Porphozym in the Treatment of Acute Attacks in AIP

NCT00418795 | Completed Phase 2

• 1 other identifier: rhPBGD-02
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

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.

Conditions

Acute Intermittent Porphyria

Keywords

Acute Intermittent Porphyria; treatment; Acute attack

Outcome Measures

Primary Outcomes (1)

• Change in Plasma PBG

Interventions

recombinant human porphobilinogen deaminase (Porphozym) DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed consent
• Confirmed diagnosis of AIP
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Zymenex A/S: lead

Study Sites (1)

Univercity Texas Medical Branch

Galveston, Texas, 77555-1109, United States

Location

MeSH Terms

Conditions

Porphyria, Acute Intermittent

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Christer Andersson, MD

  Umeå University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Expanded Access: Yes

Study Record Dates

• First Submitted: January 4, 2007
• First Posted: January 5, 2007
• Study Start: June 11, 2003
• Primary Completion: June 20, 2006
• Study Completion: June 20, 2006
• Last Updated: March 9, 2018

Record last verified: 2018-03

Locations

US (1)