Graft vs Host DiseasePhase 2NCT00388362
Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease
A Phase 2 study testing Sirolimus and Prednisone in people with Graft vs Host Disease, run by Stanford University at 1 site in United States.
Trial summary
To study the effectiveness of an immunosuppressive drug sirolimus, in the treatment of chronic graft versus host disease in combination with prednisone. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Graft vs Host Disease
- What is being tested?
- Sirolimus and Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to Aug 2012
- 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 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥ 13 years
- Weight ≥ 40 kg.
- Biopsy or clinical presentation diagnostic of chronic GVHD >100 days following allogeneic bone marrow/peripheral blood/umbilical cord blood transplantation that has failed prior corticosteroid therapy or corticosteroid taper. In the event that histological confirmation poses undue risk, clinical evaluation is sufficient.
- Women of child-bearing potential must have a negative pregnancy test before sirolimus administration and agree to use a medically acceptable contraceptive throughout the treatment period until 3 months after discontinuation of sirolimus.
- Any woman becoming pregnant during the treatment period must discontinue the use of sirolimus.
- Absolute neutrophil count (ANC) > 1000/mm³, unless receiving G-CSF to maintain neutrophil count > 500/mm³.
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- At the time of initiating sirolimus the cyclosporine trough level is recommended to be < 100 mg/dl and FK506 level is recommended to be < 5 mg/dl. FK506 or cyclosporine is to be discontinued soon after initiation of sirolimus.
- Karnofsky performance score ≥ 50 during pre-study screening.
- Written, signed, and dated informed consent
Who cannot join
- Uncontrolled systemic infection
- Unstable disease states (i.e., hepatic failure, ventilatory-dependent respiratory failure, etc.)
- Serum creatinine ≥ 3.0 mg/dL
- Platelet count ≤ 50,000/mm³
- History of Post-transplant microangiopathic hemolytic anemia
- Uncontrolled hyperlipidemia
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- Use of any investigational drug within 4 weeks of entry into the study
- Use of methotrexate or antibody therapies within 24 hours of sirolimus administration
- Inability to tolerate oral therapy for any reason
- Evidence of infiltrate, cavitation, or consolidation on chest x-ray during pre-study screening
- Known hypersensitivity to macrolide antibiotics
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: A Phase II Trial of Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease
To study the effectiveness of an immunosuppressive drug sirolimus, in the treatment of chronic graft versus host disease in combination with prednisone.
Read the detailed description
The purpose of this trial is to study the effectiveness of an immunosuppressive drug, sirolimus, in the treatment of chronic graft versus host disease in combination with prednisone. Graft versus host disease (GVHD) is a common complication in patients who have received blood or marrow transplantation from a related or unrelated donor. Chronic GVHD occurs approximately 100 days after transplantation and is the result of the donor immune system recognizing the patient's tissues as foreign and creating harmful effects on the patient's organs. We hope the use of sirolimus will decrease the significant disabling effects and deaths caused by chronic GVHD.
- Conditions
- Graft vs Host Disease
- Being tested
- Sirolimus, Prednisone
- Keywords
- GVHD
- 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.
SirolimusDrug
Also called Rapamune
Patients will receive sirolimus at 2 mg/day orally with monitoring of trough drug levels weekly for 2 weeks to achieve trough drug levels 7-12 ng/ml. Along with prednisone therapy.
PrednisoneDrug
Also called Deltasone
Prednisone therapy will remain at the dose the patient received at the time sirolimus was begun. Withdrawal of prednisone will began after first evidence of improvement of chronic GVHD.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus Therapy | Experimental | Administration of Sirolimus and Prednisone Receives: Drug: Sirolimus; Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Activity
Determined by discontinuation of immunosuppression with resolution of all reversible CGVHD manifestations. Evaluated at 2 years after enrollment
Measured: 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus
Secondary outcomes (1)
Overall Survival
Administration of Sirolimus and Prednisone
Measured: 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2006 (estimated)
- Study startNov 2005
- Primary completionNov 2010
- Study completionAug 2012
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 Stanford UniversityThe 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
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Laura Johnston, Stanford University
- Investigators
- Laura Johnston, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00388362
- Sponsor's protocol ID
- IRB-3587
- Other IDs
- 96589, BMT175
- Status last verified
- Mar 2017
- First submitted
- 12 Oct 2006
- Last updated
- 21 Apr 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease
- ClinicalTrials.gov ID
- NCT00388362
- Acronym / sponsor ID
- IRB-3587
- Conditions
- Graft vs Host Disease
- Keywords
- GVHD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- Nov 2010 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 16 Oct 2006 (estimated)
- Results
- Posted 21 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To study the effectiveness of an immunosuppressive drug sirolimus, in the treatment of chronic graft versus host disease in combination with prednisone.
Detailed description
The purpose of this trial is to study the effectiveness of an immunosuppressive drug, sirolimus, in the treatment of chronic graft versus host disease in combination with prednisone. Graft versus host disease (GVHD) is a common complication in patients who have received blood or marrow transplantation from a related or unrelated donor. Chronic GVHD occurs approximately 100 days after transplantation and is the result of the donor immune system recognizing the patient's tissues as foreign and creating harmful effects on the patient's organs. We hope the use of sirolimus will decrease the significant disabling effects and deaths caused by chronic GVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus Therapy | Experimental | Drug: Sirolimus, Drug: PrednisoneAdministration of Sirolimus and Prednisone |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Age ≥ 13 years
- Weight ≥ 40 kg.
- Biopsy or clinical presentation diagnostic of chronic GVHD >100 days following allogeneic bone marrow/peripheral blood/umbilical cord blood transplantation that has failed prior corticosteroid therapy or corticosteroid taper. In the event that histological confirmation poses undue risk, clinical evaluation is sufficient.
- Women of child-bearing potential must have a negative pregnancy test before sirolimus administration and agree to use a medically acceptable contraceptive throughout the treatment period until 3 months after discontinuation of sirolimus.
- Any woman becoming pregnant during the treatment period must discontinue the use of sirolimus.
Key exclusion criteria (11 listed)
- Uncontrolled systemic infection
- Unstable disease states (i.e., hepatic failure, ventilatory-dependent respiratory failure, etc.)
- Serum creatinine ≥ 3.0 mg/dL
- Platelet count ≤ 50,000/mm³
- History of Post-transplant microangiopathic hemolytic anemia
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 |
|---|---|---|
| SirolimusAlso: RapamunePatients will receive sirolimus at 2 mg/day orally with monitoring of trough drug levels weekly for 2 weeks to achieve trough drug levels 7-12 ng/ml. Along with prednisone therapy. | Drug | Sirolimus Therapy |
| PrednisoneAlso: DeltasonePrednisone therapy will remain at the dose the patient received at the time sirolimus was begun. Withdrawal of prednisone will began after first evidence of improvement of chronic GVHD. | Drug | Sirolimus Therapy |
Primary outcome
Clinical Activity
Time frame: 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus
Determined by discontinuation of immunosuppression with resolution of all reversible CGVHD manifestations. Evaluated at 2 years after enrollment
1 secondary outcome
Overall Survival
Time frame: 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Graft vs Host Disease
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.
Laura Johnston principal investigator
Stanford University
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 Sirolimus and Prednisone in people with Graft vs Host Disease, run by Stanford University 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 9 inclusion and 11 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 21 Apr 2017. 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 21 Apr 2017). 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?
Clinical Activity (time frame: 3 month intervals after the initiation of sirolimus until 2 years after the initiation of sirolimus). 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 21 Apr 2017.