Acute Myeloid LeukemiaPhase 2NCT04002115
Clofarabine Pre-conditioning Followed by Stem Cell Transplant for Non-remission AML
A Phase 2 study testing Clofarabine, Fludarabine and Busulfan in people with Acute Myeloid Leukemia, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial summary
The Investigators would like to study the incidence of complete remission (CR) at day +30 after Clofarabine followed by haploidentical transplant. The conditioning regimen used is Fludarabine, Busulfan (2 doses) or cyclophosphamide (2 doses) and Total Body Irradiation (TBI) with post transplant cyclophosphamide for… Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Clofarabine, Fludarabine and Busulfan
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Jun 2020to 7 Nov 2022
- Phase
- Phase 2
- 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnostic criteria of AML, induction failure without having achieved remission after at least 2 attempts at induction chemotherapy, or relapsed after any complete remission (CR).
- 18 to 75 years of age.
- Planned or scheduled to receive an allogeneic HSCT from haploidentical related donors, matched and mismatched unrelated donors.
- All organ function testing should be done within 28 days of study registration.
- Performance status: Karnofsky ≥ 70% (Appendix A).
- Cardiac: LVEF ≥ 50% by MUGA or echocardiogram.
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- Pulmonary: FEV1 and FVC ≥ 50% predicted, DLCO (corrected for hemoglobin) ≥ 50% of predicted.
- Renal: Creatinine clearance (CrCl) ≥ 60 mL/min/1.73 m2
- Hepatic: Serum bilirubin ≤1.5 x upper limit of normal (ULN); (AST)/(ALT) ≤ 2.5 x ULN; Alkaline phosphatase ≤ 2.5 x ULN.
- Both men and women need to use an approved method of birth control and/or abstinence due to unknown risks to the fetus.
Who cannot join
- Acute promyelocytic leukemia (APL)
- Known history of non-compliance with medication regimens, scheduled clinic visits, or self-care.
- In the opinion of the investigator, no appropriate caregivers identified.
- HIV1 (Human Immunodeficiency Virus-1) or HIV2 positive
- Active Hepatitis B and Hepatitis C orepatitis positive serology including HBsAg, hepatitis B core antibody, and hepatitis C antibody. Hepatitis B surface antibody positive due to vaccination or natural immunity are permitted.
- In the opinion of the physician investigator, uncontrolled medical or psychiatric disorders.
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- Uncontrolled infections requiring treatment within 14 days of registration.
- Active central nervous system (CNS) leukemia.
- Cord blood transplant excluded.
- Prior allogeneic HSCT within last 6 months.
- Patients with >= grade 2 acute GVHD.
- Patients with >=moderate chronic GVHD.
- Pregnant or Breastfeeding. Women of child bearing potential (WCBP) are required to have a negative serum or urine pregnancy test prior to initiation of conditioning regimen.
- Haploidentical related donors who are positive for DSA ≥ 5000 MFI by solid phase microarray method (Luminex).
- Any patient with steroid dose more than 10 mg/day within a week of registration .
- Autoimmune disorder requiring any active immunosuppression therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
Penn State Cancer Institute
Hershey, Pennsylvania, 17033
About this study
Official title: Clofarabine Pre-conditioning Followed by Hematopoietic Stem Cell Transplant With Post-Transplant Cyclophosphamide for Non-remission Acute Myeloid Leukemia
The Investigators would like to study the incidence of complete remission (CR) at day +30 after Clofarabine followed by haploidentical transplant. The conditioning regimen used is Fludarabine, Busulfan (2 doses) or cyclophosphamide (2 doses) and Total Body Irradiation (TBI) with post transplant cyclophosphamide for patients with Acute Myeloid Leukemia (AML) who are not in remission prior to considering allogeneic transplant with haploidentical donors.
