Graft vs Host DiseasePhase 3NCT00298324
Myfortic - Treatment for Extensive cGvHD
A Phase 3 study testing Myfortic and Prednisone and Cyclosporine in people with Graft vs Host Disease, run by European Society for Blood and Marrow Transplantation at 8 sites in 8 countries.
Trial summary
The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Graft vs Host Disease
- What is being tested?
- Myfortic and Prednisone and Cyclosporine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 8 sitesin 8 countries
- Study dates
- Sep 2006to Nov 2010
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age 18 - 60
- Any primary diagnosis requiring treatment by hematopoietic stem cell transplantation
- Recipient of a single allogeneic stem cell transplant (bone marrow or peripheral blood stem cells, or cord blood) minimum 80 days ago
- Received a graft from a related or an unrelated donor
- Conditioning regimen: Myeloablative or non-myeloablative
- Patients suffering a first episode of extensive chronic GvHD, without recurrent disease
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- The diagnosis of chronic GvHD requires the following:
- Distinction from acute GvHD
- Presence of at least one diagnostic clinical sign of chronic GvHD or presence of at least one distinctive sign confirmed by pertinent biopsy or other relevant diagnostic tests
Who cannot join
- Receiving a standard prophylaxis regimen for acute GvHD: CsA plus methotrexate, or CSA+MMF for NMA, or a T-cell depleted transplant
- Patient gives written informed consent prior to randomization
- Patient age less than 18 years or over 60 years.
- GvHD prophylaxis by tacrolimus plus methotrexate
- Delayed onset acute GvHD following NMA or DLI
- Second allogeneic stem cell transplant
Show all 12Show fewer
- Not the first episode of chronic GvHD needing systemic immunosuppressive therapy.
- Limited chronic GvHD (Seattle criteria, see Appendix 1)
- Uncontrolled systemic infection which in the opinion of the investigator is associated with an increased risk of the patient's death within 1 week of randomization
- In the opinion of the investigator, if the patient has significant medical or psychosocial problems or unstable disease status
- Pregnant or lactating females
- Known hypersensitivity to mycophenolic acid
Trial locations and contacts
8 sites in 8 countries.
8 sites in 8 countries
About this study
Official title: A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease
The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.
Read the detailed description
This clinical trial is a European, multi-center, randomized, double blinded placebo-controlled trial comparing CsA+PDN+MPA versus the reference treatment of CsA+PDN alone + placebo, in patients with extensive chronic GvHD. Randomization will be stratified according to:
- Platelet number (low versus high risk)
- Source of transplantable cells (marrow versus PBSC versus cord blood)
Patients not in progression at 6 weeks post randomization (progression defined as primary failure) will be evaluated for remission (complete or partial) at 3, 6, 9, \& 12 months post randomization
- Conditions
- Graft vs Host Disease
- Being tested
- Myfortic, Prednisone and Cyclosporine
- Keywords
- GvHD, graft versus host disease, extensive, myfortic
- 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.
MyforticDrug
1440mg twice daily
Prednisone and CyclosporineDrug
Prednisone and Cyclosporine given according to protocol. The drugs are tapered according to patient response
| Study group | Role | What they receive |
|---|---|---|
| Myfortic | Active comparator | Patients in this arm will receive Myfortic + Prednisone + Cyclosporine Receives: Drug: Myfortic |
| Standard Care/ Placebo | Other | In this arm patients will receive Prednisone + Cyclosporine + Placebo or Prednisone + Cyclosporine Receives: Drug: Prednisone and Cyclosporine |
What is being measured
The primary outcome is the main question the study is designed to answer.
To test whether the addition of Myfortic improves the efficacy of prednisone plus cyclosporine for treatment of newly diagnosed chronic GvHD, as defined by the proportion of patients with efficacy success at 1 year after enrollment.
Measured: 1 year
Secondary outcomes (6)
The hazard rates of efficacy success between the two arms. Loss of donor chimerism or recurrent malignancy before secondary systemic therapy and before discontinuation of all immunosuppressive meds will be treated as competing risks.
