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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01553149




Registration number
NCT01553149
Ethics application status
Date submitted
10/03/2012
Date registered
14/03/2012
Date last updated
3/07/2024

Titles & IDs
Public title
Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
Scientific 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
Secondary ID [1] 0 0
NCI-2012-00703
Secondary ID [2] 0 0
NCI-2012-00703
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neurofibromatosis Type 1 0 0
Recurrent Childhood Pilocytic Astrocytoma 0 0
Recurrent Childhood Visual Pathway Glioma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Children's - Brain
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Lenalidomide
Other interventions - Pharmacological Study

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.

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.


Treatment: Drugs: Lenalidomide
Given PO

Other interventions: Pharmacological Study
Correlative studies
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Patients Who Demonstrate Complete or Partial Response
Timepoint [1] 0 0
26 cycles of chemotherapy - up to 3 years after enrollment
Primary outcome [2] 0 0
Number of Patients Who Demonstrate Early Progression
Timepoint [2] 0 0
Up to 180 days after enrollment
Secondary outcome [1] 0 0
Event-free Survival [EFS]
Timepoint [1] 0 0
Up to 3 years after study enrollment
Secondary outcome [2] 0 0
Overall Survival [OS]
Timepoint [2] 0 0
Up to 3 years after study enrollment
Secondary outcome [3] 0 0
Number of Patients With Toxic Events After 2 Dose Reductions
Timepoint [3] 0 0
While receiving protocol therapy up to 3 years after study enrollment
Secondary outcome [4] 0 0
Pharmacokinetic Parameters of Lenalidomide
Timepoint [4] 0 0
Between days 5-21 of course 1 and each dose reduction
Secondary outcome [5] 0 0
Magnetic Resonance Imaging Sequence
Timepoint [5] 0 0
Up to 3 years

Eligibility
Key inclusion criteria
* Patients must have a body surface area (BSA) >= 0.4 m^2 at the time of study enrollment
* Patients must have a pilocytic astrocytoma or optic pathway glioma that has relapsed, progressed, or become refractory to conventional therapy; patients with neurofibromatosis (NF-1) are eligible
* Patients must have histologic verification of malignancy; histologic confirmation for patients with optic pathway gliomas will not be required
* Patients must have measurable residual disease, defined as tumor that is measurable in two perpendicular diameters on magnetic resonance imaging (MRI); for a lesion to be considered measurable, it must be at least twice the slice thickness on MRI (i.e. visible on more than one slice)
* To document the degree of residual tumor, the following must be obtained:

* All patients must have a brain MRI with and without contrast (gadolinium) within 1 week prior to study enrollment; for patients on steroids, baseline MRI scans must be performed after at least 1 week at a stable or decreasing dose of steroids
* All patients with a history of spinal or leptomeningeal disease, and those patients with symptoms suspicious of spinal disease, must have a spine MRI with and without contrast (gadolinium) performed within 2 weeks prior to study enrollment
* Patients must have a Lansky or Karnofsky performance status score of >= 60%; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
* Patients must have been treated with at least one prior treatment regimen that included carboplatin; patients who have received prior radiation therapy for this tumor are eligible
* Patients must have recovered (to Common Toxicity Criteria [CTC] version [v.]4.0 =< grade 1 unless indicated below) from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, with the exception of alopecia, weight changes and grade I or II lymphopenia

* Myelosuppressive chemotherapy: must not have received within 3 weeks of entry onto this study (6 weeks if prior nitrosourea or mitomycin-C)
* Biologic (anti-neoplastic agent): at least 7 days after the last dose of a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
* Immunotherapy: at least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines
* Monoclonal antibodies: at least 3 half-lives of the antibody after the last dose of a monoclonal antibody
* Radiation therapy (RT): patients must have had their last fraction of craniospinal RT >= 6 months prior to study entry and their last fraction of focal RT >= 4 weeks prior to study entry; if the lesion used for on-study criteria is in the radiation field, there must be evidence of tumor progression after radiation therapy was completed
* Study specific limitations on prior therapy:

* Patients who have received thalidomide are eligible if all acute thalidomide-related toxicity has resolved
* Patients must not have received lenalidomide previously
* Growth factor(s): must not have received within 2 weeks of entry onto this study
* Steroids: patients who are receiving corticosteroids must be on a stable or decreasing dose for at least 1 week prior to baseline MRI
* Peripheral absolute neutrophil count (ANC) >= 1,000/uL
* Platelet count >= 100,000/uL (transfusion independent)
* Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions)
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/m^2 OR a serum creatinine based on age/gender as follows:

