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ALL, ChildhoodPhase 1NCT02879643

Vincristine Sulfate Liposome Injection (Marqibo®) in Combination With UK ALL R3 Induction Chemotherapy

A Phase 1 study testing Marqibo in people with ALL, Childhood and Lymphoblastic Leukemia, Acute, Childhood, run by Therapeutic Advances in Childhood Leukemia Consortium at 35 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a pilot study utilizing Marqibo® (vincristine sulfate liposome injection) combined with dexamethasone, mitoxantrone and asparaginase (UK ALL R3) for relapsed acute lymphoblastic leukemia (ALL). Read more about this study

Who is it for?
Ages 1 year to 21 years
What is being tested?
Marqibo
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
35 sitesin 3 countries
Study dates
31 Jan 2017to 31 Dec 2024
Phase
Phase 1
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 1 year to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age
  • -Patients must be ≥ 1 and ≤ 21 years of age at the time of enrollment.
  • Diagnosis
  • Cohort A: Patients must have a diagnosis of acute lymphoblastic leukemia (ALL) or mixed phenotypic acute leukemia with ≥ 5% blasts in the bone marrow (M2 or M3), with or without extramedullary disease) or a diagnosis of lymphoblastic lymphoma.
  • Cohorts B \& C: Patients must have a diagnosis of acute lymphoblastic leukemia (ALL), lymphoblastic lymphoma, or mixed phenotypic acute leukemia with any level of detectable disease (minimal residual disease level acceptable) with or without extramedullary disease
  • Performance Level -Karnofsky > 50% for patients > 16 years of age and Lansky > 50% for patients ≤ 16 years of age.
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  • Prior Therapy
  • Patients must have recovered from the acute toxic effects (≤ Grade 2 or baseline) of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, unless otherwise specified. Subjects with disease related cytopenias will be eligible.
  • Patients must have relapsed or refractory disease after attaining at least a first remission. They may be in first to third relapse..
  • Patients with Philadelphia chromosome t(9;22) positive disease must have received at least two prior tyrosine kinase inhibitors.
  • Patients who have experienced their relapse after a Hematopoietic stem cell transplantation (HSCT) are eligible, provided they have no evidence of graft-versus-host disease (GVHD) and are at least 100 days post-transplant at the time of enrollment.
  • Prior anthracycline lifetime cumulative exposure: Patients must have less than 320 mg/m2 (or 400 mg/m2 if prior cardioprotection) lifetime exposure of anthracycline chemotherapy.
  • Cohort A: Patients must have less than 320 mg/m2 (or 400 mg/m2 if prior cardioprotection) lifetime exposure of anthracycline chemotherapy (See Appendix 2 for anthracycline calculation worksheet).
  • Cohorts B \& C: There is no limit on prior anthracycline exposure.
  • Hematopoietic growth factors: It must have been at least seven days since the completion of therapy with granulocyte colony-stimulating factor (GCSF) or other growth factors at the time of enrollment. It must have been at least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).
  • Biologic anti-neoplastic agents: At least seven days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond seven days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair or vice chair.
  • Monoclonal antibodies: At least three half-lives (or 30 days-whichever is longer) of the antibody must have elapsed after the last dose of monoclonal antibody. (e.g., Rituximab = 66 days, Epratuzumab = 69 days)
  • Immunotherapy: At least 30 days after the completion of any type of immunotherapy, e.g. tumor vaccines, chimeric antigen receptor T-cells.
  • Recent prior chemotherapy: At least 10 days after standard vincristine and the completion of any type of chemotherapy induction regimen. At least 3 weeks after radiation therapy. At least 30 days after the completion of any investigational neoplastic agent is also required. An investigational agent is defined as any drug that is not approved and licensed for sale by the FDA for institutions in the United States, by Health Canada for institutions in Canada and by The Therapeutic Goods Administration for institutions in Australia.
  • Exceptions:
  • There is no time restriction in regard to prior intrathecal chemotherapy provided there is complete recovery from any acute toxic effects of such; it is allowable to enroll a patient that has received IT Cytarabine (ARA-C), IT Methotrexate (MTX) or triple IT therapy within 14 days of enrollment as part of their evaluation to diagnose disease relapse. The IT therapy given within 14 days of initiation of protocol specified chemotherapy, may substitute for the day 1 IT in cohorts A and B
  • Subjects with rapidly progressive disease may receive hydroxyurea until they begin study therapy;
  • Patients who relapse while on maintenance-type ALL therapy or are receiving maintenance therapy for disease stabilization will not require a wash-out period before entry into this study. However, there must be at least 10 days after any dose of standard vincristine.
  • Renal and Hepatic Function
  • Renal function: Patient's serum creatinine must be ≤ 1.5 x institutional upper limit of normal (ULN) according to age. If the serum creatinine is greater than 1.5 times normal, the patient must have a calculated creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 70milliliter/min/1.73m2. Alternatively, a 24-hour creatinine clearance may also be used.
  • Hepatic function: alanine aminotransferase (ALT) and aspartate aminotransferase (AST) must be < 5 x institutional upper limit of norm ULN. Total bilirubin must be ≤ 1.5 x ULN (except in the case of subjects with documented Gilbert's disease ≤ 5 × ULN).
  • Cardiac Function
  • -Patients must have a shortening fraction ≥ 27% or an ejection fraction ≥ 55% by echocardiogram, cardiac MRI or multigated acquisition scan (MUGA).
  • Reproductive Function
  • Female patients must not be pregnant and those of childbearing potential must have a negative urine or serum pregnancy test confirmed within one week prior to enrollment.
  • Female patients with infants must agree not to breastfeed their infants while on this study.
  • Male and female patients of childbearing potential must agree to use an effective method of contraception during the study.

