Porphozym in the Treatment of Acute Attacks in AIP

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00418795
• Date of registration: 04/01/2007
• Prospective Registration: No
• Primary sponsor: Zymenex A/S
• Public title: Porphozym in the Treatment of Acute Attacks in AIP
• Scientific 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
• Date of first enrolment: June 11, 2003
• Target sample size: 36
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00418795
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double.
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

• Name: Christer Andersson, MD
• Affiliation: Umeå University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Informed consent

- Confirmed diagnosis of AIP

- Previous attacks with PBG above the reference level of the laboratory AND exclusion of
variegate porphyria (florescence emission of plasma samples is maximal at 626 nm in
VP) AND exclusion of hereditary coproporphyria (HCP) (increased ratio of fecal
coproporphyrin III to coproporphyrin I found in HCP)

- Acute attack of AIP verified by presence of abdominal and/or back and/or limb pain,
diagnosed by the investigator as being caused by AIP

- Urine PBG above 6 mmol/mol creatinine (5 times upper reference level of the central
laboratory)

- Male or female aged above 18 year

Exclusion criteria are:

- First acute attack in AIP

- Other reasons for abdominal and/or back and/or limb pain as judged by the investigator

- Therapy with human hemin within 7 days prior to administration of trial drug

- Treatment with any investigational drug within 4 weeks prior to this trial

- Known or suspected allergy to the trial product or related products

- Pregnant or breast-feeding women and women who intend to become pregnant prior to or
during the trial

- Women of child-bearing potential who are not using acceptable methods of contraception
(systemic contraception, IUD, barrier method or GnRH analogues)

- Previous documented renal impairment defined as above 150 mmol/L or 1.7 mg/dL serum
creatinine, indicating a reduction in kidney function of 50% or more

- Any disease or condition that the investigator judges would interfere with the trial

- Previous randomization in this trial



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Acute Intermittent Porphyria

Intervention(s)

Drug: recombinant human porphobilinogen deaminase (Porphozym)

Primary Outcome(s)

Change in Plasma PBG

Secondary ID(s)

rhPBGD-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors