More Patients Needed
for Atenolol vs. Losartan Clinical Trial

by Hal Dietz, MD, and Ron Lacro, MD, Co-Principal Investigators

As we reach the midpoint of the atenolol vs. losartan clinical trial, it is essential that we provide you with this important update and, once again, request your participation. As of November 1, 2008, 315 of the 604 patients needed have been enrolled in the trial. The Data and Safety Monitoring Board of the Pediatric Heart Network (PHN), which reviews all the information collected from the study, meets every six months to discuss the status of the research. The Board met on October 27, 2008, and voted unanimously to continue the study. There weren’t any major safety concerns noted in the trial. Patients—even those less than a year of age—are tolerating the study medications well without any serious adverse effects.

Recruitment is going well, but maintaining the same pace of enrollment is absolutely required for us to complete this trial successfully and answer the question at hand. This may be challenging because many of the study sites have worked through their patient populations already. Failure of adequate enrollment will result in failure of the study. We need your help now.

Eight new enrollment sites have been added in the past year. This allows many patients access to an enrollment site that is closer to where they live. Please call the NMF (800-8-MARFAN, ext. 22) or visit the Clinical Trials section of the NMF website or PHN website (www.pediatricheartnetwork.com ) for the complete list of enrollment sites. Most important, please consider enrolling in the trial sooner rather than later if you or your child is eligible.

Please note that some people taking losartan outside of the context of the trial may not be receiving adequate dosing. Participation in the trial would still be open to such individuals, but would require stopping losartan and randomization to either losartan or atenolol. This would provide confidence that they would be receiving optimized dosing of one of the study medications.

In addition to participating in the primary atenolol vs. losartan study, patients have the opportunity to enroll in two significant ancillary studies. One is looking for genetic factors that influence the response to therapy, while the second one is trying to determine if losartan can influence musculoskeletal aspects of Marfan syndrome. This is our only opportunity to study these very important issues. Please ask about these studies and consider enrolling in them as well.

The NMF has successfully secured financial assistance to help with travel costs, echocardiogram costs (if your insurance provider will not allow out-of-network tests or for patients without insurance) and genetic testing costs, if required. Please contact the coordinators at the enrollment sites or the NMF directly to find out how to take advantage of these resources.

Please remember that the only data currently available on losartan are for mice with Marfan syndrome and a small number of people with very severe and quickly advancing Marfan syndrome. A randomized trial is the only way to know whether or not losartan is more effective than atenolol (or vice versa) in people with Marfan syndrome and whether or not specific subgroups will do better or worse on one medication or another. A randomized trial is also the most efficient (quickest) way to get the information we need to make the best treatment decisions.