Porphozym in the Treatment of Acute Attacks in AIP

March 7, 2018 updated by: Zymenex A/S

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment

36

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Galveston, Texas, United States, 77555-1109
        • Univercity Texas Medical Branch

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: DOUBLE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Change in Plasma PBG

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Zymenex A/S

Investigators

  • Principal Investigator: Christer Andersson, MD, Umea University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

June 11, 2003

Primary Completion (ACTUAL)

June 20, 2006

Study Completion (ACTUAL)

June 20, 2006

Study Registration Dates

First Submitted

January 4, 2007

First Submitted That Met QC Criteria

January 4, 2007

First Posted (ESTIMATE)

January 5, 2007

Study Record Updates

Last Update Posted (ACTUAL)

March 9, 2018

Last Update Submitted That Met QC Criteria

March 7, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • rhPBGD-02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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