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Primary MyelofibrosisPhase 1/2NCT02806375

PTCy and Ruxolitinib GVHD Prophylaxis in Myelofibrosis

A Phase 1/2 study testing Allogeneic hematopoietic stem cell transplantation, Busulfan and Fludarabine monophosphate in people with Primary Myelofibrosis and Myeloproliferative Disorders, run by St. Petersburg State Pavlov Medical University at 1 site in Russia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

A number of groups have demonstrated very low incidence of acute and chronic graft-versus-host disease (GVHD) with post-transplantation cyclophosphamide (PTCy) in haploidentical and unrelated allogeneic stem cell transplantation (SCT). Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Allogeneic hematopoietic stem cell transplantation, Busulfan and Fludarabine monophosphate
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteRussia
Study dates
Jan 2016to Apr 2019
Phase
Phase 1/2
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 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have an indication for allogeneic hematopoietic stem cell transplantation
  • Diagnosis:
  • Primary myelofibrosis Secondary myelofibrosis
  • Signed informed consent
  • Matched related, 8-10/10 HLA-matched unrelated or haploidentical donor available. The HLA typing is performed by the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1.
  • No second tumors
  • No severe concurrent illness

Who cannot join

  • Moderate or severe cardiac dysfunction, left ventricular ejection fraction <50%
  • Moderate or severe decrease in pulmonary function, FEV1 <70% or DLCO<70% of predicted
  • Respiratory distress >grade I
  • Severe organ dysfunction: AST or ALT >5 upper normal limits, bilirubin >1.5 upper normal limits, creatinine >2 upper normal limits
  • Creatinine clearance < 60 mL/min
  • Uncontrolled bacterial or fungal infection at the time of enrollment
Show all 10Show fewer
  • Requirement for vasopressor support at the time of enrollment
  • Karnofsky index <30%
  • Pregnancy
  • Somatic or psychiatric disorder making the patient unable to sign informed consent

Trial locations and contacts

1 site in 1 country.

Russia1 site

  • First Pavlov State Medical University of St. Petersburg

    Saint Petersburg, 197089

Other trials in Russia

About this study

Official title: Graft-versus-host Disease Prophylaxis With Post-transplantation Cyclophosphamide and Ruxolitinib in Patients With Myelofibrosis

A number of groups have demonstrated very low incidence of acute and chronic graft-versus-host disease (GVHD) with post-transplantation cyclophosphamide (PTCy) in haploidentical and unrelated allogeneic stem cell transplantation (SCT). Still the relapse of the underlining malignancy is a problem after this prophylaxis. Ruxolitinib is currently one of the most promising drugs in the treatment of steroid-refractory GVHD. On the other hand, its primary indication is myelofibrosis, and it was demonstrated that ruxolitinib before allogeneic SCT might improve the outcome. This pilot trial evaluates whether the combination of PTCy and ruxolitinib facilitates adequate GVHD control, and decreases the risk of graft failure and disease progression in myelofibrosis patients.

Keywords
Myelofibrosis, Ruxolitinib, Cyclophosphamide, Immunosuppressive Agents, Immune System Diseases, Busulfan, Fludarabine, Antineoplastic Agents, Alkylating, Myeloablative Agonists, Hematopoietic Stem Cell Transplantation, Allogeneic
Medical subject headings
Primary Myelofibrosis, Myeloproliferative Disorders, Immune System Diseases

What participants receive

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

Study groups in NCT02806375
Study groupRoleWhat they receive
PTCy and ruxolitinibExperimentalNot described
Receives: Procedure: Allogeneic hematopoietic stem cell transplantation; Drug: Busulfan; Drug: Fludarabine monophosphate; Drug: Cyclophosphamide; Drug: Ruxolitinib

What is being measured

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

  1. Incidence of acute graft-versus-host disease, grades II-IV

    Measured: 180 days

  2. Incidence of chronic GVHD, moderate and severe (NIH criteria)

    Measured: 365 days

Secondary outcomes (7)

  1. Incidence of primary or secondary graft failure

    Measured: 60 days

  2. Non-relapse mortality analysis

    Non-relapse mortality is defined as any death in absence of relapse or progressive disease. Summarized using Kaplan-Meier and cumulative incidence estimates.

    Measured: 365 days

  3. Overall survival analysis

    Summarized using Kaplan-Meier and cumulative incidence estimates.

    Measured: 365 days

  4. Event-free survival analysis

    Event is defined as relapse or death in the specified time frame. Summarized using Kaplan-Meier and cumulative incidence estimates.

    Measured: 365 days

  5. Relapse rate analysis

    Summarized using Kaplan-Meier and cumulative incidence estimates.

    Measured: 365 days

  6. Number of participants with treatment-related adverse events as assessed by CTCAE v4.03

    Toxicity parameters based on NCI CTCAE 4.03 grades: hepatotoxicity (liver function tests), nephrotoxicity (creatinine), neurotoxicity (attending physician assessment), mucositis (attending physician assessment), hemorrhagic cystitis (attending physician assessment), cardiotoxicity (ECG, echocardiography). Additional toxicity parameters: incidence and severity of veno-occlusive disease, incidence of transplant-associated microangiopathy

    Measured: 100 days

  7. Infectious complications, including analysis of severe bacterial, fungal and viral infections incidence

    Measured: 100 days

Timeline

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

  1. First posted20 Jun 2016 (estimated)
  2. Study startJan 2016
  3. Primary completionDec 2018
  4. Study completionApr 2019

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02806375
Sponsor's protocol ID
04/16-n
Status last verified
Apr 2019
First submitted
8 Jun 2016
Last updated
4 Apr 2019
Results posted
No
Data monitoring committee
No
Individual data sharing
Undecided: Consultations with lawyers are ongoing about how IPD initiative fits the local law "About personal data 152-fz".

Publications

  • Morozova EV, Barabanshikova MV, Moiseev IS, Shakirova AI, Barhatov IM, Ushal IE, Rodionov GG, Moiseev SI, Surkova EA, Lapin SV, Vlasova JJ, Rudakova TA, Darskaya EI, Baykov VV, Alyanski AL, Bondarenko SN, Afanasyev BV. A Prospective Pilot Study of Graft-versus-Host Disease Prophylaxis with Post-Transplantation Cyclophosphamide and Ruxolitinib in Patients with Myelofibrosis. Acta Haematol. 2021;144(2):158-165. doi: 10.1159/000506758. Epub 2020 Apr 23.

    PubMed 32325461

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Allogeneic hematopoietic stem cell transplantation, Busulfan and Fludarabine monophosphate in people with Primary Myelofibrosis and Myeloproliferative Disorders, run by St. Petersburg State Pavlov Medical University at 1 site in Russia.

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 7 inclusion and 10 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 Apr 2019. 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 Russia. 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 combined 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.

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?

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?

Incidence of acute graft-versus-host disease, grades II-IV (time frame: 180 days). The study lists 2 primary outcomes in all. 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 NCT02806375, 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 4 Apr 2019.

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