NCT01309997

Brief Summary

This randomized phase II trial is evaluating how well imatinib mesylate works compared to rituximab in treating cutaneous sclerosis in patients with chronic graft- versus-host disease (GVHD). Both imatinib and rituximab have been reported to decrease skin thickening and improve skin and joint flexibility in people with cutaneous sclerosis due to chronic GVHD.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
72

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Mar 2011

Longer than P75 for phase_2

Geographic Reach
1 country

10 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

March 1, 2011

Completed
Same day until next milestone

Study Start

First participant enrolled

March 1, 2011

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 7, 2011

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
7 months until next milestone

Results Posted

Study results publicly available

June 15, 2016

Completed
Last Updated

June 15, 2016

Status Verified

May 1, 2016

Enrollment Period

3.8 years

First QC Date

March 1, 2011

Results QC Date

September 21, 2015

Last Update Submit

May 10, 2016

Conditions

Keywords

ImatinibRituximabChronic Graft-vs-Host Disease

Outcome Measures

Primary Outcomes (1)

  • Significant Clinical Response

    Assessed by decline in an affected area's skin score as measured with the Vienna Skin Scale (from 4 \[worst\] to 2, 3 to 1, or 2 to 0 \[best\]) without a concurrent increase of two or more points in another area OR by an increase in the range of motion of the shoulders, elbows or wrists by two points (in a 1-7 scale where 1 is worst and 7 is best) or of the ankles by one point (in a 1 to 4 scale where 1 is worst and 4 is best) without a concurrent worsening in another area.

    6 months

Secondary Outcomes (6)

  • Patients Who Were Able to Taper Corticosteroids

    6 months

  • Cumulative Incidence of Treatment Failure

    6 months

  • Number of Patients Achieving Improvement in Cutaneous Sclerosis

    6 months

  • Baseline Histopathologic Score in the Two Treatment Arms

    Enrollment

  • Patients With Any Percentage Decline in Any Grade of Sclerosis Without Increase in Percentage of Higher Grades of Sclerosis in Other Areas on the Vienna Skin Scale

    6 months

  • +1 more secondary outcomes

Study Arms (2)

Arm I (enzyme inhibitor)

EXPERIMENTAL

Patients receive imatinib mesylate PO QD for 6 months in the absence of progression of sclerosis or unacceptable toxicity.

Drug: imatinib mesylate

Arm II (monoclonal antibody)

EXPERIMENTAL

Patients receive rituximab IV on days 1, 8, 15, and 22. A second treatment cycle is repeated at 3 months for a total of 8 doses of rituximab in the absence of progression of sclerosis or unacceptable toxicity.

Biological: rituximab

Interventions

Given PO

Also known as: CGP 57148, Gleevec, Glivec
Arm I (enzyme inhibitor)
rituximabBIOLOGICAL

Given IV

Also known as: IDEC-C2B8, IDEC-C2B8 monoclonal antibody, Mabthera, MOAB IDEC-C2B8, Rituxan
Arm II (monoclonal antibody)

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis within the past 18 months of cutaneous sclerosis after hematopoietic cell transplant (HCT) with sclerotic skin, morphea, myofascial involvement or joint contractures; must have a score of 2 or greater on the Vienna skin scale in any area, or a range-of-motion (ROM) score of 5 or less at the shoulder, elbow or wrist, or 3 or less at the ankle
  • No medication added for the treatment of graft versus host disease (GVHD) within the past 4 weeks
  • Receiving corticosteroids at a dose greater than required for treatment of adrenal insufficiency, unless the physician documents why steroids are contraindicated
  • Age 2-99 years
  • Karnofsky performance status \>= 60% at enrollment
  • All females of childbearing potential must have a negative serum or urine pregnancy test =\< 7 days prior to starting study therapy
  • All females of childbearing potential must agree to use a form of Food and Drug Administration (FDA) approved contraception from enrollment to one month after study treatment ends
  • Subject has the ability to understand and willingness to sign a written informed consent document

You may not qualify if:

  • Total bilirubin \> 1.5x upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2.5 x ULN
  • Renal insufficiency (serum creatinine \> 2.0 mg/dl)
  • Platelets \< 30,000/ul or absolute neutrophil count \< 1500/ul
  • Known hypersensitivity to rituximab or other anti-B cell antibodies
  • Known imatinib intolerance or allergy
  • Evidence of any active viral, bacterial, or fungal infection that is progressive despite appropriate treatment
  • Hepatitis B surface antigen positive
  • Hepatitis B core antibody positive, unless hepatitis B virus (HBV) deoxyribonucleic acid (DNA) is undetectable
  • Hepatitis C antibody positive, unless hepatitis C virus (HCV) ribonucleic acid (RNA) is undetectable
  • Pregnant, lactating, or planning a pregnancy while in the study
  • Distal leg skin score 3 or higher as the only manifestation of sclerosis
  • Prior treatment of chronic GVHD with imatinib, rituximab, or any other monoclonal B-cell antibody (e.g. ofatumumab)
  • Receipt of imatinib within the previous 6 months for any indication
  • Receipt of any monoclonal B-cell antibody (e.g. rituximab, ofatumumab) within the previous 12 months for any indication
  • +7 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Stanford University Hospitals and Clinics

Stanford, California, 94305, United States

Location

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, 33612, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Weill Cornell Medical College

New York, New York, 10065, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

Froedtert and the Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

  • Arai S, Pidala J, Pusic I, Chai X, Jaglowski S, Khera N, Palmer J, Chen GL, Jagasia MH, Mayer SA, Wood WA, Green M, Hyun TS, Inamoto Y, Storer BE, Miklos DB, Shulman HM, Martin PJ, Sarantopoulos S, Lee SJ, Flowers ME. A Randomized Phase II Crossover Study of Imatinib or Rituximab for Cutaneous Sclerosis after Hematopoietic Cell Transplantation. Clin Cancer Res. 2016 Jan 15;22(2):319-27. doi: 10.1158/1078-0432.CCR-15-1443. Epub 2015 Sep 16.

MeSH Terms

Conditions

Graft vs Host DiseaseScleroderma, Systemic

Interventions

Imatinib MesylateRituximab

Condition Hierarchy (Ancestors)

Immune System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPyrimidinesAntibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Results Point of Contact

Title
Stephanie J. Lee MD MPH
Organization
FHCRC

Study Officials

  • Stephanie Lee

    Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

March 1, 2011

First Posted

March 7, 2011

Study Start

March 1, 2011

Primary Completion

December 1, 2014

Study Completion

December 1, 2015

Last Updated

June 15, 2016

Results First Posted

June 15, 2016

Record last verified: 2016-05

Data Sharing

IPD Sharing
Will share

De-identified patient data and samples may be available with the appropriate approvals. Contact the PI for information.

Locations