Graft vs Host DiseasePhase 2NCT00770757
CC-4047 (Pomalidomide) for Graft vs. Host Disease
A Phase 2 study testing CC-4047 in people with Graft vs Host Disease, run by Washington University School of Medicine at 1 site in United States.
Trial summary
This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft vs Host Disease
- What is being tested?
- CC-4047
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2009to Oct 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 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.
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- 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.
- Other cancer less than or equal to 2 years prior study-entry except:
- Basal cell carcinoma of the skin,
- Squamous cell carcinoma of the skin,
- Carcinoma in situ of the cervix,
- Carcinoma in situ of the breast, or
- Prostate cancer (Tumor, Node, Metastasis [TNM] stage T1a or T1b)
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63122
About this study
Official title: A Phase 2, Open-Label, Single-Arm, Pilot Study of Safety and Efficacy of CC-4047 (Pomalidomide) in Patients With Advanced Chronic Graft-Versus-Host Disease Developing After Allogeneic Hematological Stem Cell Transplantation
This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease.
Read the detailed description
Chronic Graft vs. Host Disease is a major complication after allogeneic hematopoietic stem cell transplantation developing in 30 - 70% of patients. It is a multisystem alloimmune and autoimmune disorder with a negative impact on quality of life and functional status, increased need for extended immunosuppression and is the leading cause of late transplant related mortality.
CC-4047 is a novel immune modulatory drug that is a thalidomide analog with a 4,000 fold greater inhibition of TNF-α production related to thalidomide. Several features of CC-4047 suggest that this drug may be useful in treating chronic GVHD including in vitro suppression of TNF-α production, increasing Th1 and stimulation of IL-12 and sIL-Rα.
This study is an open-label, single-arm, pilot study of efficacy and safety of CC-4047 in patients with advanced chronic GvHD who failed to achieve a response with high-dose corticosteroids or second line systemic immunosuppressive therapy.
- Conditions
- Graft vs Host Disease
- Being tested
- CC-4047
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC-4047Drug
Also called Pomalidomide
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: CC-4047 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
-Toxicities will be graded according to the NCI CTCAE v3.0.
Measured: 30 days after last dose of CC-4047 or until resolution of event
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2008 (estimated)
- Study startFeb 2009
- Primary completionOct 2010
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- John F. DiPersio, M.D., Ph.D., Principal investigator, Washington Univerisity School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00770757
- Sponsor's protocol ID
- 08-1093 / 201106102
- Status last verified
- Jan 2016
- First submitted
- 7 Oct 2008
- Last updated
- 25 Feb 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Single-Arm, Pilot Study of Safety and Efficacy of CC-4047 (Pomalidomide) in Patients With Advanced Chronic Graft-Versus-Host Disease Developing After Allogeneic Hematological Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT00770757
- Acronym / sponsor ID
- 08-1093 / 201106102
- Conditions
- Graft vs Host Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- Feb 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 10 Oct 2008 (estimated)
- Results
- Posted 25 Feb 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will test the safety and effectiveness CC-4047 (pomalidomide) in patients with advanced, steroid refractory graft-versus-host disease.
Detailed description
Chronic Graft vs. Host Disease is a major complication after allogeneic hematopoietic stem cell transplantation developing in 30 - 70% of patients. It is a multisystem alloimmune and autoimmune disorder with a negative impact on quality of life and functional status, increased need for extended immunosuppression and is the leading cause of late transplant related mortality.
CC-4047 is a novel immune modulatory drug that is a thalidomide analog with a 4,000 fold greater inhibition of TNF-α production related to thalidomide. Several features of CC-4047 suggest that this drug may be useful in treating chronic GVHD including in vitro suppression of TNF-α production, increasing Th1 and stimulation of IL-12 and sIL-Rα.
This study is an open-label, single-arm, pilot study of efficacy and safety of CC-4047 in patients with advanced chronic GvHD who failed to achieve a response with high-dose corticosteroids or second line systemic immunosuppressive therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CC-4047 Arm | Experimental | Drug: CC-4047CC-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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (21 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CC-4047Also: Pomalidomide | Drug | CC-4047 Arm |
Primary outcome
Overall Response (Complete Response + Partial Response + Other)
Time frame: 1 year after last dose of CC-4047
* 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 secondary outcome
Safety as Measured by the Most Common Adverse Effects and Reasons for Dose Reductions or Study-discontinuation
Time frame: 30 days after last dose of CC-4047 or until resolution of event
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John F. DiPersio, M.D., Ph.D. principal investigator
Washington Univerisity School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CC-4047 in people with Graft vs Host Disease, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Feb 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response (Complete Response + Partial Response + Other) (time frame: 1 year after last dose of CC-4047). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2016.