Read the detailed description
Approximately 30-40% of patients with acute myeloid leukemia (AML) experience induction failures. In these patients who do not achieve remission with two cycles of standard induction therapies, the probability of achieving remission with subsequent inductions is limited. Hematopoietic stem cell transplantation (HSCT) is the only curative option for these patients, but high relapse rate and transplant-related mortality often preclude them to proceed to transplant. Thus, AML not in remission at time of HSCT remains a huge unmet need in current HSCT practice, particularly if the patient does not have a Human Leukocyte Antigen (HLA)-matched donor identified by the time of two induction failures.
Salvage chemotherapy with clofarabine appears to be another promising option in relapsed and refractory AML. Clofarabine is a second-generation purine nucleoside analog with substantial single-agent activity in adult patients with AML. It is an effective immunosuppressive agent and several trials have shown the feasibility of conditioning with clofarabine-based regimen.
In the past, a conditioning regimen of clofarabine with busulfan (4 doses) has been successfully used prior to allogeneic stem cell transplantation for non-remission AML with day +30 complete remission rates were 90-100%. However, these patients were transplanted with HLA matched donors. This study will examine those patients undergoing transplantation from haploidentical related donor or matched and mismatched unrelated donors.
Achieving a long-term remission is clearly the goal of AML treatment. The investigators would like to propose a protocol for non-remission AML and expand the patient population to older than 55 years of age as well as those who relapsed after initial allogeneic transplant to improve enrolling patients in the near future. The investigators have many patients achieving remission but for those without remission, clofarabine preconditioning may be a reliable protocol to bring these patients into the early complete remission.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Clofarabine, Fludarabine, Busulfan, Total Body Irradiation (TBI), Cyclophosphamide
- Keywords
- Clofarabine, Haploidentical stem cell transplantation, matched and mismatched unrelated donors, Non-remission AML
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofarabineDrug
Also called Clolar
Clofarabine to be administered pre-stem cell transplant infusion ("Day 0") once a day for 5 days total.
FludarabineDrug
Also called Fludara
Fludarabine will be administered once a day for 4 days as part of the transplant conditioning regimen.
BusulfanDrug
Also called Busulfex
Busulfan will be administered once a day for 2 days as part of the transplant conditioning regimen.
Total Body Irradiation (TBI)Procedure or surgery
Also called TBI
TBI will be administered at a dose of 200cGys on Day -1 prior to transplant
CyclophosphamideDrug
Also called Cytoxan
Cyclophosphamide will be given once a day for 2 days after the transplant infusion.
Granulocyte Colony-Stimulating FactorDrug
Also called Filgrastim G-CSF
G-CSF will be administered to subjects starting on Day +5 and will continue as clinically indicated
TacrolimusDrug
Also called Prograf
Tacrolimus will be administered to subjects starting on Day +5 and will continue as clinically indicated
CellceptDrug
Also called Mycophenolate Mofetil (MMF)
Mycophenolate Mofetil will be administered to subjects starting on Day +5 and will continue as clinically indicated
| Study group | Role | What they receive |
|---|---|---|
| Clofarabine 30 mg/m^2 | Experimental | Day -14 through Day -10 Clofarabine 30 mg/m^2, Day - 9 Day of rest, Day - 8 Day of rest, Day - 7 Day of rest, Day - 6 Fludarabine 40 mg/m^2 IV and Busulfan 3.2 mg/kg IV (Regimen A, Fludarabine 24 mg/m^2 IV and Cyclophosphamide 14.5 mg/kg IV for Regimen B), Day - 5 Fludarabine 40 mg/m^2 IV and Busulfan 3.2 mg/kg IV (Regimen A, Fludarabine 24 mg/m^2 IV and Cyclophosphamide 14.5 mg/kg IV for Regimen B), Day - 4 Fludarabine 40 mg/m^2 IV(Regimen A, Fludarabine 24 mg/m^2 IV for Regimen B), Day - 3 Fludarabine 40 mg/m^2 IV(Regimen A, Fludarabine 24 mg/m^2 IV for Regimen B), Day - 2 Day of Rest, Day -1 Total Body Irradiation 200 cGys, Day 0 stem cell transplant infusion, Day +1 Day of rest, Day +2 Day of rest, Day +3 Cyclophosphamide 50 mg/kg IV, Day +4 Cyclophosphamide 50 mg/kg IV, Day +5 Start G-CSF, Tacrolimus, and MMF. Receives: Drug: Clofarabine; Drug: Fludarabine; Drug: Busulfan; Procedure: Total Body Irradiation (TBI); Drug: Cyclophosphamide; Drug: Granulocyte Colony-Stimulating Factor; Drug: Tacrolimus; Drug: Cellcept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission (CR) Rate at Day 30 Post HSCT
