Porphozym in the Treatment of Acute Attacks in AIP

Brief Title

Porphozym in the Treatment of Acute Attacks in AIP

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

Brief Summary

      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.
    

Detailed Description

      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.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Change in Plasma PBG


Condition

Acute Intermittent Porphyria

Intervention

recombinant human porphobilinogen deaminase (Porphozym)


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

36

Start Date

June 11, 2003

Completion Date

June 20, 2006

Primary Completion Date

June 20, 2006

Eligibility 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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Christer Andersson, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00418795

Organization ID

rhPBGD-02



Study Sponsor

Zymenex A/S


Study Sponsor

Christer Andersson, MD, Principal Investigator, Umeå University


Verification Date

March 2018