RecruitingPhase 1

A Study to Assess Adverse Events and How Intravenous (IV) Pivekimab Sunirine Moves Through the Body in Pediatric Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML)

A Phase 1b Study of the Safety and Pharmacokinetics of Pivekimab Sunirine in Pediatric Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
6 Months to 17 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Blood draws

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests pivekimab sunirine in children with acute myeloid leukemia (AML), a fast-growing blood cancer that has come back or has not responded to treatment. It looks at side effects and how the drug moves through the body. The drug is given through a vein (IV).

Acute myeloid leukemia (AML) is an aggressive blood cancer, withwith few options for participants who relapse after treatment or who don't respond to treatment. This study will assess the adverse events and how pivekimab sunirine moves through the body in pediatric participants with relapsed or refractory (R/R) AML. Pivekimab sunirine is a drug being evaluated in the treatment of AML. This is an open label, single arm study, participants will be enrolled in 1 of the 3 cohorts based on their age and will receive pivekimab sunirine at a dose based on their weight. Around 18 pediatric participants with a diagnosis of AML will be enrolled in the study at approximately 30 sites around the world. Participants will receive intravenous (IV) pivekimab sunirine alone. The total study duration is approximately 28 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have AML confirmed by a tissue test. It must have come back at least twice or resisted at least one cycle of each of two or more treatment plans.
  • Have leukemia cells that test positive for CD123, a marker on the cells.
  • Have at least 5% immature leukemia cells in the bone marrow when the cancer returns or resists treatment, before study screening.
  • Meet activity and daily-function score limits: Lansky or Karnofsky score of at least 50, or ECOG score of 2 or less.
  • May have leukemia in the brain or spinal cord area, but must have no related signs or nerve symptoms. Treatment into the fluid around the brain and spinal cord may continue, but no other whole-body treatment aimed at this area is allowed at study entry.
  • If too young to give legal consent, have a parent or guardian who can and will give consent. The child must also agree when appropriate for their age and country.

Who can't join

  • Have known serious heart disease.
  • Have Down syndrome, acute promyelocytic leukemia (APL), or juvenile myelomonocytic leukemia (JMML).
  • Have leukemia in the brain or spinal cord area that causes symptoms.
  • Have ever had VOD/SOS, a condition that blocks small blood vessels in the liver.
  • Had a blood-forming stem cell transplant within 6 months before screening, with no active graft-versus-host disease (GvHD), and are off medicines to treat or prevent GvHD or transplant rejection, except a stable steroid dose. GvHD is when donor cells attack the body.
  • Have ever received CAR-T therapy, a treatment that changes immune cells to fight cancer.
  • Have another current cancer that needs treatment.
  • Are currently getting treatment to fight cancer, such as radiation, cancer drugs, or other study drugs. Treatment into the fluid around the brain and spinal cord is allowed.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Must have histologically confirmed acute myeloid leukemia (AML) meeting one of the following disease criteria: * Second or greater relapse. OR * Disease refractory to second or subsequent line of therapy (defined as resistant disease after at least one cycle of each treatment regimen). * Must have myeloid leukemic blasts that are CD123-positive by flow cytometry as determined by the treating institution. * Has \>= 5% myeloid leukemic blasts in bone marrow at time of relapse or refractory disease and prior to Screening for this study. * Performance status by Lansky (\< 16 years old at evaluation) or Karnofsky (\>= 16 years old at evaluation) score \>= 50 or ECOG score \<= 2. * May have status of central nervous system (CNS)1, CNS2, or CNS3 disease without clinical signs or neurologic symptoms suggestive of CNS leukemia, such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome. Participants receiving intrathecal therapy and no additional CNS-directed systemic therapy at study entry are eligible and may continue treatment as clinically indicated in accordance with institutional practice. * For those participants who have not reached the age of consent, parent or legal guardian with the willingness and ability to provide informed consent and participant willing and able to give assent, as appropriate for age and country. Exclusion Criteria: * Known clinically significant cardiac disease. * Down syndrome. * Acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML). * Symptomatic central nervous system (CNS3) disease * Prior history of any severity veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) of the liver. * Prior history of hematopoietic stem cell transplant within 6 months prior to Screening without evidence of active GvHD at the time of screening and the participant is off medications to treat or prevent either post-transplant graft-versus-host disease (GvHD) or post-transplant rejection (except for a stable dose of corticosteroids). * Have received prior Chimeric Antigen Receptor T-cell (CAR-T) therapy. * Any other known current malignancy requiring therapy. * Currently receiving anticancer therapy with antineoplastic intent, including radiotherapy, systemic therapy small molecules, monoclonal antibodies, other investigational agents, or high-dose chemotherapy with the exception of intrathecal therapy.

Study sites (5)

CA

GA

  • Children'S Healthcare Of Atlanta - Arthur M. Blank Hospital /ID# 275740 · Atlanta, GA 30329More trials in Atlanta

NY

TN

TX