Active — Not RecruitingPhase 2

Study of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms

United States25 participantsStarted 2023-01-17

Plain-language summary

The purpose of this study is to learn the effects of treatment with an investigational drug, CPX-351 in patients with secondary myeloid neoplasms (SMNs).

Who can participate

Age range

1 Year – 21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Myelosuppressive chemotherapy: Must not have received myelosuppressive chemotherapy within 3 weeks of entry onto this study (excluding hydroxyurea). Cyto-reduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of CPX-351.
. Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with steroids, retinoids or hypomethylating agents. Note: For agents that have known adverse events occurring beyond 7 days after administration (i.e. monoclonal antibodies), this period must be extended beyond the time during which acute adverse events are known to occur.
. Radiation therapy (RT): ≥ 2 weeks for local palliative RT (small port); ≥ 6 months must have elapsed if prior craniospinal RT or if ≥ 50% radiation of pelvis; ≥ 6 weeks must have elapsed if other substantial BM radiation. Note: Patients must have received ≤ than 13.6 Gy prior radiation to the mediastinum. Patients with prior cumulative doxorubicin equivalent \> 400 mg/m2 and prior radiation (any dose) to the mediastinum are not eligible for the protocol.
. Hematopoietic stem cell transplantation: No evidence of active graft vs. host disease for at least 4 weeks. For allogeneic HSCT patients, ≥ 3 months must have elapsed since HSCT.
. Intrathecal cytotoxic therapy:
. Growth factors:

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Composite complete remission (CR) rates after one or two courses of CPX-351

Timeframe: After one or 2 course of CPX-351, no later than day 42 from the start of each course of chemotherapy

Complete remission with incomplete peripheral blood recovery (CRi) rates after one or two courses of CPX-351

Timeframe: After one or 2 course of CPX-351, no later than day 42 from the start of each course of chemotherapy

Safety and tolerability in patients under 22 years of age with SMN treated with one or two courses of CPX-351 before HSCT

Timeframe: After one or 2 course of CPX-351, prior to hematopoietic stem cell transplantation (HSCT), three to four weeks from the hematologic recovery

Trial details

NCT IDNCT05656248

SponsorSt. Jude Children's Research Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-08-28

View on ClinicalTrials.gov More Myeloid Neoplasm trials

Who can participate

Age range

1 Year – 21 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Myelosuppressive chemotherapy: Must not have received myelosuppressive chemotherapy within 3 weeks of entry onto this study (excluding hydroxyurea). Cyto-reduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of CPX-351.
. Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with steroids, retinoids or hypomethylating agents. Note: For agents that have known adverse events occurring beyond 7 days after administration (i.e. monoclonal antibodies), this period must be extended beyond the time during which acute adverse events are known to occur.
. Radiation therapy (RT): ≥ 2 weeks for local palliative RT (small port); ≥ 6 months must have elapsed if prior craniospinal RT or if ≥ 50% radiation of pelvis; ≥ 6 weeks must have elapsed if other substantial BM radiation. Note: Patients must have received ≤ than 13.6 Gy prior radiation to the mediastinum. Patients with prior cumulative doxorubicin equivalent \> 400 mg/m2 and prior radiation (any dose) to the mediastinum are not eligible for the protocol.
. Hematopoietic stem cell transplantation: No evidence of active graft vs. host disease for at least 4 weeks. For allogeneic HSCT patients, ≥ 3 months must have elapsed since HSCT.
. Intrathecal cytotoxic therapy:
. Growth factors:

What they're measuring

Composite complete remission (CR) rates after one or two courses of CPX-351

Timeframe: After one or 2 course of CPX-351, no later than day 42 from the start of each course of chemotherapy

Complete remission with incomplete peripheral blood recovery (CRi) rates after one or two courses of CPX-351

Timeframe: After one or 2 course of CPX-351, no later than day 42 from the start of each course of chemotherapy

Safety and tolerability in patients under 22 years of age with SMN treated with one or two courses of CPX-351 before HSCT

Timeframe: After one or 2 course of CPX-351, prior to hematopoietic stem cell transplantation (HSCT), three to four weeks from the hematologic recovery