Possible NF drugs / Clinical trials

<body topmargin=0 leftmargin=0 marginheight=0 marginwidth=0> <table cellpadding=0 cellspacing=0 border=0 height="100%"><tr> <td valign="top" width=200 BGCOLOR="#FFFFFF" TEXT="#080000" background="044d4c03.jpg"> <bR> <bR> <div> <A HREF="id23.htm" TARGET="_top" TITLE="Latest Research"><IMG SRC="064791f0.jpg" border=0 width="121" height="31" ALIGN="BOTTOM" HSPACE="0" VSPACE="0"></A></div> <bR> <div> <A HREF="id22.htm" TARGET="_top" TITLE="Help find a cure!"><IMG SRC="06a791b0.jpg" border=0 width="121" height="27" ALIGN="BOTTOM" HSPACE="0" VSPACE="0"></A></div> <bR> <div> <A HREF="http://www.pbgs2.com/cgi-bin/guestbook.cgi" TARGET="_blank" TITLE="http://www.pbgs2.com/cgi-bin/guestbook.c"><IMG SRC="0597a1f0.jpg" border=0 width="122" height="31" ALIGN="BOTTOM" HSPACE="0" VSPACE="0"></A></div> </td> <td valign="top" width="100%" BGCOLOR="#FFFFFF" TEXT="#080000"> <div> <FONT SIZE="5" COLOR="#FFCC33" FACE="Arial" ><B>NF TEAM</B></FONT> <FONT SIZE="1" COLOR="#333399" FACE="Arial" >   |     <A 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<div> <B><U>Clinical Trials</U></B></div> <div> <I>Sponsored by</I></div> <div> <A HREF="http://www.nci.nih.gov/" TARGET="_blank" TITLE="http://www.nci.nih.gov/"><B>National Cancer Institute (NCI)</B></A></div> <div> Protocol Number: 04-C-0080</div> <div> <B>Title: </B><FONT SIZE="2" COLOR="#000000" FACE="Arial" ><B>Pirfenidone in Children and Young Adults with Neurofibromatosis type I and Progressive Plexiform Neurofibromas</B></FONT></div> <bR> <div> <B>View Summary below </B><B>or</B><B> click</B><A HREF="http://clinicalstudies.info.nih.gov/detail/A_2004-C-0080.html" TARGET="_blank" TITLE="http://clinicalstudies.info.nih.gov/deta"><B> </B></A><A HREF="http://clinicalstudies.info.nih.gov/detail/A_2004-C-0080.html" TARGET="_blank" TITLE="http://clinicalstudies.info.nih.gov/deta"><B>HERE</B></A><B> for the complete article</B></div> <div> <B>Summary: </B>This study will determine 1) if the experimental drug pirfenidone can slow the growth of plexiform neurofibromas in patients with neurofibromatosis type I (NF1); 2) if pirfenidone can cause progressive plexiform neurofibromas to shrink; and 3) what side effects pirfenidone may cause in children and young adults with NF1. NF1 is a genetic disease in which patients can develop neurofibromas (tumors involving a nerve) or plexiform neurofibromas (tumors involving a nerve and its branches). The only effective treatment for plexiform neurofibromas is surgery; however, often it is not possible to surgically remove all the tumors because of their location. Pirfenidone has been effective in treating several fibrosing (scarring) conditions. In addition, because the drug targets the same growth factors that are present in plexiform neurofibromas, it may also be effective in controlling tumors in patients with NF1.</div> <bR> <div> Patients between 3 and 21 years of age with NF1 and progressive plexiform neurofibromas whose tumors cannot be completely removed surgically or who refuse surgical treatment may be eligible for this study. Candidates will be screened with a medical history and physical examination, blood and urine tests, magnetic resonance imaging of the neurofibromas, and tumor biopsy if medically indicated.</div> <bR> <div> Study participants will take pirfenidone by mouth 3 times a day every day for 28 days. This constitutes one 28-day treatment cycle. The drug dose during the first week of the trial will be lower than the normal dose to help prevent nausea. Patients (or their parents) will record the time each dose is taken, any drug side effects, and any other drugs taken besides the study drug. Patients whose tumor remains stable or shrinks with pirfenidone treatment may continue treatment as long as the drug is well tolerated. Those whose tumor grows during the study will be taken off the study.