A Phase I Study of MGCD0103 Given Twice Weekly in Patients With Leukemia or Myelodysplastic Syndromes

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2012
• Main ID: NCT00324194
• Date of registration: 08/05/2006
• Primary sponsor: MethylGene Inc.
• Public title: A Phase I Study of MGCD0103 Given Twice Weekly in Patients With Leukemia or Myelodysplastic Syndromes
• Scientific title: A Phase I Study of MGCD0103 Given as a Twice Weekly Oral Dose in Patients With Leukemia or Myelodysplastic Syndromes
• Date of first enrolment: February 2005
• Target sample size: 25
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00324194
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Countries of recruitment

Canada, United States

Contacts

• Name: Gregory Reid, MSc, MBA
• Affiliation: MethylGene Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients must have a diagnosis of one of the following:

- relapsed or refractory AML or ALL that has failed to respond to standard
therapy, has progressed despite standard therapy.

- relapsed or refractory Myelodysplastic Syndromes including CMML (chronic
myelomonocytic leukemia) or other myeloproliferative disorders provided there is
an MDS component.

- previously untreated AML or Myelodysplastic Syndromes in patients >60 years of
age who refused or are not candidates for induction chemotherapy

- Patients with relapsed or refractory CML that has failed to respond to imatinib
therapy or standard therapy, has progressed despite standard therapy, or for
which no standard therapy exists.

- ECOG performance status of 0, 1, or 2.

- Age =18 years.

- Laboratory requirements.

- Patients or their legal representative must be able to read, understand, and sign a
written informed consent (approved by the IRB/EC) prior to study entry.

Exclusion Criteria:

- Patients with other active malignancy except basal cell carcinoma or cervical
intraepithelial neoplasia (CIN / cervical in situ).

- Patients with suspicion of CNS involvement (tests are not required to rule out CNS
involvement in the absence of signs or symptoms).

- Pregnant or lactating women. Women of child-bearing potential must have a negative
serum pregnancy test documented within 7 days prior to registration on study.

- Patients and their partners, if either are of childbearing potential, not using
adequate birth control measures throughout the course of the study. Both men and
women enrolled on study must agree to use a medically acceptable effective form of
contraception during the study and for 90 days following the last dose of study
medication. An effective form of contraception is an oral contraceptive or a double
barrier method, such as condom with diaphragm.

- Patients with serious illnesses, medical conditions, or other medical history,
including laboratory results, which, in the investigator's opinion, would be likely
to interfere with a patient's participation in the study, or with the interpretation
of the results.

- Patients who have been treated with any investigational drug within 30 days prior to
study initiation (an investigational drug is one for which there is no approved
indication), or who are receiving concurrent treatment with other experimental drugs
or anti-cancer therapy.

- Known hypersensitivity to any of the components of MGCD0103.

- Known HIV or Hepatitis B or C (tests do not need to be performed to rule out any of
these infections).

- Any psychiatric illness/social situations that would, in the judgment of the
investigator, limit compliance with study requirements.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Leukemia

Myelodysplastic Syndromes

Intervention(s)

Drug: MGCD0103

Primary Outcome(s)

Safety and tolerability. [Time Frame: 1 year (anticipated)]

Secondary Outcome(s)

Clinical response. [Time Frame: 1 year (anticipated)]

Dose limiting toxicities [Time Frame: 1 year (anticipated)]

Pharmacodynamics (histone acetylation, biomarkers) [Time Frame: 1 year (anticipated)]

Pharmacokinetics [Time Frame: 1 year (anticipated)]

Secondary ID(s)

0103-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors