Hematopoetic Stem Cell TransplantPhase 1NCT01926899
A Multicenter Phase I Study Evaluating the Addition of Bortezomib to an Established Acute Graft Versus Host Disease Prophylaxis Regimen in Pediatric Allogeneic Hematopoietic Stem Cell Transplant Patients
A Phase 1 study testing Bortezomib administration in people with Hematopoetic Stem Cell Transplant, run by Indiana University at 2 sites in United States.
Trial summary
The objective of this study is to determine the maximum tolerated dose (MTD) of bortezomib in combination with calcineurin inhibitor and methotrexate as acute graft versus host disease (aGVHD) prophylaxis in pediatric patients undergoing allogeniec hematopoietic stem cell transplant (alloHSCT) Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Hematopoetic Stem Cell Transplant
- What is being tested?
- Bortezomib administration
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Aug 2013to 15 Mar 2018
- 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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
- Female subject of childbearing potential agree to practice 2 effective methods of contraception from the time of signing the informed consent form through 30 days after the last dose of bortezomib, or agree to completely abstain from heterosexual intercourse.
- Male subjects, even if surgically sterilized (i.e., status postvasectomy) must agree to 1 of the following: practice effective barrier contraception during the entire study treatment period and through a minimum of 30 days after the last dose of study drug, or completely abstain from heterosexual intercourse.
- Subjects must be greater than or equal to 1 years old and less 22 years old.
- Subjects must have any malignant or non-malignant condition that requires treatment with alloHSCT
- Karnofsky or Lansky performance score greater than 60%
- Subjects must have a 9 of 10 (HLA A, B, C, DR, and DQ) or 10 of 10 HLA matched-related or MUD for bone marrow or peripheral blood alloHSCT
- Subjects must meet other institutional criteria for alloHSCT
Who cannot join
- • Patient has greater than 1.5 times upper limit normal (ULN) Total Bilirubin
- Patient has greater than or equal to Grade 2 peripheral neuropathy
- Patient had myocardial infarction within 6 months prior to enrollment or has New York Heart 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 must be documented by the investigator as not medically relevant.
- Patient has hypersensitivity to bortezomib, boron, or mannitol.
- Female subject is pregnant or lactating. Confirmation that the subject is not pregnant must be established by a negative serum beta-human chorionic gonadotropin (beta hCG) pregnancy test result obtained during screening. Pregnancy testing is not required for postmenopausal or surgically sterilized women.
- Female patients who are lactating or have a positive serum pregnancy test during the screening period, or a positive urine pregnancy test on Day 1 before first dose of study drug, if applicable.
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- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
- Diagnosed or treated for another malignancy within 2 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.
- Participation in clinical trials with other investigational agents not included in this trial, within 14 days of the start of this trial and throughout the duration of this trial.
- 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.
- Current active infection per physician determination
- HIV positive
- Previous myeloablative autoHSCT or alloHSCT in previous 12 months
- ALT and AST greater than 5 times ULN for age
- Creatinine clearance or glomerular filtration rate (GFR) less than 60 ml/min/1.73
- Shortening fraction less than 26% or ejection fraction less than 45%
- Diffusing capacity of carbon monoxide (DLCO), volume exhaled at end of first second of forced expiration (FEV1), diffusion capacity less than 50% of predicted (corrected for hemoglobin); if unable to perform lung function tests, oxygen saturation less than 94% on room air
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Indiana University
Indianapolis, Indiana, 46202
Texas Transplant Institute
San Antonio, Texas, 78229
About this study
Official title: A Multicenter Phase I Study Evaluating the Addition of Bortezomib to an Established Acute Graft Versus Host Disease (aGVHD) Prophylaxis Regimen in Pediatric Allogeneic Hematopoietic Stem Cell Transplant (Allo HSCT) Patients
The objective of this study is to determine the maximum tolerated dose (MTD) of bortezomib in combination with calcineurin inhibitor and methotrexate as acute graft versus host disease (aGVHD) prophylaxis in pediatric patients undergoing allogeniec hematopoietic stem cell transplant (alloHSCT)
- Conditions
- Hematopoetic Stem Cell Transplant
- Being tested
- Bortezomib administration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bortezomib administrationDrug
| Study group | Role | What they receive |
|---|---|---|
| Bortezomib administration | Experimental | 0.7 milligrams per meter squared given on Day 0 and Day +3 Receives: Drug: Bortezomib administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose of bortezomib
Measured: 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2013 (estimated)
- Study start1 Aug 2013
- Primary completion1 Feb 2018
- Study completion15 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoetic Stem Cell TransplantStudies of the same condition, open and under way.
- More trials run by Indiana 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 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, David Delgado, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01926899
- Sponsor's protocol ID
- 1209009655
- Status last verified
- Mar 2018
- First submitted
- 19 Aug 2013
- Last updated
- 22 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase I Study Evaluating the Addition of Bortezomib to an Established Acute Graft Versus Host Disease (aGVHD) Prophylaxis Regimen in Pediatric Allogeneic Hematopoietic Stem Cell Transplant (Allo HSCT) Patients
- ClinicalTrials.gov ID
- NCT01926899
- Acronym / sponsor ID
- 1209009655
- Conditions
- Hematopoetic Stem Cell Transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 1 Aug 2013 (actual)
- Primary completion
- 1 Feb 2018 (actual)
- Study completion
- 15 Mar 2018 (actual)
- First posted
- 21 Aug 2013 (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 objective of this study is to determine the maximum tolerated dose (MTD) of bortezomib in combination with calcineurin inhibitor and methotrexate as acute graft versus host disease (aGVHD) prophylaxis in pediatric patients undergoing allogeniec hematopoietic stem cell transplant (alloHSCT)
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bortezomib administration | Experimental | Drug: Bortezomib administration0.7 milligrams per meter squared given on Day 0 and Day +3 |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (17 listed)
- • Patient has greater than 1.5 times upper limit normal (ULN) Total Bilirubin
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 |
|---|---|---|
| Bortezomib administration | Drug | Bortezomib administration |
Primary outcome
Maximum tolerated dose of bortezomib
Time frame: 100 days post transplant
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 22 Mar 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Bortezomib administration in people with Hematopoetic Stem Cell Transplant, run by Indiana University 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 8 inclusion and 17 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 22 Mar 2018. 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?
Maximum tolerated dose of bortezomib (time frame: 100 days post transplant). 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 22 Mar 2018.