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Lenalidomide in Treating Young Patients With Relapsed or Refractory Solid Tumors or Myelodysplastic Syndromes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00104962
Recruitment Status : Completed
First Posted : March 4, 2005
Last Update Posted : June 11, 2014
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Brief Summary:
This phase I trial is studying the side effects and best dose of lenalidomide in treating young patients with relapsed or refractory solid tumors or myelodysplastic syndromes. Lenalidomide may stop the growth of solid tumors or myelodysplastic syndromes by blocking blood flow to the cancer. It may also stimulate the immune system in different ways and stop cancer cells from growing.

Condition or disease Intervention/treatment Phase
Childhood Myelodysplastic Syndromes de Novo Myelodysplastic Syndromes Previously Treated Myelodysplastic Syndromes Refractory Anemia Refractory Anemia With Excess Blasts Refractory Anemia With Ringed Sideroblasts Refractory Cytopenia With Multilineage Dysplasia Secondary Myelodysplastic Syndromes Unspecified Childhood Solid Tumor, Protocol Specific Drug: lenalidomide Phase 1

Detailed Description:


I. Determine the maximum tolerated dose and recommended phase II dose of lenalidomide in pediatric patients with relapsed or refractory solid tumors.

II. Determine the toxic effects of this drug in these patients. III. Determine the pharmacokinetics of this drug in these patients.


I. Determine, preliminarily, the feasibility of administering this drug to pediatric patients with relapsed or refractory myelodysplastic syndromes.

II. Determine, preliminarily, the antitumor activity of this drug in both patient populations.

III. Determine immunologic changes in patients treated with this drug.

OUTLINE: This is a dose-escalation, multicenter study. Patients are stratified according to diagnosis (solid tumor vs myelodysplastic syndromes [MDS]).

Patients receive oral lenalidomide once daily on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients with solid tumors receive escalating doses of lenalidomide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose-limiting toxicity. Patients with MDS receive a fixed dose (do not undergo dose escalation) of lenalidomide. After completion of study treatment, patients are followed annually.

PROJECTED ACCRUAL: A total of 24 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of CC-5013 (Lenalidomide NSC# 703813) in Pediatric Patients With Relapsed/Refractory Solid Tumors or Myelodysplastic Syndrome
Study Start Date : March 2005
Actual Primary Completion Date : June 2009
Actual Study Completion Date : June 2009

Arm Intervention/treatment
Experimental: Treatment (lenalidomide)
Patients receive oral lenalidomide once daily on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Drug: lenalidomide
Given orally
Other Names:
  • CC-5013
  • IMiD-1
  • Revlimid

Primary Outcome Measures :
  1. Maximum tolerated dose of lenolidomide defined as the maximum dose at which fewer than one-third of patients experience DLT [ Time Frame: 28 days ]
    Graded using the CTCAE version 3.0.

Secondary Outcome Measures :
  1. Overall response assessed using RECIST criteria [ Time Frame: Up to 30 days after completion of study treatment ]
    A patient will be considered to have responded if she or he demonstrates either a bone marrow or cytogenetic response. Each patient will be classified according to the maximum response of the two criteria, where the classification from maximum to minimum will be: CR, PR or nonresponse.

  2. Adverse events defined using the NCI CTCAE version 3.0 [ Time Frame: Up to 30 days after completion of study treatment ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of 1 of the following:

    • Histologically confirmed solid tumor

      • No brain tumors
    • Myelodysplastic syndromes (MDS)

      • No refractory anemia with excess blasts in transformation or other forms of acute myeloid leukemia (AML)
      • No FAB diagnosis of refractory anemia with excess blasts in transition and other forms of AML
      • Newly diagnosed MDS with chromosome 5q abnormalities
  • Relapsed or refractory disease including relapse after stem cell transplantation
  • Measurable or evaluable disease (solid tumor patients only)
  • No known curative or life-prolonging therapy exists
  • No bone marrow involvement by tumor (solid tumor patients only)
  • No CNS tumors
  • Performance status - Karnofsky 50-100% (for patients > 10 years of age)
  • Performance status - Lansky 50-100% (for patients ≤ 10 years of age)
  • Absolute neutrophil count ≥ 1,000/mm^3 (for patients with solid tumors)
  • Platelet count ≥ 100,000/mm^3 (30,000 for patients with MDS)

    • Only patients with MDS may receive transfusions to support platelet counts
  • Hemoglobin ≥ 8.0 g/dL (transfusions allowed)
  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • ALT ≤ 110*
  • Albumin ≥ 2 g/dL
  • Creatinine clearance OR radioisotope glomerular filtration rate ≥ 70 mL/min
  • Creatinine based on age as follows:

    • Creatinine ≤ 0.8 mg/dL (for patients ≤ 5 years of age)
    • Creatinine ≤ 1 mg/dL (for patients 6 to 10 years of age)
    • Creatinine ≤ 1.2 mg/dL (for patients 11 to 15 years of age)
    • Creatinine ≤ 1.5 mg/dL (for patients over 15 years of age)
  • No parent or sibling with a known history of venous thrombosis before the age of 50 OR arterial thrombosis before the age of 40
  • No thromboembolic event except catheter-related thrombosis
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective double-method contraception 4 weeks before, during, and for ≥ 4 weeks after completion of study treatment
  • Body surface area ≥ 0.4m^2
  • No uncontrolled infection
  • No active graft-vs-host disease from prior stem cell transplant or rescue
  • Recovered from prior immunotherapy
  • At least 1 week since prior biologic agents
  • At least 1 week since prior hematologic growth factors (2 weeks for pegfilgrastim)
  • At least 3 months since prior stem cell transplant or rescue (without total body irradiation [TBI])
  • Prior thalidomide allowed
  • No other concurrent immunotherapy
  • No other concurrent biologic therapy
  • More than 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas) and recovered
  • No concurrent chemotherapy
  • Concurrent dexamethasone allowed provided the dose has been either decreasing or stable for the past 7 days
  • See Biologic therapy
  • Recovered from prior radiotherapy
  • At least 2 weeks since prior local palliative (small port) radiotherapy
  • At least 6 months since prior TBI, craniospinal radiotherapy, or radiotherapy to ≥ 50% of the pelvis
  • At least 6 weeks since other prior substantial bone marrow radiotherapy
  • No concurrent radiotherapy
  • No other concurrent investigational drugs or agents
  • No other concurrent anticancer agents

Information from the National Library of Medicine

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 identifier (NCT number): NCT00104962

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United States, California
COG Phase I Consortium
Arcadia, California, United States, 91006-3776
Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: Stacey Berg COG Phase I Consortium
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT00104962    
Obsolete Identifiers: NCT00269711
Other Study ID Numbers: NCI-2012-01820
NCI-2012-01820 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
ADVL0319 ( Other Identifier: COG Phase I Consortium )
ADVL0319 ( Other Identifier: CTEP )
U01CA097452 ( U.S. NIH Grant/Contract )
First Posted: March 4, 2005    Key Record Dates
Last Update Posted: June 11, 2014
Last Verified: December 2012
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Anemia, Refractory
Anemia, Refractory, with Excess of Blasts
Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents