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Porphozym in the Treatment of Acute Attacks in AIP

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ClinicalTrials.gov Identifier: NCT00418795
Recruitment Status : Completed
First Posted : January 5, 2007
Last Update Posted : March 9, 2018
Sponsor:
Information provided by:
Chiesi Farmaceutici S.p.A.

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.

Condition or disease Intervention/treatment Phase
Acute Intermittent Porphyria Drug: recombinant human porphobilinogen deaminase (Porphozym) Phase 2 Phase 3

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

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  (Clinical Trial)
Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
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
Actual Study Start Date : June 11, 2003
Actual Primary Completion Date : June 20, 2006
Actual Study Completion Date : June 20, 2006





Primary Outcome Measures :
  1. Change in Plasma PBG


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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00418795     History of Changes
Other Study ID Numbers: rhPBGD-02
First Posted: January 5, 2007    Key Record Dates
Last Update Posted: March 9, 2018
Last Verified: March 2018

Keywords provided by Chiesi Farmaceutici S.p.A.:
Acute Intermittent Porphyria
treatment
Acute attack

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