PorphyriaPhase 1/2NCT00004789
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
A Phase 1/2 study testing heme arginate and tin mesoporphyrin in people with Porphyria, run by National Center for Research Resources (NCRR).
Trial summary
OBJECTIVES: I. Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria. II. Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients. III. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Porphyria
- What is being tested?
- heme arginate and tin mesoporphyrin
- How many people
- 59 participants
- Where
- No sites listed yet
- Study dates
- Jul 1993to not given
- Phase
- Phase 1/2
- 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
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Well-documented acute porphyria, i.e.: Acute intermittent porphyria Variegate porphyria Hereditary coproporphyria
--Patient Characteristics--
- No pregnant women
About this study
OBJECTIVES: I. Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria.
II. Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients.
III. Estimate the lowest effective dose of heme arginate (0.3, 1.0, or 3.0 mg/kg) in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission.
IV. Evaluate the safety and efficacy of tin mesoporphyrin used in combination with heme arginate in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission.
Read the detailed description
PROTOCOL OUTLINE: This project involves 4 studies: an unblinded multicenter treatment study, an unblinded prevention study, a controlled dose-ranging study, and a controlled drug combination dose-ranging study.
In the treatment study, participants with acute attacks of porphyria receive intravenous heme arginate daily for 4 days. Treatment may be extended to 7 days in patients with severe attacks and slow recovery; these data are analyzed separately.
In the prevention study, participants receive weekly infusions of heme arginate for 6 months. Treatment may be extended in selected cases; these data are analyzed separately. If an acute attack occurs, patients are treated with a standard course of heme arginate. Participants are followed weekly for 6 months after preventive treatment.
In the dose-ranging study, 4 groups of 3 patients are randomly assigned to standard and low-dose heme arginate infusions, administered daily for 4 days. After a washout of 4-12 weeks, patients are crossed to a second dose. In 2 of the 4 groups, patients cross between heme arginate and a normal saline control.
In the combination dose-ranging study, 4 groups of 3 patients are randomly assigned to 1 of 2 doses of tin mesoporphyrin. After a 4-12 week washout, these patients are crossed to a standard or low-dose heme arginate infusion. After a second 4-12 week washout, patients are given a combination infusion of tin mesoporphyrin followed by heme arginate.
All participants are given a special diet to prevent fluctuations in porphyrin precursors.
- Conditions
- Porphyria
- Being tested
- heme arginate, tin mesoporphyrin
- Keywords
- inborn errors of metabolism, acute porphyria, rare disease
- Medical subject headings
- Porphyrias, Metabolism, Inborn Errors, Porphyria, Acute Intermittent, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
heme arginateDrug
tin mesoporphyrinDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startJul 1993
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 1/2
- Primary purpose
- Treatment
- Enrolment
- 59 participants
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- University of Texas
- Investigators
- Karl Elmo Anderson, Study chair, University of Texas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004789
- Sponsor's protocol ID
- 199/11887
- Other IDs
- UTMB-398
- Status last verified
- Dec 2001
- First submitted
- 24 Feb 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
- ClinicalTrials.gov ID
- NCT00004789
- Acronym / sponsor ID
- 199/11887
- Conditions
- Porphyria
- Keywords
- inborn errors of metabolism, acute porphyria, rare disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- University of Texas
- Study start
- Jul 1993
- First posted
- 25 Feb 2000 (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.
OBJECTIVES: I. Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria.
II. Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients.
III. Estimate the lowest effective dose of heme arginate (0.3, 1.0, or 3.0 mg/kg) in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission.
IV. Evaluate the safety and efficacy of tin mesoporphyrin used in combination with heme arginate in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission.
Detailed description
PROTOCOL OUTLINE: This project involves 4 studies: an unblinded multicenter treatment study, an unblinded prevention study, a controlled dose-ranging study, and a controlled drug combination dose-ranging study.
In the treatment study, participants with acute attacks of porphyria receive intravenous heme arginate daily for 4 days. Treatment may be extended to 7 days in patients with severe attacks and slow recovery; these data are analyzed separately.
In the prevention study, participants receive weekly infusions of heme arginate for 6 months. Treatment may be extended in selected cases; these data are analyzed separately. If an acute attack occurs, patients are treated with a standard course of heme arginate. Participants are followed weekly for 6 months after preventive treatment.
In the dose-ranging study, 4 groups of 3 patients are randomly assigned to standard and low-dose heme arginate infusions, administered daily for 4 days. After a washout of 4-12 weeks, patients are crossed to a second dose. In 2 of the 4 groups, patients cross between heme arginate and a normal saline control.
In the combination dose-ranging study, 4 groups of 3 patients are randomly assigned to 1 of 2 doses of tin mesoporphyrin. After a 4-12 week washout, these patients are crossed to a standard or low-dose heme arginate infusion. After a second 4-12 week washout, patients are given a combination infusion of tin mesoporphyrin followed by heme arginate.
All participants are given a special diet to prevent fluctuations in porphyrin precursors.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| heme arginate | Drug | – |
| tin mesoporphyrin | Drug | – |
Primary outcome
Not reported in the source record.
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 24 Jun 2005.
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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.
Karl Elmo Anderson study chair
University of Texas
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing heme arginate and tin mesoporphyrin in people with Porphyria, run by National Center for Research Resources (NCRR).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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.
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 24 Jun 2005.