Clinical Study Offers Patients with 5 Disease States a Novel Advantage

By Rivkah Lewin

A clinical study is providing a novel benefit for patients that would have been unlikely before the FDA established its formal “compassionate use” program in 1987.  When patients have completed participation in the study, they can keep taking the medication—ibrutinib—if they are still benefiting from treatment, in the opinion of their treating physician.  They can continue receiving the drug until it becomes commercially available.  

This perk was enabled by FDA’s compassionate use expanded-access pathway for patients with a serious or immediately life-threatening disease or condition.  Such patients can gain access to investigational medical products  for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.  (For more information, go to Expanded Access | FDA)

After three years, however, those receiving the medication will be re-evaluated.  Patients who have no access to ibrutinib for their underlying disease in their region, and who meet other requirements, are eligible to receive this benefit.

Patients who are still benefiting from the treatment who are enrolled in this treatment protocol, will receive oral continuous dosing with ibrutinib at the same dose and schedule they were receiving at the end of the parent study.  They may continue treatment until ibrutinib becomes commercially available for the indication of the parent study.

Patients with five different disease states may participate:  Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Leukemia, B-cell; Graft vs. Host Disease; and Solid Tumor.  The clinical trial is being performed in 91 locations, including the U.S., Australia, Canada, and France.  To contact the clinical study in their area, click on Extended Treatment Protocol for Subjects Continuing to Benefit From Ibrutinib. – Full Text View – ClinicalTrials.gov, a webpage of NIH U.S. National Library of Medicine, ClinicalTrials.gov., scroll down, click on “Show 91 Study Locations,” and contact the location in their area.  Refer to this study by its ClinicalTrials.gov identification code NCT number: NCT03229200.

To be eligible for the parent study,  patients must be 18 years and older.  They must currently be participating in an ibrutinib trial, still be deriving clinical benefit from treatment, in the opinion of their treating physician, and not have access to commercial ibrutinib in their region.  They must have received ongoing treatment with ibrutinib, have completed all assessments in their parent study, and wish to continue treatment.  Both males and females of reproductive potential must agree to use both a highly effective method of birth control and a barrier method of birth control during the period of therapy and for 90 days after the last dose of the drug.

Patients are not eligible if they meet any requirement in the parent study which would require permanently discontinuing treatment with ibrutinib.  Those who have any condition or situation which their treating physician feels may interfere significantly with their participation in the study will not be able to participate.  Females who are pregnant, breastfeeding, or planning to become pregnant will be excluded, as will males who plan to father a child while enrolled in the study.  Healthy volunteers will also not be accepted.

The study is sponsored by Pharmacyclics Switzerland GmbH, with collaborator Janssen Biotech, Inc., including Johnson & Johnson.