Acute Intermittent Porphyria Clinical Trial
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
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.
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.
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