Chronic Graft-versus-host DiseasePhase 2NCT01158105
Bortezomib for the Treatment of Refractory Chronic Graft-vs-Host Disease(cGVHD)
A Phase 2 study testing Bortezomib in people with Chronic Graft-versus-host Disease, run by Baylor Research Institute at 1 site in United States.
Trial summary
The purpose of this study is to see if bortezomib (Velcade) is effective in the treatment of refractory cGVHD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft-versus-host Disease
- What is being tested?
- Bortezomib
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2010to Jan 2017
- Phase
- Phase 2
- 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
- Each patient must meet all of the following inclusion criteria to be enrolled in the study:
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
- ≥18 years old.
- Able to understand and sign informed consent.
- Diagnosis of steroid-refractory cGVHD is defined as either failure to improve after 2 months or progression after 1 month of standard steroid based therapy.
- No previous treatment with Bortezomib for cGVHD
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- ECOG PS<3
- Total Bilirubin ≤ 1.5x ULN
- Life expectancy > 3months.
- Female subject is either post-menopausal or surgically sterilized or willing to use an acceptable method of birth control for the duration of the study and through a minimum of 30 days after the last dose of bortezomib.
- Male subject agrees to use an acceptable method for contraception for the duration of the study and through a minimum of 30 days after the last dose of study drug.
Who cannot join
- Patients meeting any of the following exclusion criteria are not to be enrolled in the study.
- Patient has a platelet count of <50x 10^9/L within 14 days before enrollment.
- Patient has an absolute neutrophil count of <1.0 x 10^9/L within 14 days before enrollment.
- Patient has a calculated or measured creatinine clearance of <30 mL/minute within 14 days before enrollment.
- Patients with Total Bilirubin > 1.5x ULN
- Patient has ≥ Grade 2 peripheral neuropathy within 14 days before enrollment.
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- Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure (see section 8.4), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at Screening has to be documented by the investigator as not medically relevant.
- Patient has hypersensitivity to bortezomib, boron or mannitol.
- Female subject is pregnant or breast-feeding. Confirmation that the subject is not pregnant must be established by a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test result obtained during screening. Pregnancy testing is not required for post-menopausal or surgically sterilized women.
- Patient has received other investigational drugs with 14 days before enrollment
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
- Primary malignancy (for which the transplant was received ) not in remission
- Diagnosed or treated for another malignancy within 3 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy.
- Radiation therapy within 3 weeks before randomization. Enrollment of subjects who require concurrent radiotherapy (which must be localized in its field size) should be deferred until the radiotherapy is completed and 3 weeks have elapsed since the last date of therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
Charles A. Cancer Center, Baylor University Medical Center
Dallas, Texas, 75246
About this study
Official title: Phase 2 Study of Bortezomib (Velcade) for the Treatment of Steroid Refractory Chronic Graft-vs-Host Disease
The purpose of this study is to see if bortezomib (Velcade) is effective in the treatment of refractory cGVHD.
Read the detailed description
Graft vs. host disease represents the most formidable problem following allogeneic hematopoietic stem cell transplantation for hematological diseases. Chronic graft-versus-host disease (cGVHD) is the most serious and common long-term complication of this treatment in patients surviving more than 100 days. Some patients are requiring immunosuppressive therapy for years. cGVHD is associated with a high degree of morbidity and mortality and remains a major cause of late death. When standard treatment with steroids is ineffective or poorly tolerated, effective therapeutic options are limited. Patients with uncontrolled cGVHD with initial therapy including systemic steroids respond poorly to second line options. Recent studies have demonstrated Bortezomib safety in the prophylaxis, and improvement of GVHD suggesting its efficacy.This study is done to evaluate the potential of bortezomib in treating refractory cGVHD.
- Conditions
- Chronic Graft-versus-host Disease
- Being tested
- Bortezomib
- Keywords
- Steroid refractory chronic graft vs host disease (cGVHD), Bortezomib, Velcade
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
Also called Velcade
1.6 mg/m2 IV infusion on days 1, 8, 15, 22 of each 35 day cycle, for up to 6 cycles.Patients who continue to respond during the initial treatment phase with no ongoing significant adverse events will be eligible to receive up to 6 additional cycles.This maintenance dose will be administered on days 1 and 15.
| Study group | Role | What they receive |
|---|---|---|
| Bortezomib | Experimental | 1.6 mg/m2 intravenous infusion will be administered on days 1, 8, 15, 22 of each 35 day cycle, for up to 6 cycles. Patients who continue to respond during the initial treatment phase with no ongoing significant adverse events will be eligible to receive up to 6 additional cycles. This maintenance dose will be administered on days 1 and 15. Receives: Drug: Bortezomib |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the effectiveness and safety of the proteasome inhibitor Bortezomib (Velcade) in the treatment of steroid refractory chronic Graft-vs-Host Disease (cGVHD)
Total time frame including treatment and follow up is 19 months(Maximum of 6 cycles during initial treatment phase, 6 cycles in maintenance phase, and follow up phase for 6 months). Subjects will be assessed for signs and symptoms of cGVHD on day 1 of every cycle using standardized criteria for diagnosis of cGVHD, new clinical scoring system, and new guidelines for global assessment of chronic GVHD severity per NIH consensus development project on criteria for clinical trials in chronic GVHD. Safety will be assessed in terms of adverse events for 30 days following the last dose to a maximum of 6 months. Adverse events will be recorded through out the trial and will be followed by investigator until resolution.