The CR rate at 30 days (Day +30) post stem cell transplant infusion
Measured: 30 days
Secondary outcomes (6)
Non-relapse Related Mortality
Determine the rate of non-relapse related mortality at 100 days post transplant (Day +100)
Measured: 100 days
Neutrophil Engraftment
Rates of engraftment, defined as the first day of Absolute Neutrophil Count (ANC) greater than 500 for the first of three consecutive days
Measured: 1 year
Rate of Acute Graft-versus-host Disease (GVHD)
The rate of any grade (1-4) of acute GvHD as measured from day of transplantation to Day +100 using the Glucksberg criteria.
Measured: 100 days
Severity of Acute Graft-versus-host Disease (GVHD)
The highest grade (1-4) of acute GvHD experienced by participants as measured from day of transplantation to Day +100 using the Glucksberg criteria
Measured: 100 days
Rate of Chronic GVHD
The rate of any grade (1-4) of Chronic GvHD as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria.
Measured: 1 year
Severity of Chronic GVHD
The highest overall grade (1-4) of chronic GvHD experienced by participants as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2019
- Study start3 Jun 2020
- Primary completion30 Aug 2022
- Study completion7 Nov 2022
Why it stopped: terminated due to low accrual
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 Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Dose de-escalation: Enrollment will begin at 30 mg/m2 to analyze any grade 4-5 organ toxicities as defined in Section 12.2. If the trial is stopped due to excessive toxicities, then dose de-escalation to 20 mg/m2 will occur for the next cohort of participants. For each participant, the observation period is from start of treatment through post-transplant day +30. Adverse effects will be continuously monitored as patients are enrolled. The trial will be stopped when the toxicity rate exceeds 20% with a posterior probability of 80% and a margin of no more than 5%. This leads to the following stopping rule: the trial will be stopped if 2 of the first 3 subjects experience grade 4-5 organ toxicity, or 3 out of 6, 4 out 9, 5 out of 12, 6 out of 16, or 7 out 19.
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Seema Naik, MD, Milton S. Hershey Medical Center
- Investigators
- Seema Naik, MD, Principal investigator, Penn State Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04002115
- Sponsor's protocol ID
- 18-011
- Status last verified
- Sep 2023
- First submitted
- 24 Jun 2019
- Last updated
- 14 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: At this time there is no plan to share IPD with other researchers outside of Penn State University
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clofarabine Pre-conditioning Followed by Hematopoietic Stem Cell Transplant With Post-Transplant Cyclophosphamide for Non-remission Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT04002115
- Acronym / sponsor ID
- 18-011
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Clofarabine, Haploidentical stem cell transplantation, matched and mismatched unrelated donors, Non-remission AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: terminated due to low accrual
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- 3 Jun 2020 (actual)
- Primary completion
- 30 Aug 2022 (actual)
- Study completion
- 7 Nov 2022 (actual)
- First posted
- 28 Jun 2019 (actual)
- Results
- Posted 14 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Investigators would like to study the incidence of complete remission (CR) at day +30 after Clofarabine followed by haploidentical transplant. The conditioning regimen used is Fludarabine, Busulfan (2 doses) or cyclophosphamide (2 doses) and Total Body Irradiation (TBI) with post transplant cyclophosphamide for patients with Acute Myeloid Leukemia (AML) who are not in remission prior to considering allogeneic transplant with haploidentical donors.
