Porphozym in the Treatment of Acute Attacks in AIP
A multi-centre, double-blind, randomized, placebo controlled, parallel group trial, investigating the efficacy and safety of Porphozym (recombinant human porphobilinogen deaminase)in the treatment of acute attacks in AIP.
Acute Intermittent Porphyria
Drug: recombinant human porphobilinogen deaminase (Porphozym)
Zymenex A/S has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Primary Purpose: Treatment
|Official Title:||A Multi-Centre, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Porphozym(Recombinant Human Porphobilinogen Deaminase) in the Treatment of Acute Attacks in AIP|
- Change in Plasma PBG
|Study Start Date:||February 2003|
|Estimated Study Completion Date:||July 2006|
The primary objective is: To investigate the biochemical efficacy on plasma porphobilinogen (PBG) of Porphozy(recombinant human porphobilinogen deaminase) in subjects with Acute Intermittent Porphyria (AIP) during an attack and the clinical efficacy clinical efficacy of Porphozym™, being the change in pain from baseline to 24 hours after start of treatment. The correlation between the biochemical and clinical efficacy is investigated as well. Further the safety of Porphozym™ is evaluated.
After a screening period lasting as short as possible subjects enrolled in the trial will be randomized to treatment with either Porphozym™ or placebo. Treatment is given over 48 hours. After end of treatment, the subject enters the observation period, which lasts until the discharge from the hospital. Subjects are followed up with visits 14 and 28 days after end of treatment. Additional safety follow-up will be performed 2, 4 and 6 months after end of treatment. At least 36 Subjects will be enrolled in the trial.
The trial drug,is supplied by Zymenex A/S, Denmark in vials for reconstitution in water for injections (WFI).
At start of treatment a bolus injection iv is given to decrease PBG levels ot zero. This is followed by continuous iv infusion of the enzyme over the following 48 hours.