* 0.4 mg/dL (1 month to < 6 months of age)
* 0.5 mg/dL (6 months to < 1 year of age)
* 0.6 mg/dL (1 to < 2 years of age)
* 0.8 mg/dL (2 to < 6 years of age)
* 1.0 mg/dL (6 to 10 years of age)
* 1.2 mg/dL (10 to < 13 years of age)
* 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)
* 1.7 mg/dL (male) or 1.4 mg/dL (female) (>= 16 years of age)
* Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 110 U/L; for the purpose of this study, the ULN for SGPT is 45 U/L
* Serum albumin >= 2 g/dL
* No evidence of dyspnea at rest and a pulse oximetry > 94% if there is clinical indication for determination
* Patients must be able to swallow intact capsules
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Minimum age
No limit
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Female patients who are pregnant are not eligible
* Lactating females are not eligible unless they have agreed not to breastfeed their infants while receiving protocol therapy and for 28 days after the last dose of lenalidomide
* Female patients of childbearing potential are not eligible unless they commit to complete abstinence or have been on 2 methods of birth control, including 1 highly effective method and 1 additional method at the same time (unless committing to complete abstinence of heterosexual intercourse) at least 28 days (4 weeks) prior to study enrollment; sexually active females must also agree to remain on 2 methods of birth control, during treatment (including during dose interruptions), and continuing for at least 28 days after the completion of protocol therapy; examples of methods of contraception are as follows:

* Highly effective methods (must use at least 1):

* Intrauterine device (IUD)
* Hormonal (prescription birth control pills, injections, implants)
* Tubal ligation
* Partner's vasectomy
* Additional effective methods:

* Male condom
* Diaphragm
* Cervical cap The two methods of birth control requirement applies to all sexually active females unless they have undergone a hysterectomy or bilateral oophorectomy
* Female patients of childbearing potential (including those who commit to complete abstinence) are not eligible unless they agree to ongoing pregnancy testing and counseling every 28 days about pregnancy precautions and risks of fetal exposure
* Male patients of child fathering potential are not eligible unless they have agreed to use latex condoms during intercourse with a woman of childbearing potential while receiving treatment and for 28 days thereafter
* Patients with a history of thromboembolism unrelated to a central line, or patients with a known predisposition syndrome for thromboembolism are not eligible
* Patients who have an uncontrolled or untreated infection are not eligible
* Patients with known overt cardiac disease, including but not limited to a history of myocardial infarction, severe or unstable angina, clinically significant peripheral vascular disease, grade 2 or greater heart failure, or serious and inadequately controlled cardiac arrhythmia are not eligible
* Patients with a significant systemic illness that is not well-controlled in the opinion of the treating physician are not eligible

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/07/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/12/2023
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
QLD,VIC,WA
Recruitment hospital [1] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [2] 0 0
Royal Children's Hospital-Brisbane - Herston
Recruitment hospital [3] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [4] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [5] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment postcode(s) [4] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Colorado
Country [6] 0 0
United States of America
State/province [6] 0 0
Connecticut
Country [7] 0 0
United States of America
State/province [7] 0 0
Delaware
Country [8] 0 0
United States of America
State/province [8] 0 0
District of Columbia
Country [9] 0 0
United States of America
State/province [9] 0 0
Florida
Country [10] 0 0
United States of America
State/province [10] 0 0
Georgia
Country [11] 0 0
United States of America
State/province [11] 0 0
Hawaii
Country [12] 0 0
United States of America
State/province [12] 0 0
Idaho
Country [13] 0 0
United States of America
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Illinois
Country [14] 0 0
United States of America
State/province [14] 0 0
Indiana
Country [15] 0 0
United States of America
State/province [15] 0 0
Iowa
Country [16] 0 0
United States of America
State/province [16] 0 0
Kentucky
Country [17] 0 0
United States of America
State/province [17] 0 0
Louisiana
Country [18] 0 0
United States of America
State/province [18] 0 0
Maryland
Country [19] 0 0
United States of America
State/province [19] 0 0
Massachusetts
Country [20] 0 0
United States of America
State/province [20] 0 0
Michigan
Country [21] 0 0
United States of America
State/province [21] 0 0
Minnesota
Country [22] 0 0
United States of America
State/province [22] 0 0
Mississippi
Country [23] 0 0
United States of America
State/province [23] 0 0
Missouri
Country [24] 0 0
United States of America
State/province [24] 0 0
Nebraska
Country [25] 0 0
United States of America
State/province [25] 0 0
Nevada
Country [26] 0 0
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New Hampshire
Country [27] 0 0
United States of America
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New Jersey
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New Mexico
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New York
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North Carolina
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Ohio
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Oklahoma
Country [33] 0 0
United States of America
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Oregon
Country [34] 0 0
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Pennsylvania
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South Carolina
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South Dakota
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United States of America
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Tennessee
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Texas
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Utah
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Virginia
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Washington
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United States of America
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Wisconsin
Country [43] 0 0
Canada
State/province [43] 0 0
British Columbia
Country [44] 0 0
Canada
State/province [44] 0 0
Nova Scotia
Country [45] 0 0
Canada
State/province [45] 0 0
Ontario
Country [46] 0 0
Canada
State/province [46] 0 0
Quebec
Country [47] 0 0
New Zealand
State/province [47] 0 0
Auckland
Country [48] 0 0
New Zealand
State/province [48] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Government body
Name
National Cancer Institute (NCI)
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Katherine E Warren
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01553149