Detailed description
Approximately 30-40% of patients with acute myeloid leukemia (AML) experience induction failures. In these patients who do not achieve remission with two cycles of standard induction therapies, the probability of achieving remission with subsequent inductions is limited. Hematopoietic stem cell transplantation (HSCT) is the only curative option for these patients, but high relapse rate and transplant-related mortality often preclude them to proceed to transplant. Thus, AML not in remission at time of HSCT remains a huge unmet need in current HSCT practice, particularly if the patient does not have a Human Leukocyte Antigen (HLA)-matched donor identified by the time of two induction failures.
Salvage chemotherapy with clofarabine appears to be another promising option in relapsed and refractory AML. Clofarabine is a second-generation purine nucleoside analog with substantial single-agent activity in adult patients with AML. It is an effective immunosuppressive agent and several trials have shown the feasibility of conditioning with clofarabine-based regimen.
In the past, a conditioning regimen of clofarabine with busulfan (4 doses) has been successfully used prior to allogeneic stem cell transplantation for non-remission AML with day +30 complete remission rates were 90-100%. However, these patients were transplanted with HLA matched donors. This study will examine those patients undergoing transplantation from haploidentical related donor or matched and mismatched unrelated donors.
Achieving a long-term remission is clearly the goal of AML treatment. The investigators would like to propose a protocol for non-remission AML and expand the patient population to older than 55 years of age as well as those who relapsed after initial allogeneic transplant to improve enrolling patients in the near future. The investigators have many patients achieving remission but for those without remission, clofarabine preconditioning may be a reliable protocol to bring these patients into the early complete remission.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Dose de-escalation: Enrollment will begin at 30 mg/m2 to analyze any grade 4-5 organ toxicities as defined in Section 12.2. If the trial is stopped due to excessive toxicities, then dose de-escalation to 20 mg/m2 will occur for the next cohort of participants. For each participant, the observation period is from start of treatment through post-transplant day +30. Adverse effects will be continuously monitored as patients are enrolled. The trial will be stopped when the toxicity rate exceeds 20% with a posterior probability of 80% and a margin of no more than 5%. This leads to the following stopping rule: the trial will be stopped if 2 of the first 3 subjects experience grade 4-5 organ toxicity, or 3 out of 6, 4 out 9, 5 out of 12, 6 out of 16, or 7 out 19.
| Arm | Type | Receives |
|---|---|---|
| Clofarabine 30 mg/m^2 | Experimental | Drug: Clofarabine, Drug: Fludarabine, Drug: Busulfan, Procedure: Total Body Irradiation (TBI), Drug: Cyclophosphamide, Drug: Granulocyte Colony-Stimulating Factor, Drug: Tacrolimus, Drug: CellceptDay -14 through Day -10 Clofarabine 30 mg/m^2, Day - 9 Day of rest, Day - 8 Day of rest, Day - 7 Day of rest, Day - 6 Fludarabine 40 mg/m^2 IV and Busulfan 3.2 mg/kg IV (Regimen A, Fludarabine 24 mg/m^2 IV and Cyclophosphamide 14.5 mg/kg IV for Regimen B), Day - 5 Fludarabine 40 mg/m^2 IV and Busulfan 3.2 mg/kg IV (Regimen A, Fludarabine 24 mg/m^2 IV and Cyclophosphamide 14.5 mg/kg IV for Regimen B), Day - 4 Fludarabine 40 mg/m^2 IV(Regimen A, Fludarabine 24 mg/m^2 IV for Regimen B), Day - 3 Fludarabine 40 mg/m^2 IV(Regimen A, Fludarabine 24 mg/m^2 IV for Regimen B), Day - 2 Day of Rest, Day -1 Total Body Irradiation 200 cGys, Day 0 stem cell transplant infusion, Day +1 Day of rest, Day +2 Day of rest, Day +3 Cyclophosphamide 50 mg/kg IV, Day +4 Cyclophosphamide 50 mg/kg IV, Day +5 Start G-CSF, Tacrolimus, and MMF. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Diagnostic criteria of AML, induction failure without having achieved remission after at least 2 attempts at induction chemotherapy, or relapsed after any complete remission (CR).
