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Phase I Dose Escalation Trial of Bortezomib in Combination With Thalidomide in Patients With Myelodysplasia

Phase 1
18 Years
Not Enrolling
Myelodysplastic Syndrome

Thank you

Trial Information

Phase I Dose Escalation Trial of Bortezomib in Combination With Thalidomide in Patients With Myelodysplasia

Initial studies using Velcade in myelodysplasia with early results demonstrating that 35%
had a partial response and 25% had stable disease. The combination of Velcade and
thalidomide has been studied in patients with multiple myeloma, but not in patients with
myelodysplasia. The CRR in the MM patients was 22%, with a good safety profile.

This is a phase 1, prospective, open-label, dose escalation study to evaluate the DLT and
MTD of velcade with given in combination with thalidomide in patients with myelodysplasia.
Treatment will be given as an outpatient. Patients will receive 4 days of Velcade (days 1,
4, 8, 11) and 21 days of thalidomide 50 mg/day for each 21 day cycle. There will be 3
cohorts of 3-6 patients each, plus 10 additional patients. The tree dose levels ill be 0.7,
1.0 and 1.3 mg/m2. Patients may continue to be treated up to 6 cycles. Cycles 2-6 will
start within 2 weeks of completion of the previous cycle. Disease response will be
evaluated after cycle 3 and 6.

Inclusion Criteria:

- Myelodysplastic syndrome with a IPSS score of 0.5 or greater

- May have had prior chemo/radiotherapy for another malignancy or myelodysplasia

- ECOG performance status of 0-2

- Life expectancy greater than 3 months

- Total bilirubin
- ALT and AST
- Calculated creatinine clearance > 30 ml/min

- Use of appropriate method of contraception during the study

- ANC > 0.5 x 10(9)

- Platelet count > 30 x 10(9)

- Consideration of treatment with 5 azacytidine is encouraged by not required

Exclusion Criteria:

- Ejection fraction < 40%

- myocardial infarction within 6 months of enrollment of New York Heart Association
Class III or IV heart failure, uncontrolled angina, uncontrolled ventricular
arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction
system abnormalities

- Women who are pregnant or breast-feeding

- Major surgery within 4 weeks prior to enrollment

- >/= grade 2 peripheral neuropathy within 14 days prior to enrollment

- Uncontrolled intercurrent illness

- Serious medical or psychiatric illness that could potentially interfere with the
completion of treatment

- Hypersensitivity to bortezomib, boron, or mannitol

- Received an investigational drug within 14 days of enrollment

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The primary objective is to establish the maximally tolerated dose of bortezomib that can be administered with thalidomide in patient with myelodysplasia.

Principal Investigator

Karen Ballen, M

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital, Harvard University


United States: Food and Drug Administration

Study ID:




Start Date:

June 2005

Completion Date:

April 2007

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic syndrome
  • Myelodysplasia
  • MDS
  • Velcade
  • Thalidomide
  • Bortezomib
  • Myelodysplastic Syndromes
  • Preleukemia



Beth Israel Deaconess Medical CenterBoston, Massachusetts  02215
Massachusetts General HospitalBoston, Massachusetts  02114-2617
Dana Farber Cancer InstituteBoston, Massachusetts  02115