Who cannot join

  • Patients will be excluded if they have isolated testicular disease.
  • Patients will be excluded if they have previously received Marqibo®.
  • Patients will be excluded if they have a known allergy to any of the drugs used in the study, with the exception that patients with an allergy to PEG-asparaginase who can receive Erwinia asparaginase are eligible. Patients unable to receive any formulation of asparaginase may only enroll on cohort C
  • Patients will be excluded if they have active, uncontrolled systemic fungal, bacterial, viral or other infection despite appropriate antibiotics or other treatment.
  • Patients who require azole antifungal agents will be excluded. Azoles must be discontinued at least one week prior to the start of Marqibo®.
  • Patients will be excluded if there is a plan to administer non-protocol chemotherapy, radiation therapy, another investigational agent or immunotherapy during the study period.
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  • Patients with pre-existing, persistent grade 2 or greater sensory or motor neuropathy from any cause will be excluded.
  • Patients will be excluded if they have, significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or adherence with the protocol treatment or procedures or interfere with consent, study participation, follow up, or interpretation of study results.Patients with Down syndrome will not be eligible for enrollment on Cohort A
  • Patients with a known history of human immunodeficiency virus (HIV) will will be excluded due to the increased risk of complications such as severe infection and unknown interaction of Marqibo® with antiretroviral drugs.
  • Active hepatitis B or C infection as defined by seropositive for hepatitis B (hepatitis B surface antigen (HBsAg)) or hepatitis C and elevated liver transaminases (defined as above the ULN per the institution normal ranges).

Trial locations and contacts

35 sites in 3 countries.

35 sites in 3 countries

United States28 sites

  • CS Mott Children's Hospital, Ann Arbor

    Ann Arbor, Michigan, 48109

  • Children's Healthcare of Atlanta at Egleston

    Atlanta, Georgia, 30322

  • The Children's Hospital, University of Colorado

    Aurora, Colorado, 80045

  • Sidney Kimmel Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231

  • National Cancer Institute, Pediatric Oncology Branch

    Bethesda, Maryland, 20892

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Levine Children's Hospital

    Charlotte, North Carolina, 28203

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229

  • Rainbow Babies & Children's Hospital

    Cleveland, Ohio, 44106

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75235

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

  • Texas Children's Cancer Center, Baylor

    Houston, Texas, 77030

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027

  • University of Miami

    Miami, Florida, 33136

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

  • Monroe Carell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee, 37232

  • Children's Hospital New York-Presbyterian

    New York, New York, 10032

  • Children's Hospital Orange County

    Orange, California, 92868

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

  • UCSF School of Medicine

    San Francisco, California, 94158

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • All Children's Hospital

    St. Petersburg, Florida, 33701

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

Australia4 sites

  • Royal Children's Hospital, Melbourne

    Parkville, Victoria, 3052

  • Sydney Children's Hospital

    Randwick, New South Wales, 2031

  • Lady Cilento Children's Hospital

    South Brisbane, Queensland, 4101

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145

Other trials in Australia

Canada3 sites

  • Sainte-Justine University Hospital Center

    Montréal, Québec, H3T-1C5

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3V4

Other trials in Canada

About this study

Official title: A Pilot Study of Vincristine Sulfate Liposome Injection (Marqibo®) in Combination With UK ALL R3 Induction Chemotherapy for Children, Adolescents, and Young Adults With Relapse of Acute Lymphoblastic Leukemia

This is a pilot study utilizing Marqibo® (vincristine sulfate liposome injection) combined with dexamethasone, mitoxantrone and asparaginase (UK ALL R3) for relapsed acute lymphoblastic leukemia (ALL).

