Myelodysplastic SyndromesPhase 1NCT03393611
CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome
A Phase 1 study testing CPX-351, Fludarabine and Melphalan in people with Myelodysplastic Syndromes and Leukemia, Myeloid, Acute, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
This pilot study is designed to evaluate outcomes with the combination of CPX-351 salvage therapy and haplo-cord graft stem cell transplantation for subjects with relapsed or refractory AML or myelodysplastic syndrome. Read more about this study
- Who is it for?
- Ages 18 to 89 years
- Conditions the study is for
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia, Relapsed Adult Acute Myeloid
- What is being tested?
- CPX-351, Fludarabine and Melphalan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Nov 2012to 18 Nov 2021
- 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 89 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject must have refractory or relapsed Acute Myeloid Leukemia (AML) according to previously established criteria:
- Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy
- First relapse
- Relapse refractory to salvage chemotherapy
- Second or subsequent relapse
- Subjects with Myelodysplastic Syndrome (MDS):
Show all 15Show fewer
- Either Refractory Anemia with Excess Blasts I or Refractory Anemia with Excess Blasts II (RAEB I or RAEB II)
- Karnofsky performance status ≥ 70
- Willing to participate as a research subject and sign an informed consent form
- Adequate physical function measured by:
- Cardiac: asymptomatic, or if symptomatic then Left Ventricular Ejection Fraction (LVEF) at rest must be ≥ 45% and must improve with exercise
- Hepatic: ≤3 x upper limit of normal (ULN) alanine aminotransferase (ALT) and ≤ 1.5 total serum bilirubin, unless liver is involved with the disease or there is congenital benign hyperbilirubinemia
- Renal: serum creatinine within normal range, or if serum creatinine is outside the normal range, then calculated creatinine clearance ≥ 60 ml/min
- Pulmonary: asymptomatic, or if symptomatic, diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 45% of predicted (corrected for hemoglobin)
- If subject has prior malignancy, must be without any evidence of disease of that prior malignancy for at least 2 years (excludes skin cancers that may have been excised within that 2 year period).
Who cannot join
- Serious active or uncontrolled infection or medical condition
- Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
- Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent
- Other systemic anticancer therapy or ongoing clinically relevant toxicities from such therapy (at discretion of the investigator)
- History of and/or current evidence of myocardial impairment (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, congestive heart failure), resulting in heart failure by New York Heart Association Class III or IV staging.
- Subjects with Wilson disease or other Copper-related disorders.
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medical College
New York, New York, 10021
About this study
Official title: A Pilot Study of a Novel Sequential Treatment Utilizing CPX-351 as Salvage Chemotherapy Followed by Allogeneic Stem-Cell Transplantation (SCT) Utilizing a Haplo-cord Graft for Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndrome
This pilot study is designed to evaluate outcomes with the combination of CPX-351 salvage therapy and haplo-cord graft stem cell transplantation for subjects with relapsed or refractory AML or myelodysplastic syndrome.
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia, Relapsed Adult Acute Myeloid, Myelodysplastic Syndromes, Previously Treated
- Being tested
- CPX-351, Fludarabine, Melphalan, Rabbit Anti-Human T-Lymphocyte Globulin, Haplo-Cord Stem Cell Transplantation
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
Also called Cytarabine:Daunorubicin Liposome Injection
Salvage Chemotherapy: CPX-351 at 120 u/m2 on Days -21, -19, and -17
FludarabineDrug
Also called Fludara
Fludarabine 150 mg/m2 (30 mg/m2/day x 5 days, Day -7 to Day -3)
MelphalanDrug
Also called Alkeran
Melphalan 140 mg/m2 (Day -2)
Rabbit Anti-Human T-Lymphocyte GlobulinDrug
Also called Thymoglobulin
Rabbit ATG (rATG)-thymoglobulin 4.5 mg/kg (1.5 mg/kg/day x 3 days)
Haplo-Cord Stem Cell TransplantationBiological
Allogeneic stem cell transplantation using a haploidentical donor and umbilical cord blood unit.
