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Phase I Study of CC-5013 (Lenalidomide NSC# 703813, IND # 70116) in Pediatric Patients With Relapsed/Refractory Solid Tumors or Myelodysplastic Syndrome

Phase 1
1 Year
21 Years
Not Enrolling
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

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Trial Information

Phase I Study of CC-5013 (Lenalidomide NSC# 703813, IND # 70116) in Pediatric Patients With Relapsed/Refractory Solid Tumors or Myelodysplastic Syndrome


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

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.

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

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Maximum tolerated dose of lenolidomide defined as the maximum dose at which fewer than one-third of patients experience DLT

Outcome Description:

Graded using the CTCAE version 3.0.

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

Stacey Berg

Investigator Role:

Principal Investigator

Investigator Affiliation:

COG Phase I Consortium


United States: Food and Drug Administration

Study ID:




Start Date:

March 2005

Completion Date:

Related Keywords:

  • 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
  • Anemia
  • Anemia, Refractory
  • Anemia, Refractory, with Excess of Blasts
  • Myelodysplastic Syndromes
  • Preleukemia
  • Neoplasms



COG Phase I Consortium Arcadia, California  91006-3776