</div> <bR> <div> In addition to drug therapy, participants will undergo the following tests and procedures:</div> <bR> <div> -Blood tests to monitor drug side effects before cycles 2, 3, 4, 7, and 10, and at the completion of every 6 cycles after that</div> <bR> <div> -Physical examinations at the same times as the blood tests, but at intervals of every three cycles after cycle 10</div> <bR> <div> -MRI scans to measure the size of the tumors before treatment starts, again before cycles 4, 7, and 10, and every six cycles after that</div> <bR> <div> -For patients 18 years old and younger, quality of life questionnaires before treatment starts, before cycle 4, and then after completion of every 6 cycles</div> <bR> <div> Tumor tissue samples may also be obtained from patients scheduled to have a biopsy (surgical removal of a sample of tumor tissue for examination) or surgery as part of their medical management. The samples will be analyzed for cell components of plexiform neurofibromas. Information from these studies may help researchers determine which patients may benefit from treatment with pirfenidone. </div> <div> ****************************************************************************************</div> <div> <B>National Institutes of Health / Clinical Research Studies</B></div> <DIV ALIGN="LEFT"></DIV> <div> Protocol Number: 01-C-0222 </div> <bR> <div> <B>Title: </B>A Phase II Randomized, Cross-Over, Double-Blinded, Placebo-Controlled Trial of the Farnesyltransferase Inhibitor R115777 in Pediatric Patients with Neurofibromatosis Type I and Progressive Plexiform Neurofibromas </div> <bR> <div> <B>View Summary below or click </B><A HREF="http://clinicalstudies.info.nih.gov/detail/A_2001-C-0222.html" TARGET="_blank" TITLE="http://clinicalstudies.info.nih.gov/deta"><B>HERE</B></A><B> for the complete article</B></div> <div> <B>Summary: </B>This study will examine whether the experimental drug R115777 can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects are related to treatment. Plexiform tumors arise from nerves; the only effective treatment is surgical removal. Often, however, not all the tumors can be removed, because of their number or location. </div> <div> Patients with NF1 have a reduced amount of the protein neurofibrin. Neurofibrin is thought to help control the activity of another protein, called ras, which regulates cell growth. Too little neurofibrin, therefore, may allow for uncontrolled cell growth and tumor formation. R115777 interferes with the function of the ras and other proteins. In test tube and animal studies, R115777 has blocked the growth of cancer cells. This study will examine whether the drug is effective against plexiform tumors. </div> <div> Patients with NF1 and progressive plexiform neurofibromas between 3 and 25 years of age may be eligible for this study. Patients whose tumors can be successfully removed surgically may not participate in this study. Candidates will have a medical history and physical and eye examinations, blood and urine tests, and magnetic resonance imaging (MRI). Photographs will be taken of tumors visible on the body surface.</div> <div> Study participants will be randomly assigned to receive either R115777 or placebo (an inactive substance). They will take R115777 or placebo tablets every 12 hours for 21 days, followed by a 7-day rest period. This constitutes one 28-day treatment cycle. Treatment will continue as long as the tumors remain stable or shrink and side effects are tolerable. The treatment will be switched (for example, from placebo to R115777) or stopped if the tumors grow and will be stopped if side effects are unacceptable. Patients (or their parents) will be asked to keep a record of side effects. </div> <div> For the first 3 months of treatment, patients will have a physical examination and blood tests every other week. Blood tests will also be done before cycles 3, 6, 9 and 12 to measure protein levels and the level of a substance called nerve growth factor. MRI scans will be done periodically throughout treatment to measure the size of the tumors. Cells will be collected from inside the cheek before the first treatment dose and at one point in time after at least 14 days of treatment on each study phase (R115777 or placebo). A tissue biopsy (surgical removal of a small tumor tissue sample) will be requested before treatment begins and again at least 2 weeks after treatment starts, if tumor nodules are easily accessible for biopsy. Tumor samples may also be obtained from tissue removed from patients who must undergo surgery for medical management of their disease. The tissue samples will be analyzed for changes in the NF1 and ras genes and in the ras protein.</div> <div> Patients (or their parents) will also be asked periodically throughout the course of treatment to fill out questionnaires assessing quality of life. </div> <DIV ALIGN="LEFT"></DIV> <div> <A HREF="index.htm" TARGET="_top" TITLE="NF TEAM"><IMG SRC="05257250.jpg" border=0 width="87" height="37" ALIGN="BOTTOM" HSPACE="0" VSPACE="0"></A></div> <bR> <bR> </td> </tr></table></body>