CC-4047 (Pomalidomide) for Graft vs. Host Disease

NCT00770757 | Completed Phase 2 Results

• 1 other identifier: 08-1093 / 201106102
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease.

Conditions

Graft vs Host Disease

Outcome Measures

Primary Outcomes (1)

• Overall Response (Complete Response + Partial Response + Other)

  * CR is defined as complete resolution in all of signs and symptoms at all affected organs and tissues * PR is defined as improvement in greater than or equal to 1 organ/tissue with no progression in any other affected organ/tissue * Improvement in chronic GvHD symptoms less than what meets the definition of a PR is defined as other * Progressive disease is defined as failure of therapy to control chronic GvHD despite increasing the dose of primary therapy or adding second line treatments * No response is defined as no change in disease.

  1 year after last dose of CC-4047

Secondary Outcomes (1)

• Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation

  30 days after last dose of CC-4047 or until resolution of event

Study Arms (1)

CC-4047 Arm

EXPERIMENTAL

CC-4047 2 mg orally every day for 1 course (12 weeks or 84 days). Every 28 days of treatment are considered as 1 cycle and every 3 cycles are are considered as 1 course of treatment. A total of 4 courses of treatment are planned (12 months).

Drug: CC-4047

Interventions

CC-4047 DRUG

Also known as: Pomalidomide

CC-4047 Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must be greater than or equal to 18 years of age at the time of consent.
• Must be able to adhere to the study visit schedule and other protocol requirements.
• Chronic graft versus host disease (GHVD) developing after allogeneic hematological stem cell transplantation diagnosed using NIH criteria for diagnosis and staging of chronic GvHD (including both "classic chronic GvHD" and "overlap syndrome")
• Must have moderate or severe chronic GvHD according to Global Staging System for Chronic GvHD or mild chronic GvHD with platelet count less than 100 x 109/L
• Must have failed to achieve response to high dose corticosteroid (average 0.5 mg/kg/day prednisone or equivalent for greater than or equal to 8 weeks), or have failed second line systemic immunosuppressive therapy.
• If taking corticosteroids at the time of enrollment, must be on stable or tapering schedule without corticosteroid pulses in the preceding 8 weeks.
• If taking secondary systemic immunosuppressive therapy at the time of enrolment, must be on stable or tapering schedule in the preceding 4 weeks.
• Karnofsky performance score (KPS) greater than or equal to 60%.
• Life expectancy greater than or equal to 3 months.
• Female of childbearing potential (FCBP) must agree to use two reliable forms of contraception simultaneously or to practice complete abstinence from heterosexual intercourse for at least 28 days before starting study drug, while participating in the study, and for at least 28 days after discontinuation from the study. The two methods of reliable contraception must include one highly effective method (i.e. intrauterine device (IUD), hormonal \[birth control pills, injections, or implants\], tubal ligation, partner's vasectomy) and one additional effective (barrier) method (i.e. latex condom, diaphragm, cervical cap). FCBP must be referred to a qualified provider of contraceptive methods if needed.
• FCBP must agree to pregnancy testing and contraceptive counseling every 28 days during the study. FCBP must also refrain from donating blood and/or egg while participating in the study and for at least 28 days after discontinuation from this study
• FCBP must have two negative pregnancy tests (sensitivity of at least 25 mIU/mL) prior to starting study drug. The first pregnancy test must be performed within 10-14 days prior to the start of study drug and the second pregnancy test must be performed within 24 hours prior to the start of study drug.
• Must agree to abstain from breastfeeding during study participation and for at least 28 days after study drug discontinuation.
• Male Subjects must agree to complete abstinence or to use a condom during sexual contact with with a pregnant female or a female of childbearing potential while participating in the study and for at least 90 days following study drug discontinuation even if he has undergone a successful vasectomy
• Must agree to counseling about sexual contact and the potential risks of fetal exposure to pomalidomide every 28 days.

You may not qualify if:

• Pregnant or lactating females.
• New immunosuppressive therapy started within the preceding 4 weeks.
• Extracorporeal photopheresis within the preceding 3 months.
• Hypersensitivity to any immune modulator drug (IMiD™).
• Unable to take prophylactic anticoagulation.
• Any condition which places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
• Acute, persistent, recurrent or late-onset acute GvHD defined by NIH criteria.
• Any of the following laboratory values at registration:
• absolute neutrophil count (ANC) less than 1.0 x 109/L,
• platelets less than 75 x 109/L, or
• creatinine clearance less than 50 mL/min (Cockroft-Gault formula).
• Uncontrolled infection requiring systemic antibiotics.
• Known human immunodeficiency virus (HIV), hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infection.
• Known uncontrolled arrhythmias or symptomatic heart disease or left ventricular ejection fraction less than 40% (an ECHO should be performed as clinically indicated)
• Recurrence of cancer for which the transplant was done except for presence of minimal residual disease by PCR.

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63122, United States

Location

Related Links

• Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

pomalidomide

Condition Hierarchy (Ancestors)

Immune System Diseases

Results Point of Contact

• Title: John F. DiPersio, M.D., Ph.D.
• Organization: Washington University School of Medicine

jdipersi@dom.wustl.edu

314-454-8603

Study Officials

• John F. DiPersio, M.D., Ph.D.

  Washington Univerisity School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 7, 2008
• First Posted: October 10, 2008
• Study Start: February 1, 2009
• Primary Completion: October 1, 2010
• Study Completion: October 1, 2011
• Last Updated: February 25, 2016
• Results First Posted: February 25, 2016

Record last verified: 2016-01

Locations

US (1)