Read the detailed description

This study will utilize Marqibo® as a replacement for standard vincristine in combination with chemotherapy for children with relapsed ALL. The hypothesis is that the incorporation of Marqibo® with combination chemotherapy will be safe and feasible. In the context of this pilot study, overall outcomes and efficacy will be a secondary objective. It is hypothesized that data from this combination may show improved efficacy including, complete remission (CR), minimal residual disease (MRD) negativity, and progression free survival (PFS) rates and safety (i.e., neurotoxicity) in comparison to outcomes in historical regimens, including the UK ALL R3 with standard vincristine.

Being tested
Marqibo
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MarqiboDrug

    The focus of the study design and statistical analysis is to determine whether Marqibo® can be substituted for standard vincristine and successfully administered to patients with relapsed ALL in three different treatment cohorts: (A) treatment with the UK ALL R3 regimen; (B) treatment with UK ALL R3 regimen without mitoxantrone, and (C) treatment with ALL maintenance therapy. Secondary objectives include estimating rates of serious toxicities and therapy delays in each of these cohorts.

Study groups in NCT02879643
Study groupRoleWhat they receive
Cohort A: Marqibo and UK ALL R3 backboneExperimental* Marqibo®: given by intravenous (IV) infusion on days 1, 8, 15 and 22. * Dexamethasone orally twice daily on days 1-5 and 15-19. * Mitoxantrone: given by intravenous (IV) infusion on days 1 and 2. * PEG-asparaginase: given as an injection into the muscle on says 3 and 17. * Methotrexate IT: given intrathecally (used to treat the brain and spinal cord and is given using a needle inserted into the spinal canal) on days 1 and 8.
Receives: Drug: Marqibo
Cohort B: Marqibo and lower intensity UK ALL R3 backboneExperimental* Marqibo®: given by intravenous (IV) infusion on days 1, 8, 15 and 22. * Dexamethasone orally twice daily on days 1-5 and 15-19. * PEG-asparaginase: given as an injection into the muscle on days 3 and 17. * Methotrexate IT: given intrathecally (used to treat the brain and spinal cord and is given using a needle inserted into the spinal canal) on days 1 and 8.
Receives: Drug: Marqibo
Cohort C: Marqibo and maintenance regimenExperimental* Marqibo®: given by intravenous (IV) infusion on day 1 * Dexamethasone orally twice daily on days 1-5 * Methotrexate: given orally on days 1 and 8 * Mercaptopurine: given orally daily on days 1-13
Receives: Drug: Marqibo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Dose Limiting Toxicities as a Measure of Safety and Tolerability

    Number of Participants with Dose Limiting Toxicities as a Measure of Safety and Tolerability at different dose levels of Marqibo. Dose Level 1 = 1.5 mg/m^2. Dose Level 2 = 2.0 mg/m^2. Cohort A and B accrued at both Dose Levels. Cohort C accrued only at Dose Level 2.

    Measured: 9 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Aug 2016 (estimated)
  2. Study start31 Jan 2017
  3. Primary completion29 Jul 2022
  4. Study completion31 Dec 2024

Resources

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02879643
Sponsor's protocol ID
T2012-002
Status last verified
Jul 2022
First submitted
16 Aug 2016
Last updated
22 Oct 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Shah NN, Schafer ES, Chi YY, Malvar J, Heym KM, Place AE, Burns M, Chang BH, Slone T, Verma A, Gossai N, Shaw PH, Burke MJ, Hermiston M, Schore RJ, Cooper T, Pauly M, Rushing T, Jarosinski P, Florendo E, Yates B, Widemann BC, Peer CJ, Figg WD, Silverman LB, Bhojwani D, Wayne AS. Vincristine Sulfate Liposome Injection With Combination Chemotherapy for Children, Adolescents, and Young Adults With Relapsed Acute Lymphoblastic Leukemia: A Therapeutic Advances in Childhood Leukemia and Lymphoma Consortium Trial. Pediatr Blood Cancer. 2025 May;72(5):e31584. doi: 10.1002/pbc.31584. Epub 2025 Feb 12.

    PubMed 39937083

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Marqibo in people with ALL, Childhood and Lymphoblastic Leukemia, Acute, Childhood, run by Therapeutic Advances in Childhood Leukemia Consortium at 35 sites in 3 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 32 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: finished, as of the sponsor's last update on 22 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 35 sites in United States, Australia, Canada. 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?

Yes. Results are posted in the registry (first posted 22 Oct 2025). 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?

Number of Participants With Dose Limiting Toxicities as a Measure of Safety and Tolerability (time frame: 9 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02879643, which also lists 1 linked publication.

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 22 Oct 2025.

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