| Study group | Role | What they receive |
|---|---|---|
| CPX-351 Salvage Therapy and Transplant | Experimental | Subjects will receive CPX-351 salvage chemotherapy on Day -21, -19, and -17 as a bridge to allogeneic stem cell transplantation using a Fludarabine/Melphalan/rATG conditioning regimen and a haplo-cord graft. Receives: Drug: CPX-351; Drug: Fludarabine; Drug: Melphalan; Drug: Rabbit Anti-Human T-Lymphocyte Globulin; Biological: Haplo-Cord Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Evaluated using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE)
Measured: 1 year
Neutrophil Engraftment
Evaluate the time to neutrophil engraftment, defined as the first day in which absolute neutrophil count (ANC) >500/mm3 for three consecutive days
Measured: 100 days
Overall Survival at Day 100, 6 months, and 1 year
Evaluate survival of subjects alive, with or without presence of disease, at the designated time points
Measured: Day 100, 6 months, and 1 year post-transplant
Disease-Free Survival at Day 100, 6 months, and 1 year
Evaluate survival of subjects alive without disease at the designated time points
Measured: Day 100, 6 months, and 1 year post-transplant
Secondary outcomes (2)
Non-Relapse Mortality
Death that cannot be explained by persistence, relapse, or progression of underlying disease
Measured: Day 100
Relapse Rate
Time to first relapse or progression of underlying disease after initiation of protocol therapy
Measured: Day 100, 6 months, 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2018
- Study start30 Nov 2012
- Primary completion30 Aug 2021
- Study completion18 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe 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
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Sebastian Mayer, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03393611
- Sponsor's protocol ID
- 1107011830
- Status last verified
- Dec 2022
- First submitted
- 7 Dec 2017
- Last updated
- 21 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of a Novel Sequential Treatment Utilizing CPX-351 as Salvage Chemotherapy Followed by Allogeneic Stem-Cell Transplantation (SCT) Utilizing a Haplo-cord Graft for Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT03393611
- Acronym / sponsor ID
- 1107011830
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Leukemia, Relapsed Adult Acute Myeloid, Myelodysplastic Syndromes, Previously Treated
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 30 Nov 2012 (actual)
- Primary completion
- 30 Aug 2021 (actual)
- Study completion
- 18 Nov 2021 (actual)
- First posted
- 8 Jan 2018 (actual)
- 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.
This pilot study is designed to evaluate outcomes with the combination of CPX-351 salvage therapy and haplo-cord graft stem cell transplantation for subjects with relapsed or refractory AML or myelodysplastic syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CPX-351 Salvage Therapy and Transplant | Experimental | Drug: CPX-351, Drug: Fludarabine, Drug: Melphalan, Drug: Rabbit Anti-Human T-Lymphocyte Globulin, Biological: Haplo-Cord Stem Cell TransplantationSubjects will receive CPX-351 salvage chemotherapy on Day -21, -19, and -17 as a bridge to allogeneic stem cell transplantation using a Fludarabine/Melphalan/rATG conditioning regimen and a haplo-cord graft. |
Who may qualify
- Ages
- 18 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Subject must have refractory or relapsed Acute Myeloid Leukemia (AML) according to previously established criteria:
- Subjects with Myelodysplastic Syndrome (MDS):
- Karnofsky performance status ≥ 70
- Willing to participate as a research subject and sign an informed consent form
- Adequate physical function measured by:
Key exclusion criteria (6 listed)
- Serious active or uncontrolled infection or medical condition
- Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
- Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent
- Other systemic anticancer therapy or ongoing clinically relevant toxicities from such therapy (at discretion of the investigator)
- History of and/or current evidence of myocardial impairment (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, congestive heart failure), resulting in heart failure by New York Heart Association Class III or IV staging.
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 |
|---|---|---|
| CPX-351Also: Cytarabine:Daunorubicin Liposome InjectionSalvage Chemotherapy: CPX-351 at 120 u/m2 on Days -21, -19, and -17 | Drug | CPX-351 Salvage Therapy and Transplant |
| FludarabineAlso: FludaraFludarabine 150 mg/m2 (30 mg/m2/day x 5 days, Day -7 to Day -3) | Drug | CPX-351 Salvage Therapy and Transplant |
| MelphalanAlso: AlkeranMelphalan 140 mg/m2 (Day -2) | Drug | CPX-351 Salvage Therapy and Transplant |
| Rabbit Anti-Human T-Lymphocyte GlobulinAlso: ThymoglobulinRabbit ATG (rATG)-thymoglobulin 4.5 mg/kg (1.5 mg/kg/day x 3 days) | Drug | CPX-351 Salvage Therapy and Transplant |
| Haplo-Cord Stem Cell TransplantationAllogeneic stem cell transplantation using a haploidentical donor and umbilical cord blood unit. | Biological | CPX-351 Salvage Therapy and Transplant |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 1 year
Evaluated using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE)
Neutrophil Engraftment
Time frame: 100 days
Evaluate the time to neutrophil engraftment, defined as the first day in which absolute neutrophil count (ANC) >500/mm3 for three consecutive days
Overall Survival at Day 100, 6 months, and 1 year
Time frame: Day 100, 6 months, and 1 year post-transplant
Evaluate survival of subjects alive, with or without presence of disease, at the designated time points
Disease-Free Survival at Day 100, 6 months, and 1 year
Time frame: Day 100, 6 months, and 1 year post-transplant
Evaluate survival of subjects alive without disease at the designated time points
2 secondary outcomes
Non-Relapse Mortality
Time frame: Day 100
Relapse Rate
Time frame: Day 100, 6 months, 1 year
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 21 Dec 2022.
Related trials for Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Sebastian Mayer, MD principal investigator
Weill Medical College of Cornell University
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 CPX-351, Fludarabine and Melphalan in people with Myelodysplastic Syndromes and Leukemia, Myeloid, Acute, run by Weill Medical College of Cornell University 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 15 inclusion and 6 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 21 Dec 2022. 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 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?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 1 year). The study lists 4 primary outcomes in all. 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 21 Dec 2022.