Graft-versus-Host DiseasePhase 2NCT00408928
Study on the Safety and Effectiveness of VELCADE® in the Treatment of Graft-Versus-Host Disease
A Phase 2 study testing Bortezomib in people with Graft-versus-Host Disease, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.
Trial summary
The purpose of this research study is to test the safety and effectiveness of VELCADE® in the treatment of acute graft-versus-host disease (GVHD) that has not responded to steroids or has worsened when the steroid dose was decreased. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft-versus-Host Disease
- What is being tested?
- Bortezomib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to Sep 2009
- 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
- Patients must have undergone an allogeneic HSCT
- Clinical or histological evidence of AGVHD
- Has been treated with a minimum of 2mg/kg of methylprednisolone per day or equivalent dose of steroids and either one of the following:
- Has had a minimum of 3 days of steroids including the day of assignment and has progressive disease.
- Has had a minimum of 7 days of steroids including the day of assignment and has had no response.
- AGVHD progresses at anytime when steroids are tapered to less than 2mg/kg/day of methylprednisolone or its equivalent.
Show all 10Show fewer
- Performance status ECOG 0-2
- Patients must be willing to use contraception if they have childbearing potential
- Able to give informed consent
- Patients must be > 18 years of age, with no upper age limit.
Who cannot join
- Performance status of ECOG >2.
- >Grade3 peripheral neuropathy at the time of enrollment
- Patient has a creatinine clearance (calculated or measured) of <30mL/min at the time of enrollment.
- Myocardial infarction within 6 months prior to enrollment or has New York Hospital Association (NYHA) Class III or IV heart failure, 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/co-investigator as not medically relevant.
- Patient has hypersensitivity to bortezomib, boron or mannitol.
- Female subject is pregnant or breast-feeding.
- Patient has received other investigational drug within 14 days prior to enrollment.
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study or to obtain informed consent.
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
About this study
Official title: Phase II Trial of VELCADE® (Bortezomib) for Steroid Refractory Acute GVHD
The purpose of this research study is to test the safety and effectiveness of VELCADE® in the treatment of acute graft-versus-host disease (GVHD) that has not responded to steroids or has worsened when the steroid dose was decreased. VELCADE® is a drug that inhibits certain immune reactions that happen when lymphocytes encounter foreign substances. We are doing this research to determine if VELCADE® may be useful in treating GVHD.
Read the detailed description
Graft-versus-host disease (GVHD) is a serious complication after bone marrow transplantation from another donor. GVHD is caused by certain cells called lymphocytes. Normally these cells make immune reactions that help protect the body from foreign substances that cause infection. Here, these cells attack the normal tissues of the body as if they were foreign substances. This interferes with the normal function of vital organs and results in their damage. In GVHD these cells attack the skin, liver and bowel. GVHD also increases the chances of infection.
VELCADE® is a drug that inhibits certain immune reactions that happen when lymphocytes encounter foreign substances. We are doing this research to determine if VELCADE® may be useful in treating GVHD.
VELCADE® is approved by the Food and Drug Administration (FDA) for the treatment of multiple myeloma in patients who have received at least two prior therapies and have demonstrated disease progression on their last therapy. Its effectiveness is also being tested in other cancers. The dose of the drug being used in this research study is the same as what is used for the treatment of multiple myeloma. It has not been approved by the FDA for use in GVHD. Therefore, using VELCADE® for GVHD is experimental in this research study.
- Conditions
- Graft-versus-Host Disease
- Being tested
- Bortezomib
- Keywords
- Graft-versus-Host Disease, bortezomib
- 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.
BortezomibDrug
Also called VELCADE, PS-341
Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib.
| Study group | Role | What they receive |
|---|---|---|
| Bortezomib for Treatment of GHVD | Experimental | To determine if bortezomib (VELCADE®) will successfully inhibit T-cell responses in clinically acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (HSCT). Receives: Drug: Bortezomib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response to Bortezomib (VELCADE®)
Response is the primary endpoint of this study and will be scored on day 21 (3 weeks after the first dose of VELCADE) and every 3 weeks subsequently. Patients who progress or expire before the end of the study will be considered non-responders.
Patients are evaluated for response in an organ if they have AGVHD in that organ at the start of treatment with VELCADE or if AGVHD develops after the start of VELCADE, but before the time period of evaluation. Complete response in an organ is defined as no evidence clinical or biochemical signs of AGVHD. For the overall assessment, it is defined as complete resolution of rash, abnormal LFTs, and absence of diarrhea attributed to AGVHD.
Partial response is defined as a one stage decrease in any organ system without worsening in other organ systems.
Measured: Through 30 days post-treatment
Secondary outcomes (1)
Number of Toxicities Related to Bortezomib (VELCADE®)
Observe the toxicities of VELCADE® when administered to recipients of allogeneic hematopoietic stem cell transplant in the setting of steroid refractory or steroid dependent acute graft-versus-host disease.
