Phase 2 Trial Recruiting Scleroderma Patients with PAH in Test of Rituxan

Magdalena Kegel avatar

by Magdalena Kegel |

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A clinical trial exploring if Rituxan (rituximab) is effective in managing pulmonary arterial hypertension (PAH) in patients with scleroderma is now recruiting participants in numerous locations across the U.S.

Rituxan is an immunotherapy drug in the form of an antibody against the CD20 molecule, found on the surface of certain immune cells. The drug can prevent the actions of these immune cells. Rituxan, produced by Genentech, is an approved treatment for several types of non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and certain rheumatoid arthritis patients. It is an investigative therapy for any type of PAH.

The trial (NCT01086540) is a Phase 2 study with the main goal of changing the resistance in lung blood vessels. This will be measured by right heart catheterization after 24 weeks of treatment.

While the efficacy of the treatment is a primary objective, study researchers will also evaluate the safety of Rituxan in scleroderma patients and assess a number of other disease-related parameters, such as the time it takes for patients to get worse, number of new digital ulcers, severity of Raynaud phenomenon, exercise capacity, and quality of life. In addition, researchers will test lung function and evaluate biomarkers indicating disease progression.

Participants will be randomized to receive either Rituxan or placebo in a double-blind manner, meaning that neither patients nor study staff will be aware of which group a patient belongs to. All patients will continue taking their standard treatment — prostanoids, endothelin receptor antagonists, and phosphodiesterase 5 (PDE-5) inhibitors — during the trial.

The trial will also include a sub-study, called the RESTORE Sub-study, where researchers will measure changes in heart parameters using magnetic resonance imaging (MRI). These measurements will act as surrogates of right ventricle function and prognosis. Enrollment in the sub-study will be in parallel to that of the main trial.

Adult patients, ages 18-75, are eligible to enroll in the study. Participants need to have a New York Heart Association (NYHA) Functional Class II, III, or IV PAH, be able to do at least 100 meters on the 6-minute Walking Distance test, and must maintain oxygen saturation of at least 90% at rest, with or without oxygen.

Researchers also set up a list of factors excluding patients from eligibility. PAH patients not eligible for the study include those with PAH for more than five years, those with chronic infections or a history of certain serious infections, kidney disease, cancer within five years of the trial, low blood pressure, lung transplant, history of heart disease, and moderate or severe lung fibrosis.

Also, patients who have recently been treated with cyclophosphamide, immunocompromising biologic drugs or Remicade (infliximab), high-dose corticosteroids, and certain other immunosuppressants or immunomodulating drugs are also not allowed to participate. Patients who have ever been exposed to any treatment depleting lymphocytes or B-cells are not eligible either.

For a full list of inclusion and exclusion criteria, please visit the trial web page.

The trial will run at about two dozen locations across the U.S. For a full list of locations and contact details, please see the locations section of the trial web page. Patients or their physicians can contact the study staff to discuss details and the possibility of signing up for the study.

Researchers point out that the choice to participate in a clinical trial is an important personal decision, recommending that patients discuss the decisions with their doctor and family members before joining.