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A Phase I Study of a Combination of 5-azacitidine Followed by Lenalidomide in High-risk MDS or Relapsed/Refractory AML Patients With Cytogenetic Abnormalities Including -5 or Del(5q)

Phase 1
18 Years
Open (Enrolling)
Myelodysplastic Syndromes, Acute Myelogenous Leukemia

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Trial Information

A Phase I Study of a Combination of 5-azacitidine Followed by Lenalidomide in High-risk MDS or Relapsed/Refractory AML Patients With Cytogenetic Abnormalities Including -5 or Del(5q)

Cytogenetics are the main predictors of outcome in patients with AML. In fact, a monosomy 5
or del (5q) as single aberration are poor prognostic markers. Overall, the complete response
rate for conventionally treated patients with newly-diagnosed AML with chromosome 5
abnormalities is about 31% to 37 % and all patients rapidly relapse if not rescued by
allogeneic HSCT. The situation is almost similar in patients with high-risk MDS.Vidaza® has
been shown in clinical trials to achieve remission rates in about 29% (CR+PR) of the
patients while a total of 49% achieve improvement of blood counts.Revlimid® is also able to
achieve complete remissions in advanced MDS and even overt leukemia with or without
chromosome 5 abnormalities. Nevertheless, response rates are lower compared to low-risk MDS
(IPSS Low/INT-1). Therefore, Revlimid® seems to be too weak as a single agent, but a
promising compound for a combination therapy.

Inclusion Criteria:

- Understand and voluntarily sign an informed consent form.

- Age >=18 years at the time of signing the informed consent form.

- Able to adhere to the study visit schedule and other protocol requirements.

- Relapsed or refractory AML (>30% blasts, FAB classification)with karyotype
abnormalities involving monosomy 5 or del(5q) or MDS and t-MDS INT-2 or HIGH
according to IPSS classification with karyotype abnormalities involving monosomy 5 or
del(5q) either previously treated or untreated

- Not eligible for an immediate allogeneic HSCT (due to donor unavailability)

- All previous MDS or AML specific therapy with exception of corticosteroids not
exceeding doses of 10mg/day prednisone must have been discontinued at least 1 week
prior to study enrollment.

- Non-hematological toxicity (except alopecia) resulting from previous treatment must
be resolved to WHO CTC Grade ≤ 2.

- ECOG performance status of < 3 at study entry.

- Laboratory test results within these ranges:Serum creatinine <= 2.0 mg/dL, Total
bilirubin <= 3 x ULN, AST (SGOT) and ALT (SGPT) <= 3 x ULN

- Females of childbearing potential must agree to use a reliable form of contraception
or to practice complete abstinence from heterosexual intercourse during the following
time periods related to this study: 1) for at least 28 days before starting study
drug; 2) while participating in the study; and 3) for at least 28 days after
discontinuation from the study.

Exclusion Criteria:

- Any serious medical condition, laboratory abnormality, or psychiatric illness that
would prevent the subject from signing the informed consent form.

- Pregnant or breast feeding females. (Lactating females must agree not to breast feed
while on study).

- Any condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study.

- Known hypersensitivity to thalidomide, lenalidomide, 5-azacitidine or mannitol.

- Myocardial infarction within 6 months before study entry, New York Heart Association
Class III or IV heart failure, uncontrolled angina or severe uncontrolled ventricular

- The development of erythema nodosum if characterized by a desquamating rash while
taking thalidomide or similar drugs.

- Uncontrolled lung disease.

- Known positive for HIV or acute infectious hepatitis, type A, B or C.

- Participation in another clinical study in the 4 weeks prior to enrollment or during
this study.

Type of Study:


Study Design:

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

Outcome Measure:

Maximum tolerated dose (MTD) of Revlimid® (lenalidomide)in combination with Vidaza®(5-azacitidine)

Outcome Time Frame:

during first cycle of therapy

Safety Issue:


Principal Investigator

Uwe Platzbecker, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus


Germany: Federal Institute for Drugs and Medical Devices

Study ID:




Start Date:

June 2009

Completion Date:

July 2013

Related Keywords:

  • Myelodysplastic Syndromes
  • Acute Myelogenous Leukemia
  • monosomy 5
  • del5q
  • lenalidomide
  • azacitidine
  • Chromosome Aberrations
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia