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LeukemiaPhase 1NCT02484261

Monitoring and Treatment of Relapsed Leukemia Following Allogeneic Hematopoietic Stem Cell Transplantation in Children

A Phase 1 study testing Bortezomib and Pravastatin in people with Leukemia, run by Reuven Schore at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study aims to monitor patients for relapse of the leukemia following allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in order to identify patients early in relapse, with a low burden of disease, when interventions may be more successful by monitoring of peripheral blood lineage specific chimerism. Read more about this study

Who is it for?
Ages 22 months to 21 years
Conditions the study is for
Leukemia
What is being tested?
Bortezomib and Pravastatin
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
May 2015to 18 May 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 22 months to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Voluntary written informed consent by patient or legal guardian (as appropriate) 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performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care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 patients who:
  • Are postmenopausal for at least 1 year before the Screening visit, OR
  • Are surgically sterile, OR
  • If they are of childbearing potential, agree to practice 2 effective methods of contraception, at the same time, from the time of signing the informed consent through 30 days after the last dose of study treatment, OR agree to completely abstain from heterosexual intercourse
  • OR Male patients, even if surgically sterilized (i.e., status postvasectomy), who:
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  • Agree to practice effective barrier contraception during the entire study treatment period and through 30 days after the last dose of study treatment, OR
  • Agree to completely abstain from heterosexual intercourse
  • Lansky(if age <15)/Karnofsky (if age >16 yr) performance status > 50%
  • Hepatic function: total bilirubin ≤ 2.5 mg/dl, and ALT/AST < or equal to 5 x the upper limit of normal
  • Renal function: Creatinine clearance > 60 ml/min/1.73 m2 (as determined by 24 hour collection or nuclear (glomerular filtration rate) GFR or Cystatin C calculation)
  • Cardiac function: LVEF > 50% or LVSF > 27%
  • Pulmonary function tests: DLCO and FEV1 > 50% (if able to perform PFTs, if not able to perform PFTs SAO2> 94% on room air).
  • Acute leukemia or Myelodysplastic syndrome (MDS)
  • Inclusion Criteria to initiate therapy with bortezomib and pravastatin
  • Lansky performance status (if age < 15) /Karnofsky (if age >16 yr) performance status > 50%
  • Hepatic function: total bilirubin ≤ 2.5 mg/dl, and ALT/AST < or equal to 5 x the upper limit of normal (ULN)
  • Renal function: Cr < ULN for age
  • Relapse of systemic disease documented by any of the following: morphology, flow cytometry, cytogenetics and/or FISH.
  • Patient has a platelet count of more than 20,000/μL within 7 days before enrollment. Patients may be transfused prior to this evaluation but may not be platelet refractory at enrollment.
  • Patient has an absolute neutrophil count of greater than 500 /μL within 7 days before enrollment.
  • Patients with confirmed relapse post-HSCT who were not previously enrolled on monitoring portion of protocol, will also be eligible to enroll and receive novel combination if entry criteria met at time of relapse.

Who cannot join

  • Patients meeting any of the following exclusion criteria are not to be treated on the study:
  • 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 patients who are lactating or pregnant.
  • Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
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  • Evidence of residual or relapse of another malignancy(other than the hematological malignancy which is the indication for stem cell transplant)at the time of transplant preparative regimen initiation
  • Participation in clinical trials with other investigational agents not included in this trial, within 14 days of the start of bortezomib/pravastatin and throughout the duration of therapy.
  • Radiation therapy within 3 weeks before start of bortezomib/pravastatin. 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

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Pilot Study to Evaluate the Tolerability of Velcade (Bortezomib) and Pravastatin for the Treatment of Relapsed Leukemia Following Allogeneic Hematopoietic Stem Cell Transplantation in Children

This study aims to monitor patients for relapse of the leukemia following allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in order to identify patients early in relapse, with a low burden of disease, when interventions may be more successful by monitoring of peripheral blood lineage specific chimerism. Once disease has been confirmed, patients will initiate a novel combination of bortezomib and pravastatin.

Read the detailed description

Patients will be enrolled on this study at the time of transplantation. Following HSCT, CD34+ chimerism in peripheral blood will be monitored in real time at scheduled intervals. If chimerism of CD34+ cells diminishes post-HSCT or clinical signs or symptoms of relapsed leukemia, relapse in bone marrow will be confirmed, after which immunosuppression will be withdrawn and treatment initiated with bortezomib and pravastatin. Patients will be monitored for disease response until 1 year post-HSCT or post-study therapy. Patients with confirmed relapse post-HSCT will also be eligible to enroll and receive novel combination.

Conditions
Leukemia
Being tested
Bortezomib, Pravastatin
Keywords
childhood leukemia, relapsed leukemia, Hematopoietic Stem Cell Transplantation
Medical subject headings
Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BortezomibDrug

    Also called Velcade

    1.3 mg/m2/dose subcutaneously (SQ) daily (total 4 doses) on Days 1, 4, 8 and 11 (+/- 1 day with at least 72 hours between doses) q3 weeks. (For patients continuing to receive drug in cycles 6 or greater, at investigator discretion, the schedule may be changed to 1.3 mg/m2/dose SQ daily (total 3 doses) on Days 1, 8, and 15 (+/- 1 day with at least 72 hours between doses) every 4 weeks in an effort to decrease risk of peripheral neuropathy.)

  • PravastatinDrug

    10 mg for age < 8 years, 20 mg for age 8-<13 years, 40 mg for ages >14 years

Study groups in NCT02484261
Study groupRoleWhat they receive
Monitoring phaseNo interventionPatients who have received a Stem cell transplant for Hematologic malignancies will be monitored for signs of relapse with blood tests for CD34+ chimerism. Patients with signs of relapse will have bone marrow aspirate and/or other appropriate tests to confirm presence of relapse. All patients who are confirmed to have relapsed and meet eligibility criteria to receive study drug will be eligible to receive treatment on the treatment arm.
Treatment phaseExperimentalPatients with confirmed disease will initiate therapy with bortezomib and pravastatin, a regimen that has efficacy in treatment of leukemia and graft-versus-host disease, while sparing healthy donor hematopoietic stem cells may improve the dismal survival of relapse post allogeneic transplant. Depending on response, patients may receive up to 13 cycles of therapy
Receives: Drug: Bortezomib; Drug: Pravastatin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants with Adverse Events as a Measure of Safety and Tolerability

    Tolerability of bortezomib and pravastatin in pediatric relapsed leukemia patients following allogeneic transplantation

    Measured: 3 years

Secondary outcomes (1)

  1. Number of relapses that are detected by CD34+ chimerism before clinical symptoms

    The sensitivity of detection of recurrent acute leukemia in pediatric patients by peripheral blood CD34+ chimerism in pediatric patients following allogeneic HSCT

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2015 (estimated)
  2. Study startMay 2015
  3. Primary completion18 May 2018

Why it stopped: Contractual/Funding/Accrual

Resources

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
Non-randomised
Intervention model
Crossover
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02484261
Sponsor's protocol ID
Pro00004062
Status last verified
Mar 2023
First submitted
24 Jun 2015
Last updated
15 Mar 2023
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Bortezomib and Pravastatin in people with Leukemia, run by Reuven Schore 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 22 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: stopped early, as of the sponsor's last update on 15 Mar 2023. 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 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 was stopped early (reason given: Contractual/Funding/Accrual). 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?

Number of Participants with Adverse Events as a Measure of Safety and Tolerability (time frame: 3 years). 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 NCT02484261.

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 Mar 2023; AzTrials last refreshed its copy on 9 Nov 2026.

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