A Study of Lenalidomide in Pediatric Subjects With Relapsed or Refractory Acute Myeloid Leukemia
Condition: Leukemia, Myeloid
MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
Study type: Interventional
Phase: Phase 2

Summary: 
      To determine the activity of lenalidomide in the treatment of pediatric subjects with
      relapsed/refractory acute myeloid leukemia (AML) (with second or greater relapse or
      refractory to at least 2 prior induction attempts) measured by morphological complete
      response defined as either a CR or CRi within the first 4 cycles of treatment.
    

Detailed description: 
      This is a multicenter, open-label, single-arm, Phase 2, Simon's Optimal two-stage design
      study, with an Optional Extension Phase (OEP), that will assess the activity, safety and
      pharmacokinetics (PK) of lenalidomide in pediatric subjects from 1 to ≤ 18 years of age with
      second or greater Relapsed or Refractory Acute Myeloid Leukemia (rrAML). A total of 43
      evaluable participants (18 participants in Stage 1 and an additional 25 participants in Stage
      2) are required for assessment of the primary endpoint. To allow for participants found to be
      unevaluable for the primary endpoint due to an incorrect diagnosis, not having a disease
      assessment post screening, or who discontinued prior to receiving lenalidomide, up to 4
      additional participants may be enrolled for a maximum of 47 evaluable subjects across
      approximately 70 sites. Approximately 50% of enrolled participants will be younger than 12
      years of age to provide adequate PK data for this age subset.

      If during Stage 1, at least 3 of 18 participants achieve a morphologic complete response
      (either CR or CRi) within the first 4 cycles of study treatment, then the study will proceed
      to Stage 2; otherwise, the study will be terminated. Similarly, if at the final analysis, at
      least 8 of 43 evaluable subjects across Stages 1 and 2 achieve a response (CR/CRi) within the
      first 4 cycles of study treatment, it will be concluded that lenalidomide has sufficient
      activity in pediatric Acute Myeloid Leukemia (AML) to warrant subsequent study. The optional
      extension phase (OEP) will allow participants who demonstrate clinical benefit, as assessed
      by the Investigator at the completion of 12 cycles of lenalidomide therapy, to continue
      receiving oral lenalidomide until they meet the criteria for study discontinuation. In the
      OEP, only safety, dosing, concomitant medications/procedures, and second primary malignancies
      (SPMs) will be monitored.
    

Intervention name(s): Lenalidomide
Intervention type(s): Drug
Intervention description(s): Lenalidomide will be administered orally once daily for the first 21 days of every 28-day cycle. The starting dose will be 2 mg/kg/day with a maximum dose of 70 mg/day. Number of cycles: 12, or until evidence of progressive disease. Participants will also be discontinued if unresolved toxicities as described in the protocol occur, or if dose reductions are required and subject does not tolerate minimum dose level of 1mg/kg/day.
Intervention MeSH terms: Lenalidomide
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Lenalidomide; Revlimid; Pediatric; Relapsed or Refractory; Acute Myeloid Leukemia; Leukemia