Trial · NCT01553149

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

NCT01553149, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2021-11-30

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma. Trials, record NCT01553149. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01553149

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
NCT number: NCT01553149
Status: Completed
Phases: Phase 2
Enrollment: 75 participants, actual
Lead sponsor: National Cancer Institute (NCI)
Conditions: Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
Interventions: Lenalidomide, Pharmacological Study
First posted: 2012-03-14
Last updated: 2025-01-07
Results first posted: 2021-11-30

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Patients Who Demonstrate Complete or Partial Response	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.
Primary	Number of Patients Who Demonstrate Early Progression	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.
Secondary	Event-free Survival [EFS]	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.
Secondary	Overall Survival [OS]	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.
Secondary	Number of Patients With Toxic Events After 2 Dose Reductions	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.
Secondary	Pharmacokinetic Parameters of Lenalidomide	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.
Secondary	Magnetic Resonance Imaging Sequence	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.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Patients Who Demonstrate Complete or Partial Response	26 cycles of chemotherapy - up to 3 years after enrollment	Only eligible patients are considered in the analysis of this outcome measure	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Patients Who Demonstrate Early Progression	Up to 180 days after enrollment	Only eligible patients are considered in the analysis of this outcome measure	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Event-free Survival [EFS]	Up to 3 years after study enrollment	Only eligible patients are considered in the analysis of this outcome measure	Percent Probability	NUMBER
SECONDARY	Overall Survival [OS]	Up to 3 years after study enrollment	Only eligible patients are considered in the analysis of this outcome measure	Percent Probability	NUMBER
SECONDARY	Number of Patients With Toxic Events After 2 Dose Reductions	While receiving protocol therapy up to 3 years after study enrollment	Only eligible patients are considered in the analysis of this outcome measure	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Pharmacokinetic Parameters of Lenalidomide	Between days 5-21 of course 1 and each dose reduction	Only eligible patients who have lenalidomide concentration measure are considered in the analysis of this secondary outcome measure	nanograms per mL	MEDIAN
SECONDARY	Magnetic Resonance Imaging Sequence	Up to 3 years	Scans completed and reviewed by standard magnetic resonance sequences. Only eligible patients are considered in the analysis of this outcome measure.	Number of scans in agreement	NUMBER

Source ClinicalTrials.gov

Results by group18

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Number of Patients Who Demonstrate Complete or Partial Response	Arm I (Low-dose Lenalidomide)	4		Participants
Number of Patients Who Demonstrate Complete or Partial Response	Arm II (High-dose Lenalidomide)	4		Participants
Number of Patients Who Demonstrate Early Progression	Arm I (Low-dose Lenalidomide)	6		Participants
Number of Patients Who Demonstrate Early Progression	Arm II (High-dose Lenalidomide)	4		Participants
Event-free Survival [EFS]	Arm I (Low-dose Lenalidomide)	37.84	95% Confidence Interval 22.62 to 52.97	Percent Probability
Event-free Survival [EFS]	Arm II (High-dose Lenalidomide)	39.41	95% Confidence Interval 23.72 to 54.74	Percent Probability
Overall Survival [OS]	Arm I (Low-dose Lenalidomide)	94.59	95% Confidence Interval 80.07 to 98.62	Percent Probability
Overall Survival [OS]	Arm II (High-dose Lenalidomide)	91.74	95% Confidence Interval 76.52 to 97.26	Percent Probability
Number of Patients With Toxic Events After 2 Dose Reductions	Arm I (Low-dose Lenalidomide)	2		Participants
Number of Patients With Toxic Events After 2 Dose Reductions	Arm II (High-dose Lenalidomide)	16		Participants

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Arm I (Low-dose Lenalidomide)	37
Overall Study	STARTED	Arm II (High-dose Lenalidomide)	38
Overall Study	COMPLETED	Arm I (Low-dose Lenalidomide)	32
Overall Study	COMPLETED	Arm II (High-dose Lenalidomide)	18
Overall Study	NOT COMPLETED	Arm I (Low-dose Lenalidomide)	5
Overall Study	NOT COMPLETED	Arm II (High-dose Lenalidomide)	20

Source ClinicalTrials.gov

Adverse events138

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anemia	Blood and lymphatic system disorders	Arm I (Low-dose Lenalidomide)	0	37	0
other	Anemia	Blood and lymphatic system disorders	Arm II (High-dose Lenalidomide)	2	37	2
other	Eye disorders - Other, specify	Eye disorders	Arm I (Low-dose Lenalidomide)	0	37	0
other	Eye disorders - Other, specify	Eye disorders	Arm II (High-dose Lenalidomide)	1	37	1
other	Optic nerve disorder	Eye disorders	Arm I (Low-dose Lenalidomide)	0	37	0
other	Optic nerve disorder	Eye disorders	Arm II (High-dose Lenalidomide)	1	37	1
other	Diarrhea	Gastrointestinal disorders	Arm I (Low-dose Lenalidomide)	4	37	4
other	Diarrhea	Gastrointestinal disorders	Arm II (High-dose Lenalidomide)	0	37	0
other	Nausea	Gastrointestinal disorders	Arm I (Low-dose Lenalidomide)	1	37	1
other	Nausea	Gastrointestinal disorders	Arm II (High-dose Lenalidomide)	1	37	1

1 to 10 of 138Source ClinicalTrials.gov

See all 138 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Arm I (Low-dose Lenalidomide)	4	37
Total, all-cause mortality	Arm II (High-dose Lenalidomide)	4	37
Total, other adverse events	Arm I (Low-dose Lenalidomide)	15	37
Total, other adverse events	Arm II (High-dose Lenalidomide)	30	37
Total, serious adverse events	Arm I (Low-dose Lenalidomide)	6	37
Total, serious adverse events	Arm II (High-dose Lenalidomide)	16	37

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37126770	DERIVED

Source PubMed

Grants2

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years	Source
U10CA180886	COG NCTN Network Group Operations Center	Hawkins, Douglas S.	COG RESEARCH FOUNDATION, LLC	NATIONAL CANCER INSTITUTE	2014 to 2026	NIH RePORTER
U10CA098543	Children's Oncology Group Chair's Grant	ADAMSON, PETER C.	CHILDREN'S HOSP OF PHILADELPHIA	NATIONAL CANCER INSTITUTE	2003 to 2013	NIH RePORTER

Trial comparisons5

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Arm I (Low-dose Lenalidomide) vs Arm II (High-dose Lenalidomide): Number of Patients With Toxic Events After 2 Dose Reductions	Trial comparisons
Arm I (Low-dose Lenalidomide) vs Arm II (High-dose Lenalidomide): deaths from any cause	Trial comparisons
Arm I (Low-dose Lenalidomide) vs Arm II (High-dose Lenalidomide): Number of Patients Who Demonstrate Early Progression	Trial comparisons
Arm I (Low-dose Lenalidomide) vs Arm II (High-dose Lenalidomide): Number of Patients Who Demonstrate Complete or Partial Response	Trial comparisons
Arm I (Low-dose Lenalidomide) vs Arm II (High-dose Lenalidomide): serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01553149

GET https://conductscience.org/api/trials/v1/NCT01553149/events?page=2

API reference For AI assistants