COMPOUND Q: NEW PROJECT INFORM COMMUNITY RESEARCH ALLIANCE STUDY

On March 8 the U. S. Food and Drug Administration approved a re-treatment study of compound Q (also called GLQ223, or tricho-santhin), to be administered by the Project Inform Community Research Alliance and conducted by physicians in four cities: San Francisco, Los Angeles, Miami, and New York. This trial is for patients who have been treated with the drug previously.

One hundred volunteers will be randomized into two groups, one to receive compound Q every three weeks, the other every six weeks, for up to six months. The dose will be 16 mcg/kg per administration. Unlike most clinical trials today, this one will allow patients to continue using other medications considered important for their health -- both for ethical reasons, and to obtain information about use of the drug under realistic conditions, not in a highly restricted test environment. This study, for example, will be the first to obtain practical knowledge about the combined use of compound Q and AZT.

A third group of patients, who meet the identical entry criteria (including previous use of compound Q) but do not choose to continue treatment at this time, can volunteer for a selfselected no-treatment group, which will also be followed under the protocol. (Note: as we go to press, the Institutional Review Board is still reviewing the study; there could be changes in the above design.)

The study director is Larry Waites, M. D., M. P. H., of San Francisco; the four principal investigators are Lysette Cardona, M. D., M. P. H., Los Angeles; Barbara Starrett, M. D., New York; Paula Sparti, M. D., Miami; and Alan S. Levin, M. D., San Francisco. Others, including Vera Byers, M. D., Ph.D., an expert in protein drugs, also contributed to the study design. The trial is financed in part by a grant of $250,000 from Sandoz, U. S. A. ; some community fundraising will also be necessary, as Project Inform and the local sites will have to absorb part of the cost. The drug is being contributed by Genelabs Inc., the biotechnology company in Redwood City, California, which developed the U. S. version of compound Q.

Comment

This study will not by itself be enough to lead to FDA approval of compound Q; other studies are now being run or designed by other researchers. The Project Inform Community Research Alliance study does, however, serve the following purposes:

* Many patients who have already used compound Q want to continue their treatment, and would do so with or without this program. The formal study provides the treatment without charge. At the same time, it collects systematic data which otherwise would be lost.

* Scientists usually want data from "randomized" trials -- that is, those which assign patients at random to two or more different treatment groups, which are later compared. Without randomization, it is impossible to be sure that all biases due to self-selection are accounted for, or even known. But randomization raises ethical concerns, as patients and their physicians often have good reason to choose one treatment or another, based on specifics of the individual case which are often not accounted for in the study design.

This trial provides a compromise. Volunteers who want to continue using the drug will be randomized between receiving treatment every three weeks or every six weeks. But the study designers could not have asked these volunteers, who had used compound Q before and had a very good idea of whether or not they wanted to continue, to be randomized between treatment and no treatment.

Having a self-selected no-treatment group is better than having none at all. Not only will it allow an admittedly imperfect comparison with the treatment groups, it will also provide systematic monitoring of long-term results of the compound Q which these volunteers had taken previously. And it will keep faith with those who volunteered for the original Project Inform compound Q study, by providing extensive monitoring free to them even if they do not want to continue the treatment.

* This study will not by itself lead to FDA approval, because it does not address the question of whether treatment or no treatment is best for patients who have never taken the drug before. But it will provide information on use of compound Q in real-world conditions -- unlike academic studies, which test drugs under conditions which seldom correspond to how patients are actually treated. For example, this study will allow volunteers to also use AZT, other antivirals, immune modulators, or other treatments which they and their physicians believe they should use. Therefore, it will provide information on the use of compound Q in combination with AZT.

For more information about this study, call the Project Inform hotline at 415/558-9051 (or toll-free at 800/334-7422 from within California, or 800/822-7422 from other states).

Note: The re-treatment study described in this article is only open to patients who have already been treated with compound Q. A separate study of the drug is taking place at other locations in California. For more information about that study, call the clinical trials referral hotline operated by the U. S.National Institute of Allergy and Infectious Diseases, toll-free at 800/TRIALS-A.