Porphozym in the Treatment of Acute Attacks in AIP

This study has been completed.
Sponsor:
Information provided by:
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT00418795
First received: January 4, 2007
Last updated: NA
Last verified: January 2007
History: No changes posted
  Purpose

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.


Condition Intervention Phase
Acute Intermittent Porphyria
Drug: recombinant human porphobilinogen deaminase (Porphozym)
Phase 2

Zymenex A/S has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.  

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Zymenex A/S:

Primary Outcome Measures:
  • Change in Plasma PBG

Estimated Enrollment: 36
Study Start Date: February 2003
Estimated Study Completion Date: July 2006
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00418795

Locations
United States, Texas
Univercity Texas Medical Branch
Galveston, Texas, United States, 77555-1109
Sponsors and Collaborators
Zymenex A/S
Investigators
Principal Investigator: Christer Andersson, MD Umeå University
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00418795     History of Changes
Other Study ID Numbers: rhPBGD-02
Study First Received: January 4, 2007
Last Updated: January 4, 2007
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Sweden: Medical Products Agency
Norway: Norwegian Medicines Agency
Denmark: Danish Medicines Agency
Finland: Finnish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Zymenex A/S:
Acute Intermittent Porphyria
treatment
Acute attack

Additional relevant MeSH terms:
Porphyrias
Porphyria, Acute Intermittent
Porphyrias, Hepatic
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Skin Diseases, Metabolic
Skin Diseases
Metabolic Diseases
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on October 19, 2014