Graft vs Host DiseasePhase 1NCT00760981
A Pilot Study of Imatinib Mesylate in Steroid Refractory Chronic Graft Versus Host Disease
A Phase 1 study testing Imatinib in people with Graft vs Host Disease, run by David Miklos at 2 sites in United States.
Trial summary
To determine if subjects with steroid refractory cGVHD can tolerate imatinib mesylate and whether their cGVHD responds to imatinib mesylate. 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?
- Imatinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2008to Apr 2012
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Chronic graft-vs-host disease (cGVHD) requiring systemic therapy occurring > 100 days after hematopoietic cell transplant with either:
- Persistent steroid dependence defined as the inability to taper steroid treatment to less than 0.25 mg/kg/d prednisone or its equivalent for at least 3 months.
- Progression of cGVHD signs and symptoms on steroid therapy equivalent to prednisone 0.5 mg/kg/d for at least 1 month.
- At least one of the following manifestations:
- Skin changes (rash, sclerosis, fasciitis, or ulceration).
- Abnormal eye wetness ≤ 5 mm as measured by Schirmer's test.
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- Oral mucosal changes (erythema, lichenoid changes, ulcers, or mucoceles).
- Thrombocytopenia (platelets < 50,000/uL).
- Abnormal liver function testing defined as alkaline phosphatase, AST, ALT, or total bilirubin > upper limit of normal (ULN).
- Bronchiolitis obliterans (diagnosed by a > 5% annual decline in FEV1 with the lowest post-transplant FEV1/FVC < 0.8 and an appropriate CT scan or lung biopsy).
- Has been on a fixed dose of steroids or a fixed dose of steroids and one other immunosuppressant (cyclosporine, tacrolimus, sirolimus, mycophenolate mofetil, or extracorporeal photopheresis) for ≥ 30 days before starting imatinib.
- Life expectancy ≥ 6 months.
- Ability to understand and willingness to sign a written informed consent document.
- Karnofsky performance status ≥ 3 50% (Appendix B).
- At least 18 years of age.
- If a female of reproductive potential (defined as having at least 1 menstrual period in the past 12 months), must have a negative pregnancy test performed ≤ 7 days before starting study drug.
- If a female of reproductive potential, agrees to use contraception for the duration of the trial.
- Total bilirubin < 1.5X ULN.
- Aspartate transaminase (AST) < 2.5 x ULN.
- Alanine aminotransferase (ALT) < 2.5 x ULN.
- Alkaline phosphatase < 2.5 x ULN.
- Absolute neutrophil count (ANC) > 500/uL (growth factor supplementation is allowed).
- Hematocrit > 26% (transfusion support is allowed).
- Platelet count > 20,000/uL.
Who cannot join
- Received another investigational agent ≤ 30 days before starting the study drug.
- Ongoing intercurrent illness such as an infection not responsive to antibiotics, antiviral medicines, or antifungal medicines.
- Progressive malignant disease.
- Secondary malignancy that has not been effectively treated within the past 5 years (except localized basal cell or squamous cell carcinoma).
- Imatinib intolerance or allergy.
- Participant is breast-feeding.
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- Not willing to comply with treatment or response evaluation.
- Received an allogeneic cell product [including donor lymphocyte infusion (DLI) or hematopoietic cell boost] ≤ 100 days before starting study drug.
- Steroid and/or immunosuppressant dose has changed ≤ 30 days before starting study drug.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Fred Hutchinson Cancer Research Center (FHCRC)
Seattle, Washington, 98109
Stanford University School of Medicine
Stanford, California, 94305
About this study
To determine if subjects with steroid refractory cGVHD can tolerate imatinib mesylate and whether their cGVHD responds to imatinib mesylate.
- Conditions
- Graft vs Host Disease
- Being tested
- Imatinib
- 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.
