Porphozym in the Treatment of Acute Attacks in AIP
Primary Purpose
Acute Intermittent Porphyria
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
recombinant human porphobilinogen deaminase (Porphozym)
Sponsored by
About this trial
This is an interventional treatment trial for Acute Intermittent Porphyria focused on measuring Acute Intermittent Porphyria, treatment, Acute attack
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
Sites / Locations
- Univercity Texas Medical Branch
Outcomes
Primary Outcome Measures
Change in Plasma PBG
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00418795
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2018
Overall Recruitment Status
Completed
Study Start Date
June 11, 2003 (Actual)
Primary Completion Date
June 20, 2006 (Actual)
Study Completion Date
June 20, 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Zymenex A/S
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Intermittent Porphyria
Keywords
Acute Intermittent Porphyria, treatment, Acute attack
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
36 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
recombinant human porphobilinogen deaminase (Porphozym)
Primary Outcome Measure Information:
Title
Change in Plasma PBG
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christer Andersson, MD
Organizational Affiliation
Umeå University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Univercity Texas Medical Branch
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555-1109
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Porphozym in the Treatment of Acute Attacks in AIP
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