NCT01496495

Brief Summary

This is a Phase 1 study during which patients with low or intermediate-1 risk myelodysplastic syndromes (MDS) will receive investigational study drug ARRY-614. This study has 2 parts. In the first part, patients will receive increasing doses of study drug in order to achieve the highest dose of the study drug possible that will not cause unacceptable side effects. Approximately 50 patients from the US will be enrolled in Part 1 (Completed). In the second part of the study, patients will receive the best dose of study drug determined from the first part of the study and will be followed to see what side effects and effectiveness the study drug has, if any, in treating the cancer. Approximately 30 patients from the US will be enrolled in Part 2 (Completed).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
71

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jan 2012

Typical duration for phase_1

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 28, 2011

Completed
23 days until next milestone

First Posted

Study publicly available on registry

December 21, 2011

Completed
11 days until next milestone

Study Start

First participant enrolled

January 1, 2012

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 18, 2015

Completed
Last Updated

September 9, 2020

Status Verified

September 1, 2020

Enrollment Period

2.9 years

First QC Date

November 28, 2011

Last Update Submit

September 4, 2020

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (3)

  • Establish the maximum tolerated dose (MTD) of study drug.

    Part 1, 9 months

  • Characterize the safety profile of the study drug in terms of adverse events, clinical laboratory tests and electrocardiograms.

    Part 1, 9 months; Part 2, 9 months

  • Characterize the pharmacokinetics (PK) of the study drug and a metabolite in terms of plasma concentrations.

    Part 1, 9 months; Part 2, 9 months

Secondary Outcomes (1)

  • Assess the efficacy of the study drug in terms of response, time to response, duration of response, overall survival, hematologic improvement and platelet transfusion independence/reduction.

    Part 1, 9 months; Part 2, 9 months

Study Arms (1)

ARRY-614

EXPERIMENTAL
Drug: ARRY-614, p38/Tie2 inhibitor; oral

Interventions

ARRY-614, p38/Tie2 inhibitor; oralDRUG

Part 1: multiple dose, escalating; Part 2: multiple dose, single schedule

ARRY-614

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of MDS by bone marrow biopsy.
  • International Prognostic Scoring System (IPSS) score of low or intermediate-1 risk MDS.
  • May have received prior therapy for MDS.
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1 or 2.
  • Adequate liver and renal function.
  • Additional criteria exist.

You may not qualify if:

  • History of bone marrow transplant.
  • Treatment for MDS other than transfusions or a stable dose (≥ 4 weeks) of hematopoietic growth factors on the day of the first dose of study drug.
  • Concomitant malignancies or previous malignancies with less than a 2-year disease-free interval at the time of enrollment.
  • Treatment with an investigational medicinal product that is not expected to be cleared by the first dose of study drug or that has demonstrated to have prolonged side effects.
  • Treatment with azacitidine or decitabine within 2 weeks prior to first dose of study drug.
  • Chronic use (\> 2 weeks) of greater than physiologic doses of corticosteroids (dose equivalent to \> 20 mg/day of prednisone) within 4 weeks prior to first dose of study drug.
  • Treatment with an immunomodulatory agent within 4 weeks prior to the first dose of study drug.
  • Additional criteria exist.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Emory University, Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Su H, Jiang M, Senevirathne C, Aluri S, Zhang T, Guo H, Xavier-Ferrucio J, Jin S, Tran NT, Liu SM, Sun CW, Zhu Y, Zhao Q, Chen Y, Cable L, Shen Y, Liu J, Qu CK, Han X, Klug CA, Bhatia R, Chen Y, Nimer SD, Zheng YG, Iancu-Rubin C, Jin J, Deng H, Krause DS, Xiang J, Verma A, Luo M, Zhao X. Methylation of dual-specificity phosphatase 4 controls cell differentiation. Cell Rep. 2021 Jul 27;36(4):109421. doi: 10.1016/j.celrep.2021.109421.

    PMID: 34320342DERIVED

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

pexmetinib

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 28, 2011

First Posted

December 21, 2011

Study Start

January 1, 2012

Primary Completion

December 1, 2014

Study Completion

March 18, 2015

Last Updated

September 9, 2020

Record last verified: 2020-09

Locations

US(4)