CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome

NCT ID: NCT03393611

Last Updated: 2022-12-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-30

Study Completion Date

2021-11-18

Brief Summary

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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.

Detailed Description

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Conditions

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Myelodysplastic Syndromes Leukemia, Myeloid, Acute Leukemia, Relapsed Adult Acute Myeloid Myelodysplastic Syndromes, Previously Treated

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CPX-351 Salvage Therapy and Transplant

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.

Group Type EXPERIMENTAL

CPX-351

Intervention Type DRUG

Salvage Chemotherapy: CPX-351 at 120 u/m2 on Days -21, -19, and -17

Fludarabine

Intervention Type DRUG

Fludarabine 150 mg/m2 (30 mg/m2/day x 5 days, Day -7 to Day -3)

Melphalan

Intervention Type DRUG

Melphalan 140 mg/m2 (Day -2)

Rabbit Anti-Human T-Lymphocyte Globulin

Intervention Type DRUG

Rabbit ATG (rATG)-thymoglobulin 4.5 mg/kg (1.5 mg/kg/day x 3 days)

Haplo-Cord Stem Cell Transplantation

Intervention Type BIOLOGICAL

Allogeneic stem cell transplantation using a haploidentical donor and umbilical cord blood unit.

Interventions

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CPX-351

Salvage Chemotherapy: CPX-351 at 120 u/m2 on Days -21, -19, and -17

Intervention Type DRUG

Fludarabine

Fludarabine 150 mg/m2 (30 mg/m2/day x 5 days, Day -7 to Day -3)

Intervention Type DRUG

Melphalan

Melphalan 140 mg/m2 (Day -2)

Intervention Type DRUG

Rabbit Anti-Human T-Lymphocyte Globulin

Rabbit ATG (rATG)-thymoglobulin 4.5 mg/kg (1.5 mg/kg/day x 3 days)

Intervention Type DRUG

Haplo-Cord Stem Cell Transplantation

Allogeneic stem cell transplantation using a haploidentical donor and umbilical cord blood unit.

Intervention Type BIOLOGICAL

Other Intervention Names

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Cytarabine:Daunorubicin Liposome Injection Fludara Alkeran Thymoglobulin

Eligibility Criteria

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Inclusion Criteria

1. Subject must have refractory or relapsed Acute Myeloid Leukemia (AML) according to previously established criteria:

1. Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy
2. First relapse
3. Relapse refractory to salvage chemotherapy
4. Second or subsequent relapse
2. Subjects with Myelodysplastic Syndrome (MDS):

(a) Either Refractory Anemia with Excess Blasts I or Refractory Anemia with Excess Blasts II (RAEB I or RAEB II)
3. Karnofsky performance status ≥ 70
4. Willing to participate as a research subject and sign an informed consent form
5. Adequate physical function measured by:

1. Cardiac: asymptomatic, or if symptomatic then Left Ventricular Ejection Fraction (LVEF) at rest must be ≥ 45% and must improve with exercise
2. 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
3. Renal: serum creatinine within normal range, or if serum creatinine is outside the normal range, then calculated creatinine clearance ≥ 60 ml/min
4. Pulmonary: asymptomatic, or if symptomatic, diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 45% of predicted (corrected for hemoglobin)
6. 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).

Exclusion Criteria

1. Serious active or uncontrolled infection or medical condition
2. Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
3. Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent
4. Other systemic anticancer therapy or ongoing clinically relevant toxicities from such therapy (at discretion of the investigator)
5. 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.
6. Subjects with Wilson disease or other Copper-related disorders.
Minimum Eligible Age

18 Years

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Jazz Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Weill Medical College of Cornell University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sebastian Mayer, MD

Role: PRINCIPAL_INVESTIGATOR

Weill Medical College of Cornell University

Locations

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Weill Cornell Medical College

New York, New York, United States

Site Status

Countries

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United States

Other Identifiers

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1107011830

Identifier Type: -

Identifier Source: org_study_id