The Foundation for Children with Atypical HUS

Announcement of aHUS Trial Results

Alexion Pharmaceuticals has announced interim results from its Phase 2 studies investigating Soliris® (eculizumab) as a treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS) for adolescent and adult patients in two trials: patients resistant to plasma therapy and patients on chronic plasma therapy. Abstracts summarizing these interim data have been posted on the web site of the American Society of Nephrology (ASN) at http://www.abstracts2view.com/asn (Patients/parents are welcome to register on this ASN site. Search KEYWORD: Thrombosis, links F-PO1274 and SA-FC406). These two trials are currently ongoing and data will be presented at the ASN annual meeting held November 18 - 21 in Denver, Colorado.

QUICK LINKS

[SA-FC406] Safety & Efficacy of Eculizumab in aHUS Patients Resistant to Plasma Therapy: Interim Analysis from a Phase II Trial

[F-PO1274] Safety & Efficacy of Eculizumab in aHUS Patients on Chronic Plasma Therapy: Interim Analysis of a Phase II Trial

Alexion has commenced a Phase 2, open-label, single-arm, multi-center study of eculizumab in pediatric patients with aHUS in the United States, European Union and Canada. Information about the trial, including when enrollment will begin, is posted to www.clinicaltrials.gov , Identifier Number NCT01193348. Physicians and families who are interested in participating in this clinical trial can learn more by contacting Alexion by e-mail at clinicaltrials@alxn.com , or by visiting the Alexion website at www.alexionpharma.com and clicking on the clinical trials link. QUICK LINK: http://www.clinicaltrials.gov/ct2/show/NCT01193348?term=ahus&ra...

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Tags: Alexion, Soliris, eculizumab, trials

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Comment by Bill Biermann on October 22, 2010 at 12:30pm
Alexion is doing well as a company financially also. That is good, because they should be able to help finaincially strapped families down the road as well !
Comment by Grace on October 21, 2010 at 7:52pm
Hooray! I like impressive results, including "5/7 dialysis pts became dialysis-free(P<0.01)" (from the plasma-resistant arm). Thanks for sharing.
Comment by Cheryl Biermann on October 21, 2010 at 7:29pm
Thank you for so quickly posting this information. Everyone, you should know, the administrator of this website, Linda Burke, has unbelievably quick turn-around times in getting this information to all of you!
Comment by Jonathan Aguallo on October 21, 2010 at 2:07pm
Great news!!!

WELCOME - Friends, Family Members, Patients, and Researchers - JOIN US!

The Foundation for Children with Atypical HUS encourages patients and investigators to share information and explore options/resources as we work together to gain insight into this rare complement disorder. By increasing contact opportunities with researchers and medical personnel interested in helping the aHUS community, our stories foster a better understanding of atypical hemolytic uremic syndrome.

Sharing information, inspiration and support for one another, we seek to gather together people and knowledge as we strive to improve the lives of patients and families dealing with a diagnosis of aHUS.

NEW DIAGNOSIS OF aHUS?
Be proactive! Get the medical basics of aHUS, what lab values to monitor, and areas of concern...check out the "aHUS Bootcamp" and "About aHUS" tabs at the top of this page!
If your doctor has never treated a case of aHUS, please print out our 'Doc to Doc Registry' and ask him/her to contact a physician versed in the complexities of aHUS and new options for 2011 genetic testing and treatment.

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Did you know...

CFH (Serum Complement Factor H) is a regulatory protein. The secreted protein product of CFH consists of 20 repetitive units named "short consensus repeats" or SCRs (each approximately 60 amino acids). In patients with aHUS the last 5 "pearls" in the twenty pearl strand protein, SCR16 - SCR20, should bind to protect cells but do not- they are defective in one or more of the last 5 SCR locations. If they cannot bind or stick to the kidney to protect that tissue, the platelets clump into clots that affect the glomeruli of the kidney -potentially causing acute renal failure.
  
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It is estimated that there are about 300 cases of aHUS in the U.S., and it is most common with young children. The condition is life threatening and either can be chronic or can recur at intervals.
  
more factoids...

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Your tax-deductible purchase of one or more bracelets will directly fund research to help end the tragedy and heartbreak that aHUS families live with every day. Each bracelet has an appraised value of $825, but your purchase price is only $295.

  

  
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