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Graft Vs Host DiseasePhase 2NCT02997280

Ruxolitinib for Steroid-refractory GVHD

A Phase 2 study testing Ruxolitinib Oral Tablet in people with Graft Vs Host Disease, 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

Steroid-refractory acute GVHD (srGVHD) is one of the causes of mortality after allogeneic stem cell transplantation, while steroid-refractory chronic GVHD significantly increases morbidity, aggravates quality of life and may also impact survival. Currently there is no standard treatment of srGVHD. Read more about this study

Who is it for?
Ages 1 year to 70 years
Conditions the study is for
Graft Vs Host Disease
What is being tested?
Ruxolitinib Oral Tablet
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
75 participants
Where
1 siteRussia
Study dates
Aug 2016to Apr 2019
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 1 year to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis of graft-versus-host disease established based on tissue biopsy
  • Steroid-refractory acute or chronic graft-versus-host disease according to EBMT/ELN criteria (T. Ruutu et al, 2014)
  • Age 1 to 70 years
  • Karnofsky index >30%.
  • Ability for oral drug intake
  • Life expectancy > 1 month
  • Signed informed consent

Who cannot join

  • Severe organ dysfunction: AST or ALT >5 upper normal limits (excluding cases related to liver GVHD), creatinine >2 upper normal limits
  • Requirement for vasopressor support at the time of enrollment
  • Uncontrolled bacterial or fungal infection at the time of enrollment
  • Pregnancy
  • Somatic or psychiatric disorder making the patient (or legal guardian) 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: Treatment of Steroid-refractory Acute and Chronic Graft-versus-host Disease With Inhibitor of Janus Kinases

Steroid-refractory acute GVHD (srGVHD) is one of the causes of mortality after allogeneic stem cell transplantation, while steroid-refractory chronic GVHD significantly increases morbidity, aggravates quality of life and may also impact survival. Currently there is no standard treatment of srGVHD. One of the most promising agents is Janus kinase (JAK) inhibitor ruxolitinib, which in the retrospective study demonstrated excellent response rate and survival of patients with either acute or chronic srGVHD. This study prospectively evaluates the efficacy of ruxolitinib in srGVHD patients.

Being tested
Ruxolitinib Oral Tablet
Keywords
acute graft-versus-host disease, chronic graft-versus-host disease, Steroid-refractory GVHD, ruxolitinib, JAK inhibitors
Medical subject headings
Graft vs Host Disease, Bronchiolitis Obliterans Syndrome

What participants receive

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

  • Ruxolitinib Oral TabletDrug

    Dose reduction criteria for acute GVHD: grade 4 neutropenia or grade 4 thrombocytopenia related to ruxolitinib administration based on the decision of attending physician.

    Dose reduction criteria for chronic GVHD: grade 3 neutropenia, grade 3 thrombocytopenia or anemia requiring transfusion, related to ruxolitinib administration based on the decision of attending physician.

    Reduced dose schedule: 5 mg bid for adults and children with body weight > 40 kg, 0.08 mg/kg bid for children with body weight < 40 kg.

    Treatment discontinuation criteria: complete response; absence of response after 28 days or progressive disease after 7 days for acute GVHD; absence of response after 84 days or progressive disease after 28 days for chronic GVHD; life-threatening complications.

Study groups in NCT02997280
Study groupRoleWhat they receive
Ruxolitinib treatmentExperimentalRuxolitinib 10 mg bid for adults and children with body weight > 40 kg, 0.15 mg/kg bid for children with body weight < 40 kg.
Receives: Drug: Ruxolitinib Oral Tablet

What is being measured

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

  1. Overall response rate

    Partial response for acute GVHD is defined as the improvement of at least one stage in the severity of aGVHD in one organ without deterioration in any other organ. A response had to last for at least 3 weeks. Partial response for chronic GVHD was defined as reduction in GVHD National Institute of Health (NIH) severity score at east for one organ without deterioration in any other organ. A complete response was defined as the absence of any symptoms related to GVHD. Overall response is defined as presence of partial or complete response

    Measured: 84 days

Secondary outcomes (7)

  1. 6-month overall survival

    Time from start of ruxolitinib until death or 6 months, summarized using Kaplan-Meier estimates.

    Measured: 6 months

  2. Toxicity based NCI CTC grades

    Toxicity parameters based on NCI CTCAE 4.03 grades: nasea, vomiting, anemia, thrombocytopenia, leukopenia, neutropenia, hepatotoxicity (liver function tests), nephrotoxicity (creatinine), neurotoxicity (attending physician assessment), hemorrhagic cystitis (attending physician assessment).

    Measured: 6 months

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

    Measured: 6 months

  4. GVHD relapse incidence after complete response

    Time from stopping ruxolitinib until recurrence of GVHD or 6 months, summarized using cumulative incidence estimates.

    Measured: 6 months

  5. Relapse incidence of underlying hematologic malignancy

    Time from starting ruxolitinib until hematologic relapse or 12 months, summarized using cumulative incidence estimates

    Measured: 12 months

  6. Quality of life measured by FACT BMT ver. 4 questionnaire in adults

    Measured: 6 months

  7. Quality of life measured by PedQL Stem Cell transplant module ver.1.0 questionnaire in children

    Measured: 6 months

Timeline

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

  1. First posted20 Dec 2016 (estimated)
  2. Study startAug 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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
75 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02997280
Sponsor's protocol ID
2016-29-1
Status last verified
Apr 2019
First submitted
14 Dec 2016
Last updated
5 Apr 2019
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Zeiser R, Burchert A, Lengerke C, Verbeek M, Maas-Bauer K, Metzelder SK, Spoerl S, Ditschkowski M, Ecsedi M, Sockel K, Ayuk F, Ajib S, de Fontbrune FS, Na IK, Penter L, Holtick U, Wolf D, Schuler E, Meyer E, Apostolova P, Bertz H, Marks R, Lubbert M, Wasch R, Scheid C, Stolzel F, Ordemann R, Bug G, Kobbe G, Negrin R, Brune M, Spyridonidis A, Schmitt-Graff A, van der Velden W, Huls G, Mielke S, Grigoleit GU, Kuball J, Flynn R, Ihorst G, Du J, Blazar BR, Arnold R, Kroger N, Passweg J, Halter J, Socie G, Beelen D, Peschel C, Neubauer A, Finke J, Duyster J, von Bubnoff N. Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey. Leukemia. 2015 Oct;29(10):2062-8. doi: 10.1038/leu.2015.212. Epub 2015 Jul 31.

    PubMed 26228813

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib Oral Tablet in people with Graft Vs Host Disease, 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 5 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 5 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 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?

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?

Overall response rate (time frame: 84 days). 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 NCT02997280, 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 5 Apr 2019.

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