Measured: 19 months
Secondary outcomes (1)
To examine blood cells from patients who develop steroid resistant cGVHD following allogeneic HSCT for disease related gene signatures using DNA microarray analysis techniques
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2010 (estimated)
- Study startJun 2010
- Primary completionMay 2013
- Study completionJan 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft-versus-host DiseaseStudies of the same condition, open and under way.
- More trials run by Baylor Research InstituteThe 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
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Millennium Pharmaceuticals, Inc.
- Responsible party
- Sponsor
- Investigators
- Alan Miller, MD, PhD, Principal investigator, Charles A. Caner Center, Baylor University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01158105
- Sponsor's protocol ID
- 010-037
- Status last verified
- Jan 2016
- First submitted
- 6 Jul 2010
- Last updated
- 18 Jan 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Bortezomib (Velcade) for the Treatment of Steroid Refractory Chronic Graft-vs-Host Disease
- ClinicalTrials.gov ID
- NCT01158105
- Acronym / sponsor ID
- 010-037
- Conditions
- Chronic Graft-versus-host Disease
- Keywords
- Steroid refractory chronic graft vs host disease (cGVHD), Bortezomib, Velcade
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Baylor Research InstituteUniversity, hospital or other
- Collaborators
- Millennium Pharmaceuticals, Inc.
- Study start
- Jun 2010
- Primary completion
- May 2013 (actual)
- Study completion
- Jan 2017 (estimated)
- First posted
- 8 Jul 2010 (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 see if bortezomib (Velcade) is effective in the treatment of refractory cGVHD.
Detailed description
Graft vs. host disease represents the most formidable problem following allogeneic hematopoietic stem cell transplantation for hematological diseases. Chronic graft-versus-host disease (cGVHD) is the most serious and common long-term complication of this treatment in patients surviving more than 100 days. Some patients are requiring immunosuppressive therapy for years. cGVHD is associated with a high degree of morbidity and mortality and remains a major cause of late death. When standard treatment with steroids is ineffective or poorly tolerated, effective therapeutic options are limited. Patients with uncontrolled cGVHD with initial therapy including systemic steroids respond poorly to second line options. Recent studies have demonstrated Bortezomib safety in the prophylaxis, and improvement of GVHD suggesting its efficacy.This study is done to evaluate the potential of bortezomib in treating refractory cGVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bortezomib | Experimental | Drug: Bortezomib1.6 mg/m2 intravenous infusion will be administered on days 1, 8, 15, 22 of each 35 day cycle, for up to 6 cycles. Patients who continue to respond during the initial treatment phase with no ongoing significant adverse events will be eligible to receive up to 6 additional cycles. This maintenance dose will be administered on days 1 and 15. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Each patient must meet all of the following inclusion criteria to be enrolled in the study:
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
- ≥18 years old.
- Able to understand and sign informed consent.
- Diagnosis of steroid-refractory cGVHD is defined as either failure to improve after 2 months or progression after 1 month of standard steroid based therapy.
Key exclusion criteria (14 listed)
- Patients meeting any of the following exclusion criteria are not to be enrolled in the study.
- Patient has a platelet count of <50x 10^9/L within 14 days before enrollment.
- Patient has an absolute neutrophil count of <1.0 x 10^9/L within 14 days before enrollment.
- Patient has a calculated or measured creatinine clearance of <30 mL/minute within 14 days before enrollment.
- Patients with Total Bilirubin > 1.5x ULN
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 |
|---|---|---|
| BortezomibAlso: Velcade1.6 mg/m2 IV infusion on days 1, 8, 15, 22 of each 35 day cycle, for up to 6 cycles.Patients who continue to respond during the initial treatment phase with no ongoing significant adverse events will be eligible to receive up to 6 additional cycles.This maintenance dose will be administered on days 1 and 15. | Drug | Bortezomib |
Primary outcome
To evaluate the effectiveness and safety of the proteasome inhibitor Bortezomib (Velcade) in the treatment of steroid refractory chronic Graft-vs-Host Disease (cGVHD)
Time frame: 19 months
Total time frame including treatment and follow up is 19 months(Maximum of 6 cycles during initial treatment phase, 6 cycles in maintenance phase, and follow up phase for 6 months). Subjects will be assessed for signs and symptoms of cGVHD on day 1 of every cycle using standardized criteria for diagnosis of cGVHD, new clinical scoring system, and new guidelines for global assessment of chronic GVHD severity per NIH consensus development project on criteria for clinical trials in chronic GVHD. Safety will be assessed in terms of adverse events for 30 days following the last dose to a maximum of 6 months. Adverse events will be recorded through out the trial and will be followed by investigator until resolution.
1 secondary outcome
To examine blood cells from patients who develop steroid resistant cGVHD following allogeneic HSCT for disease related gene signatures using DNA microarray analysis techniques
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2016.
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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.
Alan Miller, MD, PhD principal investigator
Charles A. Caner Center, Baylor University Medical Center
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 Bortezomib in people with Chronic Graft-versus-host Disease, run by Baylor Research Institute 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 11 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 18 Jan 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?
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 evaluate the effectiveness and safety of the proteasome inhibitor Bortezomib (Velcade) in the treatment of steroid refractory chronic Graft-vs-Host Disease (cGVHD) (time frame: 19 months). 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 18 Jan 2016.