Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
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|ClinicalTrials.gov Identifier: NCT01553149|
Recruitment Status : Active, not recruiting
First Posted : March 14, 2012
Results First Posted : November 30, 2021
Last Update Posted : September 19, 2022
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis Type 1 Recurrent Childhood Pilocytic Astrocytoma Recurrent Childhood Visual Pathway Glioma||Drug: Lenalidomide Other: Pharmacological Study||Phase 2|
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.
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||75 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Randomized Trial of Lenalidomide (NSC # 703813) in Pediatric Patients With Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas|
|Actual Study Start Date :||March 19, 2012|
|Actual Primary Completion Date :||June 30, 2020|
Experimental: Arm I (low-dose lenalidomide)
Patients receive low-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.
Other: Pharmacological Study
Experimental: Arm II (high-dose lenalidomide)
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.
Other: Pharmacological Study
- Number of Patients Who Demonstrate Complete or Partial Response [ Time Frame: 26 cycles of chemotherapy - up to 3 years after enrollment ]
Number of patients who demonstrate a complete or partial response as defined below:
Complete Response - Complete disappearance of all known disease for at least 4 weeks;
Partial Response - A reduction of at least 50% in the size of all measurable tumor as quantitated by the sum of the products of the largest diameters of measurable lesions when compared with that measurement at the time of study enrollment and maintained for at least 4 weeks.
- Number of Patients Who Demonstrate Early Progression [ Time Frame: Up to 180 days after enrollment ]Number of patients with disease progression during the first six months of protocol therapy. Disease progression is defined as ≥ 25% increase in the sum of the products of the largest diameters of the measurable lesions or the appearance of one or more new lesions when compared with the measurements of lesions at the time of enrollment.
- Event-free Survival [EFS] [ Time Frame: Up to 3 years after study enrollment ]Estimated 3-year EFS where EFS is calculated as the time from study enrollment to disease progression, disease relapse, occurrence of a second malignant neoplasm, death from any cause or last follow-up whichever occurs first. Kaplan-Meier method is used for estimation. Patients without an event are censored at last contact.
- Overall Survival [OS] [ Time Frame: Up to 3 years after study enrollment ]Estimated 3-year overall survival is calculated as the time from study enrollment to death from any cause or last follow-up whichever occurs first. Kaplan-Meier method is used for estimation. Patients alive at last contact are censored at that time.
- Number of Patients With Toxic Events After 2 Dose Reductions [ Time Frame: While receiving protocol therapy up to 3 years after study enrollment ]Number of patients who have an additional significant toxicity coded using Common Terminology Criteria for Adverse Events Version 5.0 after experiencing two dose reductions from their assigned treatment dose.
- Pharmacokinetic Parameters of Lenalidomide [ Time Frame: Between days 5-21 of course 1 and each dose reduction ]Concentration of lenalidomide obtained from any day between day 5 and 21 of the first cycle of chemotherapy in nanograms per mL.
- Magnetic Resonance Imaging Sequence [ Time Frame: Up to 3 years ]Response categories (complete response, partial response, stable disease, and progression) will be determined from the following three standard magnetic resonance sequences, T2-weighted, fluid attenuated inversion recovery, T1-weighted post-contrast. Percent agreement between the sequences will be estimated as the number of follow-up scans in which the corresponding sequence agreed divided by the total number of follow-up scans.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01553149
|Principal Investigator:||Katherine E Warren||Children's Oncology Group|