- 18 to 75 years of age.
- Planned or scheduled to receive an allogeneic HSCT from haploidentical related donors, matched and mismatched unrelated donors.
- All organ function testing should be done within 28 days of study registration.
- Both men and women need to use an approved method of birth control and/or abstinence due to unknown risks to the fetus.
Key exclusion criteria (16 listed)
- Acute promyelocytic leukemia (APL)
- Known history of non-compliance with medication regimens, scheduled clinic visits, or self-care.
- In the opinion of the investigator, no appropriate caregivers identified.
- HIV1 (Human Immunodeficiency Virus-1) or HIV2 positive
- Active Hepatitis B and Hepatitis C orepatitis positive serology including HBsAg, hepatitis B core antibody, and hepatitis C antibody. Hepatitis B surface antibody positive due to vaccination or natural immunity are permitted.
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 |
|---|---|---|
| ClofarabineAlso: ClolarClofarabine to be administered pre-stem cell transplant infusion ("Day 0") once a day for 5 days total. | Drug | Clofarabine 30 mg/m^2 |
| FludarabineAlso: FludaraFludarabine will be administered once a day for 4 days as part of the transplant conditioning regimen. | Drug | Clofarabine 30 mg/m^2 |
| BusulfanAlso: BusulfexBusulfan will be administered once a day for 2 days as part of the transplant conditioning regimen. | Drug | Clofarabine 30 mg/m^2 |
| Total Body Irradiation (TBI)Also: TBITBI will be administered at a dose of 200cGys on Day -1 prior to transplant | Procedure or surgery | Clofarabine 30 mg/m^2 |
| CyclophosphamideAlso: CytoxanCyclophosphamide will be given once a day for 2 days after the transplant infusion. | Drug | Clofarabine 30 mg/m^2 |
| Granulocyte Colony-Stimulating FactorAlso: Filgrastim G-CSFG-CSF will be administered to subjects starting on Day +5 and will continue as clinically indicated | Drug | Clofarabine 30 mg/m^2 |
| TacrolimusAlso: PrografTacrolimus will be administered to subjects starting on Day +5 and will continue as clinically indicated | Drug | Clofarabine 30 mg/m^2 |
| CellceptAlso: Mycophenolate Mofetil (MMF)Mycophenolate Mofetil will be administered to subjects starting on Day +5 and will continue as clinically indicated | Drug | Clofarabine 30 mg/m^2 |
Primary outcome
Complete Remission (CR) Rate at Day 30 Post HSCT
Time frame: 30 days
The CR rate at 30 days (Day +30) post stem cell transplant infusion
6 secondary outcomes
Non-relapse Related Mortality
Time frame: 100 days
Neutrophil Engraftment
Time frame: 1 year
Rate of Acute Graft-versus-host Disease (GVHD)
Time frame: 100 days
Severity of Acute Graft-versus-host Disease (GVHD)
Time frame: 100 days
Rate of Chronic GVHD
Time frame: 1 year
Severity of Chronic GVHD
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Penn State Cancer InstituteLink provided by the sponsor.
- Individual participant dataSharing planned: No. At this time there is no plan to share IPD with other researchers outside of Penn State University
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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.
Seema Naik, MD principal investigator
Penn State 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 2 study testing Clofarabine, Fludarabine and Busulfan in people with Acute Myeloid Leukemia, run by Milton S. Hershey Medical Center 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 10 inclusion and 16 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 14 Sep 2023. 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 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?
Yes. Results are posted in the registry (first posted 14 Sep 2023). 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?
Complete Remission (CR) Rate at Day 30 Post HSCT (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 14 Sep 2023.