Measured: 1 year
efficacy failure, and treatment failure defined as efficacy failure or premature discontinuation of study-drug administration due to toxicity
Measured: 1 year
survival without recurrent malignancy
Measured: 1 year
Overall survival
Measured: 1 year
cumulative incidence of secondary systemic treatment for cGvHD before recurrent malignancy
Measured: 1 year
the cumulative incidence of death without recurrent or malignancy
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2006 (estimated)
- Study startSep 2006
- Primary completionNov 2009
- Study completionNov 2010
Why it stopped: Due to slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Collaborators
- Novartis
- Investigators
- Gérard Socié, Study chair, Hôptial St Louis, Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00298324
- Sponsor's protocol ID
- EudraCT 2005-006178-86
- Other IDs
- EBMT-LE-0601
- Status last verified
- Apr 2015
- First submitted
- 1 Mar 2006
- Last updated
- 3 Apr 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT00298324
- Acronym / sponsor ID
- EudraCT 2005-006178-86
- Conditions
- Graft vs Host Disease
- Keywords
- GvHD, graft versus host disease, extensive, myfortic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to slow accrual
- Enrolment
- 34 participants (actual)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Collaborators
- Novartis
- Study start
- Sep 2006
- Primary completion
- Nov 2009 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 2 Mar 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.
Detailed description
This clinical trial is a European, multi-center, randomized, double blinded placebo-controlled trial comparing CsA+PDN+MPA versus the reference treatment of CsA+PDN alone + placebo, in patients with extensive chronic GvHD. Randomization will be stratified according to:
- Platelet number (low versus high risk)
- Source of transplantable cells (marrow versus PBSC versus cord blood)
Patients not in progression at 6 weeks post randomization (progression defined as primary failure) will be evaluated for remission (complete or partial) at 3, 6, 9, \& 12 months post randomization
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Myfortic | Active comparator | Drug: MyforticPatients in this arm will receive Myfortic + Prednisone + Cyclosporine |
| Standard Care/ Placebo | Other | Drug: Prednisone and CyclosporineIn this arm patients will receive Prednisone + Cyclosporine + Placebo or Prednisone + Cyclosporine |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Age 18 - 60
- Any primary diagnosis requiring treatment by hematopoietic stem cell transplantation
- Recipient of a single allogeneic stem cell transplant (bone marrow or peripheral blood stem cells, or cord blood) minimum 80 days ago
- Received a graft from a related or an unrelated donor
- Conditioning regimen: Myeloablative or non-myeloablative
Key exclusion criteria (12 listed)
- Receiving a standard prophylaxis regimen for acute GvHD: CsA plus methotrexate, or CSA+MMF for NMA, or a T-cell depleted transplant
- Patient gives written informed consent prior to randomization
- Patient age less than 18 years or over 60 years.
- GvHD prophylaxis by tacrolimus plus methotrexate
- Delayed onset acute GvHD following NMA or DLI
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 |
|---|---|---|
| Myfortic1440mg twice daily | Drug | Myfortic |
| Prednisone and CyclosporinePrednisone and Cyclosporine given according to protocol. The drugs are tapered according to patient response | Drug | Standard Care/ Placebo |
Primary outcome
To test whether the addition of Myfortic improves the efficacy of prednisone plus cyclosporine for treatment of newly diagnosed chronic GvHD, as defined by the proportion of patients with efficacy success at 1 year after enrollment.
Time frame: 1 year
6 secondary outcomes
The hazard rates of efficacy success between the two arms. Loss of donor chimerism or recurrent malignancy before secondary systemic therapy and before discontinuation of all immunosuppressive meds will be treated as competing risks.
Time frame: 1 year
efficacy failure, and treatment failure defined as efficacy failure or premature discontinuation of study-drug administration due to toxicity
Time frame: 1 year
survival without recurrent malignancy
Time frame: 1 year
Overall survival
Time frame: 1 year
cumulative incidence of secondary systemic treatment for cGvHD before recurrent malignancy
Time frame: 1 year
the cumulative incidence of death without recurrent or malignancy
Time frame: 1 year
Results and safety
The study was stopped early (reason given: Due to slow accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2015.
- sponsor's websiteLink 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.
Gérard Socié study chair
Hôptial St Louis, Paris
8 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Myfortic and Prednisone and Cyclosporine in people with Graft vs Host Disease, run by European Society for Blood and Marrow Transplantation at 8 sites in 8 countries.
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 9 inclusion and 12 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: stopped early, as of the sponsor's last update on 3 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in France, Germany, Italy, Netherlands, Spain and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Due to slow accrual). Results are not available in the source record.
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?
To test whether the addition of Myfortic improves the efficacy of prednisone plus cyclosporine for treatment of newly diagnosed chronic GvHD, as defined by the proportion of patients with efficacy success at 1 year after enrollment. (time frame: 1 year). 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 3 Apr 2015.