Measured: Through 30 days post-traeatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2006 (estimated)
- Study startNov 2005
- Primary completionSep 2008
- Study completionSep 2009
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-versus-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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- John Wagner, MD, Principal investigator, Thomas Jefferson University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00408928
- Sponsor's protocol ID
- 04U.177
- Other IDs
- 2004-19, JT 1129
- Status last verified
- May 2025
- First submitted
- 6 Dec 2006
- Last updated
- 4 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of VELCADE® (Bortezomib) for Steroid Refractory Acute GVHD
- ClinicalTrials.gov ID
- NCT00408928
- Acronym / sponsor ID
- 04U.177
- Conditions
- Graft-versus-Host Disease
- Keywords
- Graft-versus-Host Disease, bortezomib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- Sep 2008 (actual)
- Study completion
- Sep 2009 (actual)
- First posted
- 8 Dec 2006 (estimated)
- Results
- Posted 28 Nov 2013
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 research study is to test the safety and effectiveness of VELCADE® in the treatment of acute graft-versus-host disease (GVHD) that has not responded to steroids or has worsened when the steroid dose was decreased. VELCADE® is a drug that inhibits certain immune reactions that happen when lymphocytes encounter foreign substances. We are doing this research to determine if VELCADE® may be useful in treating GVHD.
Detailed description
Graft-versus-host disease (GVHD) is a serious complication after bone marrow transplantation from another donor. GVHD is caused by certain cells called lymphocytes. Normally these cells make immune reactions that help protect the body from foreign substances that cause infection. Here, these cells attack the normal tissues of the body as if they were foreign substances. This interferes with the normal function of vital organs and results in their damage. In GVHD these cells attack the skin, liver and bowel. GVHD also increases the chances of infection.
VELCADE® is a drug that inhibits certain immune reactions that happen when lymphocytes encounter foreign substances. We are doing this research to determine if VELCADE® may be useful in treating GVHD.
VELCADE® is approved by the Food and Drug Administration (FDA) for the treatment of multiple myeloma in patients who have received at least two prior therapies and have demonstrated disease progression on their last therapy. Its effectiveness is also being tested in other cancers. The dose of the drug being used in this research study is the same as what is used for the treatment of multiple myeloma. It has not been approved by the FDA for use in GVHD. Therefore, using VELCADE® for GVHD is experimental in this research study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bortezomib for Treatment of GHVD | Experimental | Drug: BortezomibTo determine if bortezomib (VELCADE®) will successfully inhibit T-cell responses in clinically acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (HSCT). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients must have undergone an allogeneic HSCT
- Clinical or histological evidence of AGVHD
- Has been treated with a minimum of 2mg/kg of methylprednisolone per day or equivalent dose of steroids and either one of the following:
- Performance status ECOG 0-2
- Patients must be willing to use contraception if they have childbearing potential
Key exclusion criteria (8 listed)
- Performance status of ECOG >2.
- >Grade3 peripheral neuropathy at the time of enrollment
- Patient has a creatinine clearance (calculated or measured) of <30mL/min at the time of enrollment.
- Myocardial infarction within 6 months prior to enrollment or has New York Hospital Association (NYHA) Class III or IV heart failure, 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/co-investigator as not medically relevant.
- Patient has hypersensitivity to bortezomib, boron or mannitol.
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: VELCADE, PS-341Bortezomib at 1.3 mg/m2/dose given twice weekly for two weeks followed by a 10-day rest period. If patients have a complete response, they will receive additional cycles of bortezomib. | Drug | Bortezomib for Treatment of GHVD |
Primary outcome
Response to Bortezomib (VELCADE®)
Time frame: Through 30 days post-treatment
Response is the primary endpoint of this study and will be scored on day 21 (3 weeks after the first dose of VELCADE) and every 3 weeks subsequently. Patients who progress or expire before the end of the study will be considered non-responders. Patients are evaluated for response in an organ if they have AGVHD in that organ at the start of treatment with VELCADE or if AGVHD develops after the start of VELCADE, but before the time period of evaluation. Complete response in an organ is defined as no evidence clinical or biochemical signs of AGVHD. For the overall assessment, it is defined as complete resolution of rash, abnormal LFTs, and absence of diarrhea attributed to AGVHD. Partial response is defined as a one stage decrease in any organ system without worsening in other organ systems.
1 secondary outcome
Number of Toxicities Related to Bortezomib (VELCADE®)
Time frame: Through 30 days post-traeatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Kimmel Cancer Center at Thomas Jefferson University, an NCI-Designated Cancer CenterLink provided by the sponsor.
- Thomas Jefferson University HospitalLink 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 Wagner, MD principal investigator
Thomas Jefferson 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 Bortezomib in people with Graft-versus-Host Disease, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson 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 10 inclusion and 8 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 4 May 2025. 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 28 Nov 2013). 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?
Response to Bortezomib (VELCADE®) (time frame: Through 30 days post-treatment). 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 4 May 2025; AzTrials last refreshed its copy on 26 Oct 2026.