ALS Clinical Trials May-June 2010

by ALSN Staff on Sat, 2010-05-01 11:54
Article Highlights:

Latest information on ALS-specific clinical trials as of April:

Cytokinetics seeks participants for CK-2017357 study

Cytokinetics, a South San Francisco biotechnology company, is seeking participants with ALS for a trial of its experimental compound CK-2017357.

CK-2017357 increases the senstivity of muscle fibers to calcium, resulting in a potential increase in muscle force generation. In March 2010, the compound was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of ALS. (Orphan Drug designation gives a company some financial incentives to develop drugs for rare diseases.)

At least 36 and up to 72 participants at four to five sites will be enrolled in this trial, the primary objective of which is to evaluate the effects of CK-2017357 on measures of skeletal muscle function and muscle fatigability in people with ALS.

Contact Katy Shaver at Massachusetts General Hospital at kshaver@partners.org. This study also is listed at www.clinicaltrials.gov.

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Study of arimoclomol in SOD1-related ALS seeks participants

Investigators at Emory University in Atlanta and Massachusetts General Hospital in Boston are seeking participants for their trial of the experimental drug arimoclomol in people with familial ALS caused by certain mutations in the SOD1 gene.

Arimoclomol, developed by CytRx Corp. of Los Angeles, is believed to increase the action of “molecular chaperones,” which may protect cells from abnormal proteins, such as toxic SOD1.

Participants must have the type of ALS that runs in families and be willing to undergo genetic testing for ALS and to learn the results. Study participants who have specific SOD1 gene mutations and who meet other study criteria will be randomly assigned to receive either arimoclomol or a placebo for a period of one year.

The trial requires two visits to a study center at the start of the trial and another visit two months later. All other study procedures are completed in the home.

For the Emory site, contact Margaret Walker at (404) 712-8578 or mlwalke@emory.edu or sod1@emory edu. For the Massachusetts General site, contact Darlene Pulley at (617) 726-6190 or dpulley@partners.org. This trial also is posted at www.clinicaltrials.gov.

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Investigators at Emory studying those at risk of developing ALS

Investigators at Emory University in Atlanta are seeking participants for their long-term study of people who are at risk for developing ALS. “At risk” is defined as individuals who have at least two family members with ALS and have a mutation (genetic change) in a gene that is known to cause ALS, such as SOD1, TDP43, FUS or others. Contact Sue Gronka at (404) 727-3813 or sgronka@emory.edu or fals@emory.edu. This study also is posted at www.clinicaltrials.gov.

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Zenvia continues to improve emotional symptoms in ALS

The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes in people with the disease.

The results were seen in an extension to the company’s phase 3 clinical trial, which concluded last fall. Avanir presented its results April 13, 2010, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto.

Designed to treat pseudobulbar affect (PBA) — uncontrollable episodes of laughing or crying caused by neurological abnormalities — Zenvia is a combination of two existing medications, dextromethorphan and quinidine. (The drug formerly was called Neurodex.)

In fall 2009, Avanir announced encouraging results for its phase 3 trial of Zenvia in people with ALS or multiple sclerosis (MS).

In the phase 3 trial, approximately 300 participants with PBA secondary to either ALS or MS were randomly assigned to receive Zenvia capsules containing 30 milligrams of dextromethorphan and 10 milligrams of quinidine; Zenvia capsules containing 20 milligrams of dextromethorphan and 10 milligrams of quinidine; or placebo capsules. All participants took the capsules orally twice a day for 12 weeks.

At the end of the 12-week, placebo-controlled trial, the investigators found both Zenvia dosages significantly reduced the number of laughing and/or crying episodes compared to the placebo, and that the drug was generally safe and well-tolerated. The results announced at the AAN meeting are from a 12-week, open-label extension trial of Zenvia.

In this extension study, people with ALS or MS who had participated in the phase 3 trial were invited to take the 30-milligram dextromethorphan formulation of Zenvia (the higher of the two dextromethorphan doses) on an open-label basis for an additional 12 weeks. In an open-label trial, participants and investigators know what everyone is taking. The company said those taking Zenvia in the extension trial continued to benefit from the drug.

Avanir plans to move forward with its application to the U.S. Food and Drug Administration (FDA) seeking market approval for Zenvia to treat PBA. It expects a decision from the FDA on this application in late 2010.

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ALSN Staff
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