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)

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Pivekimab Sunirine
  Intravenous

Первичные конечные точки

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Leading to Treatment Discontinuation [Срок оценки: Up to Approximately 24 Months]
• Maximum Observed Serum/Plasma Concentration (Cmax) of Intact Antibody-Drug Conjugate (ADC) [Срок оценки: Up to Approximately 22 Months]
• Cmax of FGN849 Payload [Срок оценки: Up to Approximately 22 Months]
• Area Under the Concentration-Time Curve (AUC) of Intact ADC [Срок оценки: Up to Approximately 22 Months]
• AUC of FGN849 payload [Срок оценки: Up to Approximately 22 Months]
• Time to Cmax (Tmax) of Intact ADC [Срок оценки: Up to Approximately 22 Months]
• Tmax of FGN849 Payload [Срок оценки: Up to Approximately 22 Months]

Вторичные конечные точки (6)

• Percentage of Participants Achieving Complete Remission (CR) [Срок оценки: Up to Approximately 28 Months]
• Percentage of Participants Achieving Composite Complete Remission (CR + complete remission with incomplete recovery [CRi]) [Срок оценки: Up to Approximately 28 Months]
• Percentage of Participants Achieving Composite Complete Remission (CR + complete remission with partial hematological [CRh]) [Срок оценки: Up to Approximately 28 Months]
• Duration of Complete Remission (DOCR) [Срок оценки: Up to Approximately 28 Months]
• Duration of Composite Complete Remission (CR + CRi) [Срок оценки: Up to Approximately 28 Months]
• Duration of Composite Complete Remission (CR + CRh) [Срок оценки: Up to Approximately 28 Months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 3 центра

• Lucile Packard Children's Hospital /ID# 276015 — Palo Alto
• New York Medical College /ID# 275597 — Valhalla
• Tristar Centennial Medical Center /ID# 275831 — Nashville

Франция · 2 центра

• Chu Bordeaux - Hopital Pellegrin /ID# 277645 — Bordeaux
• Hopital Armand Trousseau /ID# 276231 — Paris

South Korea · 2 центра

• Seoul National University Hospital /ID# 276978 — Seoul
• Samsung Medical Center /ID# 276979 — Seoul

Австралия · 1 центр

• Perth Children'S Hospital /ID# 275673 — Perth

Италия · 1 центр

• Ospedale Pediatrico Bambino Gesu /ID# 275692 — Rome

Тайвань · 1 центр

• National Taiwan University Hospital /ID# 276635 — Taipei

Идентификаторы

NCT: NCT07306832 · M25-692 · 2024-520125-36

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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