Dichlorphenamide vs. Placebo for Periodic Paralysis

• Research type

  Research Study

• Full title

  Double-blind, Placebo-controlled, Parallel Group, Phase III Study Comparing Dichlorphenamide vs. Placebo for the Treatment of Period Paralysis

• IRAS ID

  21431

• Contact name

  Michael Hanna

• Sponsor organisation

  University of Rochester School of Medicine

• Eudract number

  2008-002306-19

• ISRCTN Number

  N/A

• Research summary

  This study aims to compare the effects of dichlorphenamide vs. placebo in patients with hyperkalaemia (HYP)and hypokalaemia (HOP)periodic paralysis in lowering the rate of attacks of weakness as measured by participant self-report over the last 8 weeks of a 9-week double-blind study.The periodic paralyses (PP) are muscle channelopathies that although generally neither fatal nor life-shortening, cause major disability.The recognition and diagnosis of the periodic paralyses has been simplified by discovery of the molecular lesions that cause the diseases, but no standardized treatment has been developed.The primary periodic paralysis, hyperkalemic periodic paralysis (HYP) and hypokalemic periodic paralysis (HOP) are characterised by attacks of transient flaccid paralysis. The attacks typically follow rest, sleep or antecedent exercise. Inter-attack strength is usually considered normal early in the disease but persistent progressive weakness develops in most patients.Dichlorphenamide (DCP), a benzenedisulfonamide is approved as a treatment for decreasing intraocular pressure in the treatment of glaucoma. Previous trials have used DCP for the treatment of period paralysis and preliminary evidence have shown that DCP is effective in the prevention of episodic weakness in both HYP and HOP (Tawil et al., 2000).The study includes two arms, one each for HYP and HOP participants. The 9-week studies will investigate the prevention of attacks of weakness. The 9-week studies will be followed by 1-year open-label extension without placebo to compare the long-term effects of DCP on the course of the disease and on inter-attack weakness.The total study duration will be a maximum of 61 weeks on treatment.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  09/H0714/53

• Date of REC Opinion

  29 Dec 2009

• REC opinion

  Further Information Favourable Opinion