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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)

About this trial

This is an interventional treatment trial for Acute Intermittent Porphyria focused on measuring Acute Intermittent Porphyria, treatment, Acute attack

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

NCT ID

NCT00418795

First Posted

January 4, 2007

Last Updated

March 7, 2018

Sponsor

Zymenex A/S

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

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

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Porphozym in the Treatment of Acute Attacks in AIP

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