Viewing Study NCT00418795



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Study NCT ID: NCT00418795
Status: COMPLETED
Last Update Posted: 2018-03-09
First Post: 2007-01-04

Brief Title: Porphozym in the Treatment of Acute Attacks in AIP
Sponsor: Zymenex AS
Organization: Chiesi Farmaceutici SpA

Study Overview

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
Status: COMPLETED
Status Verified Date: 2018-03
Last Known Status: None
Delayed Posting: No
If Stopped, Why?: Not Stopped
Has Expanded Access: True
If Expanded Access, NCT#: N/A
Has Expanded Access, NCT# Status: N/A
Acronym: None
Brief Summary: A multi-centre double-blind randomized placebo controlled parallel group trial investigating the efficacy and safety of Porphozym recombinant human porphobilinogen deaminasein the treatment of acute attacks in AIP
Detailed Description: The primary objective is To investigate the biochemical efficacy on plasma porphobilinogen PBG of Porphozyrecombinant 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 drugis supplied by Zymenex AS 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

Study Oversight

Has Oversight DMC: None
Is a FDA Regulated Drug?: None
Is a FDA Regulated Device?: None
Is an Unapproved Device?: None
Is a PPSD?: None
Is a US Export?: None
Is an FDA AA801 Violation?: None