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Efficacy and Safety of Low-dose Ibrutinib and Itraconazole in Chronic Graft Versus Host Disease

Phase 2
Conditions
Chronic Graft-versus-host-disease
Interventions
Registration Number
NCT05348096
Lead Sponsor
Hospital Universitario Dr. Jose E. Gonzalez
Brief Summary

Chronic graft-versus-host disease (cGVHD) affects 30 to 70% of Allogeneic Hematopoietic Cell Transplantation, decreases the quality of life, and increases mortality. First-line treatments for cGVHD are steroids, however, up to 50% of patients do not respond to treatment. There is no well-defined second-line treatment for cGVHD, but ibrutinib, a Bruton tyrosine kinase inhibitor, has been successfully used in phase 2 clinical trials for moderate to severe steroid-refractory cGVHD and has been shown to be safe, showing rates of response of 69% at a median follow-up of 26 months. Therefore, ibrutinib was approved by the FDA for the treatment of steroid-refractory cGVHD. Also, it is known that ibrutinib is metabolized by cytochrome isoenzyme 3A4 and that itraconazole is a potent inhibitor of this hepatic isoenzyme. Therefore, the investigators hypothesized that in subjects with newly diagnosed cGVHD and in patients with steroid-refractory cGVHD, low-dose ibrutinib in combination with itraconazole might be effective and safe.

Detailed Description

In this phase 2 clinical trial, patients with newly diagnosed cGVHD and refractory cGVHD will receive low-dose ibrutinib (140mg/day) combined with a cytochrome 3A4 inhibitor (itraconazole, 100mg BID) for six months. The follow-up consists of weekly visits for the first months and then monthly for six months. The investigators will address clinical and biochemical parameters in each visit and grade severity using the NIH (2014) scale. Also, patients will answer the modified Lee symptom scale, and grade response to treatment using the National Institutes of Health (NIH) Consensus Panel Chronic GVHD Activity Assessment (2014). The investigators will grade adverse events with the Common Terminology Criteria for Adverse Events \[v5.0\]. The investigators will report proportion and time to any response, complete response, partial response, stable disease, and progression. Also, the investigators will report the proportion of patients that interrupted steroids for at least one month, the proportion of patients that interrupted every immunosuppressive therapy for at least one month, and the proportion of patients that interrupted ibrutinib specifying the cause of the interruption.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
13
Inclusion Criteria
  • Age (>18 years)

  • Any type of peripheral blood stem cell transplant (matched-related, match non-related, and haplo)

  • Any conditioning regimen

  • Newly diagnosed moderate to severe chronic graft versus host disease

  • Steroid refractory moderate to severe chronic graft versus host disease defined as progression with prednisone 1mg/kg/day, or stable disease after four to six weeks of prednisone >0.5 mg/kg/day, or disease progression when reducing prednisone below <0.5 mg/kg/día.

    1. Eastern Cooperative Oncology Group (ECOG) <= 2
Exclusion Criteria
  • Disease relapse (excluding positive minimal residual disease)
  • Secondary malignancies
  • Disease progression
  • Use of B lymphocyte cytotoxics in the last month (i.e., rituximab, bortezomib)
  • Advance stages of heart failure (NYHA III o IV)
  • Ventricular arrhythmias
  • Uncontrolled hypertension
  • Ischemic heart diseases such as unstable angina or stable angina in the last six months
  • Hepatitis B or C
  • Hypersensitivity to ibrutinib
  • Active bleeding
  • Uncontrolled acute infection
  • Hepatopathy Child-Pugh C
  • Pregnancy

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Low-dose ibrutinibLow-dose ibrutinibPatients will receive ibrutinib 140mg/day PO in combination with oral itraconazole (100mg/day) continuously for six months.
Primary Outcome Measures
NameTimeMethod
Treatment safetyUp to six months of enrollment

Treatment safety will be addressed by obtaining the proportion of patients with grade \>=3 adverse events as defined by the Common Terminology Criteria for Adverse Events \[v5.0\]. If the proportion of \>=3 adverse events is less than 20% then the treatment will be defined as safe.

Overall response rateUp to six months of enrollment

The proportion of patients with partial and/or complete response at six months of follow-up.

Secondary Outcome Measures
NameTimeMethod
Steroid-free cumulative incidenceUp to six months post enrollment

The number of patients using 0mg of prednisone for at least one month divided by the total number of patients at the time interval of the study.

Overall treatment-free survivalUp to six months post enrollment

The proportion of patients with any other treatment rather than ibrutinib at six months of follow-up.

Low-dose steroid cumulative incidenceUp to six months post enrollment

The number of patients using less than 0.15 mg/kg/day of prednisone 0 for at least one month divided by the total number of patients at the time interval of the study.

Immunosuppressive-free cumulative incidenceUp to six months post enrollment

The number of patients without any immunosuppressor divided by the total number of patients at the time interval of the study.

Overall survivalUp to six months post enrollment

Overall survival is defined as the length of time from the start of ibrutinib to the time of death.

Time to any responseFrom date of inclusion until the date of first documented response (partial or complete), assessed up to six months.

Time length from the first day of ibrutinib to any response (partial response or complete response)

Time to progressionFrom date of inclusion until the date of first documented progression, assessed up to 6 months.

Time length from the first day of ibrutinib to progression.

Complete response rateUp to six months post enrollment

The complete response rate was defined as the proportion of patients that achieve complete responses within the study's time frame.

Partial response rateUp to six months post enrollment

The partial response rate was defined as the proportion of patients that achieve partial responses within the study's time frame.

Progression rateUp to six months post enrollment

The progression rate was defined as the proportion of patients that progresses within the study's time frame.

Any adverse events rateUp to six months post enrollment

The any adverse event rate was defined as the proportion of patients with any grade adverse events within the study's time frame.

Proportion of therapy interruptionUp to six months post enrollment

The proportion of patients that need ibrutinib interruption because of unacceptable toxicity (grade \>=3).

Trial Locations

Locations (1)

Hospital Universitario Dr. José Eleuterio González

🇲🇽

Monterrey, Nuevo Leon, Mexico

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