <10% blasts in the peripheral blood.
- 4c. Subjects in first complete remission (CR1) who required two cycles of induction to achieve remission may be included at the discretion of the treating physician.
- 4d. Standard risk or intermediate risk cytogenetics in second or subsequent CR (enrolled at the discretion of the treating physician).
- 5. Acute lymphoblastic leukemia (ALL) with one of the following criteria: 5a. Second or subsequent CR 5b. Any partial remission (PR) (no circulating blasts) 5c. High-risk ALL in first CR including (Ph+, t(4:11), complex karyotype, hypodiploidy (<44 chromosomes), or positive minimal residual disease (MRD) after induction 6. Myelodysplasia, intermediate -2 (score 1.5-2.0) or high risk (score >2.5) by the International Prognostic Score System.
Key exclusion criteria (10 listed)
- Available HLA identical matched sibling donor (unless having failed a prior allogeneic transplant from an HLA identical matched sibling)
- Recipient HLA antibodies against donor HLA
- Any of the following organ dysfunctions:
- Subjects with active or uncontrolled bacterial, viral, or fungal infections requiring systemic therapy.
- Subjects who have tested positive for HIV.
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 |
|---|---|---|
| stem cell transplantOpen label, dose finding trial with two treatment regimens, Busulfan and Total Body Irradiation (TBI) with four dose escalation cohorts, to assess the efficacy of Treg/Tcon addback to partially matched related donor stem cells. | Biological | All participants |
| Stem cell transplant | Procedure or surgery | All participants |
Primary outcome
Rate of acute GVHD
Time frame: 36 months
Rate of acute Graft Versus Host Disease (GVHD) grade III-IV at Day +100 post transplant
2 secondary outcomes
Rate of Engraftment
Time frame: 36 months
Survival at Day 100
Time frame: 36 months
Results and safety
The study was stopped early (reason given: Trial discontinued prior to starting Phase II portion due to low accrual.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2020.
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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.
Michael Boyer, MD principal investigator
Huntsman Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing stem cell transplant in people with High Risk Hematologic Malignancies, run by University of Utah at 1 site 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 9 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 11 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 was stopped early (reason given: Trial discontinued prior to starting Phase II portion due to low accrual.). 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 acute GVHD (time frame: 36 months). 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 11 Aug 2020.