ImatinibDrug
Also called Gleevec
The single cohort for this study will receive 2 dose levels of imatinib, 200 mg orally daily followed by 400 mg orally daily.
| Study group | Role | What they receive |
|---|---|---|
| Imatinib | Experimental | 200 mg orally daily and 400 mg orally daily for 4 weeks. Receives: Drug: Imatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
The frequency of adverse events graded according to the CTCAE will be the primary endpoint
Measured: Subjects will be monitored at 1, 4, 8, 16, and 24 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2008 (estimated)
- Study startSep 2008
- Primary completionApr 2012
- Study completionApr 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- David Miklos
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Sponsor-investigator, David Miklos, Stanford University
- Investigators
- David Miklos, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00760981
- Sponsor's protocol ID
- IRB-14821
- Other IDs
- 14821, BMT195
- Status last verified
- Apr 2020
- First submitted
- 25 Sep 2008
- Last updated
- 15 Apr 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Chen GL, Arai S, Flowers ME, Otani JM, Qiu J, Cheng EC, McMillan A, Johnston LJ, Shizuru JA, Miklos DB. A phase 1 study of imatinib for corticosteroid-dependent/refractory chronic graft-versus-host disease: response does not correlate with anti-PDGFRA antibodies. Blood. 2011 Oct 13;118(15):4070-8. doi: 10.1182/blood-2011-03-341693. Epub 2011 Aug 9.
PubMed 21828142
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Imatinib Mesylate in Steroid Refractory Chronic Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT00760981
- Acronym / sponsor ID
- IRB-14821
- Conditions
- Graft vs Host Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- David MiklosUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Sep 2008
- Primary completion
- Apr 2012 (actual)
- Study completion
- Apr 2012 (actual)
- First posted
- 26 Sep 2008 (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.
To determine if subjects with steroid refractory cGVHD can tolerate imatinib mesylate and whether their cGVHD responds to imatinib mesylate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Imatinib | Experimental | Drug: Imatinib200 mg orally daily and 400 mg orally daily for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (24 listed)
- Chronic graft-vs-host disease (cGVHD) requiring systemic therapy occurring > 100 days after hematopoietic cell transplant with either:
- At least one of the following manifestations:
- Has been on a fixed dose of steroids or a fixed dose of steroids and one other immunosuppressant (cyclosporine, tacrolimus, sirolimus, mycophenolate mofetil, or extracorporeal photopheresis) for ≥ 30 days before starting imatinib.
- Life expectancy ≥ 6 months.
- Ability to understand and willingness to sign a written informed consent document.
Key exclusion criteria (9 listed)
- Received another investigational agent ≤ 30 days before starting the study drug.
- Ongoing intercurrent illness such as an infection not responsive to antibiotics, antiviral medicines, or antifungal medicines.
- Progressive malignant disease.
- Secondary malignancy that has not been effectively treated within the past 5 years (except localized basal cell or squamous cell carcinoma).
- Imatinib intolerance or allergy.
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 |
|---|---|---|
| ImatinibAlso: GleevecThe single cohort for this study will receive 2 dose levels of imatinib, 200 mg orally daily followed by 400 mg orally daily. | Drug | Imatinib |
Primary outcome
The frequency of adverse events graded according to the CTCAE will be the primary endpoint
Time frame: Subjects will be monitored at 1, 4, 8, 16, and 24 weeks.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2020.
- Results publication: PubMed 21828142Chen GL, Arai S, Flowers ME, Otani JM, Qiu J, Cheng EC, McMillan A, Johnston LJ, Shizuru JA, Miklos DB. A phase 1 study of imatinib for corticosteroid-dependent/refractory chronic graft-versus-host disease: response does not correlate with anti-PDGFRA antibodies. Blood. 2011 Oct 13;118(15):4070-8. doi: 10.1182/blood-2011-03-341693. Epub 2011 Aug 9.
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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.
David Miklos, MD principal investigator
Stanford University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Imatinib in people with Graft vs Host Disease, run by David Miklos at 2 sites 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 24 inclusion and 9 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 15 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The frequency of adverse events graded according to the CTCAE will be the primary endpoint (time frame: Subjects will be monitored at 1, 4, 8, 16, and 24 weeks.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00760981, which also lists 1 linked publication.
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 15 Apr 2020.