NCT01553149

Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma

A Phase II Randomized Trial of Lenalidomide (NSC # 703813) in Pediatric Patients With Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

This randomized phase II trial studies how well low-dose lenalidomide works compared with high-dose lenalidomide in treating younger patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas that have come back (recurrent), have not responded to treatment (refractory), or are growing, spreading, or getting worse (progressive). Lenalidomide is classified as an immunomodulatory drug as it boosts the immune system. It has other potential anti-tumor effects, for example, it may stop the growth of tumor cells by blocking blood flow to the tumor. It is not yet known whether low-dose lenalidomide is more or less effective than high-dose lenalidomide in treating patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas.

Detailed description

PRIMARY OBJECTIVES: I. To determine the objective response rate in children with recurrent, refractory, or progressive juvenile pilocytic astrocytomas and optic pathway gliomas who are treated with Regimen A low-dose (20 mg/m\^2/dose) or Regimen B high-dose (115 mg/m\^2/dose) lenalidomide. SECONDARY OBJECTIVES: I. To estimate the event-free survival (EFS) (based on standard two-dimensional tumor measurements, determined by each institution) of children with recurrent, refractory, or progressive juvenile pilocytic astrocytomas and optic pathway gliomas who are treated with lenalidomide. II. To compare response categories and EFS across the 3 magnetic resonance (MR) sequences (T2-weighted, fluid attenuated inversion recovery \[FLAIR\], T1-weighted post-contrast). III. To correlate steady-state pharmacokinetics of lenalidomide (1 sample obtained between days 5-21) with objective response and EFS. IV. To evaluate toxicities of long-term lenalidomide use. OUTLINE: Patients are randomized to 1 of 2 treatment arms. ARM I (Regimen A): Patients receive low-dose lenalidomide orally (PO) once daily (QD) on days 1-21. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. ARM II (Regimen B): Patients receive high-dose lenalidomide PO QD on days 1-21. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up annually for approximately 3 years.

Conditions

• Neurofibromatosis Type 1
• Recurrent Childhood Pilocytic Astrocytoma
• Recurrent Childhood Visual Pathway Glioma

Interventions

Type	Name	Description
DRUG	Lenalidomide	Given PO
OTHER	Pharmacological Study	Correlative studies

Timeline

• Start date: 2012-07-05
• Primary completion: 2020-06-30
• Completion: 2023-12-31
• First posted: 2012-03-14
• Last updated: 2025-01-07
• Results posted: 2021-11-30

Locations

126 sites across 4 countries: United States, Australia, Canada, New Zealand