Acute Intermittent PorphyriaPhase 2/3NCT00418795
Porphozym in the Treatment of Acute Attacks in AIP
A Phase 2/3 study testing recombinant human porphobilinogen deaminase (Porphozym) in people with Acute Intermittent Porphyria, run by Zymenex A/S at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Intermittent Porphyria
- What is being tested?
- recombinant human porphobilinogen deaminase (Porphozym)
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jun 2003to 20 Jun 2006
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Univercity Texas Medical Branch
Galveston, Texas, 77555-1109
About this study
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
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.
Read the 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.
- Conditions
- Acute Intermittent Porphyria
- Being tested
- recombinant human porphobilinogen deaminase (Porphozym)
- Keywords
- Acute Intermittent Porphyria, treatment, Acute attack
- Medical subject headings
- Porphyria, Acute Intermittent
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant human porphobilinogen deaminase (Porphozym)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Plasma PBG
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2007 (estimated)
- Study start11 Jun 2003
- Primary completion20 Jun 2006
- Study completion20 Jun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Intermittent PorphyriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 36 participants
Sponsor and investigators
- Lead sponsor
- Zymenex A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Christer Andersson, MD, Principal investigator, Umeå University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00418795
- Sponsor's protocol ID
- rhPBGD-02
- Status last verified
- Mar 2018
- First submitted
- 4 Jan 2007
- Last updated
- 9 Mar 2018
- Results posted
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00418795
- Acronym / sponsor ID
- rhPBGD-02
- Conditions
- Acute Intermittent Porphyria
- Keywords
- Acute Intermittent Porphyria, treatment, Acute attack
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants
- Lead sponsor
- Zymenex A/SIndustry (pharma, biotech, devices)
- Study start
- 11 Jun 2003 (actual)
- Primary completion
- 20 Jun 2006 (actual)
- Study completion
- 20 Jun 2006 (actual)
- First posted
- 5 Jan 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| recombinant human porphobilinogen deaminase (Porphozym) | Drug | – |
Primary outcome
Change in Plasma PBG
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christer Andersson, MD principal investigator
Umeå University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing recombinant human porphobilinogen deaminase (Porphozym) in people with Acute Intermittent Porphyria, run by Zymenex A/S at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Plasma PBG. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2018.