PorphyriaPhase 2NCT00004396
Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias
A Phase 2 study testing heme arginate and tin mesoporphyrin in people with Porphyria, run by National Center for Research Resources (NCRR) at 2 sites in United States.
Trial summary
OBJECTIVES: I. Compare the efficacy of heme arginate, singly or in combination with tin mesoporphyrin, in lowering porphyrin precursors in patients with asymptomatic acute intermittent porphyria. II. Evaluate and compare the safety and tolerability of these treatment regimens in this patient population. 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
- 32 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 1997to not given
- Phase
- Phase 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--
- Documented acute intermittent porphyria in remission for at least 1 month
- Increased excretion of porphyrin precursors
- Absence of neurovisceral symptoms due to porphyria for at least 1 month
--Prior/Concurrent Therapy--
- At least 1 month since prior heme preparation therapy
- Concurrent medication for coexisting condition is allowed, as long as dose and pattern of administration is held constant during study
--Patient Characteristics--
- Renal: Urinary porphobilinogen excretion of at least 20 mg every 24 hours
- Other: Not pregnant Fertile female patients must use effective contraception during and at least 6 months before study At least 1 month since any symptoms of disease No concurrent acute hemorrhagic disorder such as: Gastrointestinal bleeding Intracerebral hemorrhage No known hypersensitivity to heme arginate, tin mesoporphyrin, another heme preparation, or related heme analogue No other condition that may increase risk to patient
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Texas Medical Branch
Galveston, Texas, 77555-0209
Rockefeller University Hospital
New York, New York, 10021-6399
About this study
OBJECTIVES: I. Compare the efficacy of heme arginate, singly or in combination with tin mesoporphyrin, in lowering porphyrin precursors in patients with asymptomatic acute intermittent porphyria.
II. Evaluate and compare the safety and tolerability of these treatment regimens in this patient population.
Read the detailed description
PROTOCOL OUTLINE: This is an unblinded, dose ranging study. Patients receive heme arginate alone or in combination with tin mesoporphyrin.
Patients receive tin mesoporphyrin IV as a single dose. Heme arginate is administered as a single intravenous infusion.
Patients are entered in cohorts of 4. Subsequent cohorts of 4 patients each receive escalating doses of tin mesoporphyrin in combination with 1 of 2 different dosages of heme arginate. Subjects must maintain a constant diet.
Patients experiencing adverse reactions are followed as clinically indicated.
- Conditions
- Porphyria
- Being tested
- heme arginate, tin mesoporphyrin
- Keywords
- inborn errors of metabolism, porphyria, rare disease
- Medical subject headings
- Porphyrias, Metabolism, Inborn Errors, 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 posted19 Oct 1999 (estimated)
- Study startSep 1997
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 2
- Primary purpose
- Treatment
- Enrolment
- 32 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
- NCT00004396
- Sponsor's protocol ID
- 199/13185
- Other IDs
- UTMB-96-476, UTMB-96-318, UTMB-FDR000710, UTMB-FDR001459
- Status last verified
- Dec 2003
- First submitted
- 18 Oct 1999
- 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
- Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias
- ClinicalTrials.gov ID
- NCT00004396
- Acronym / sponsor ID
- 199/13185
- Conditions
- Porphyria
- Keywords
- inborn errors of metabolism, porphyria, rare disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- University of Texas
- Study start
- Sep 1997
- First posted
- 19 Oct 1999 (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. Compare the efficacy of heme arginate, singly or in combination with tin mesoporphyrin, in lowering porphyrin precursors in patients with asymptomatic acute intermittent porphyria.
II. Evaluate and compare the safety and tolerability of these treatment regimens in this patient population.
Detailed description
PROTOCOL OUTLINE: This is an unblinded, dose ranging study. Patients receive heme arginate alone or in combination with tin mesoporphyrin.
Patients receive tin mesoporphyrin IV as a single dose. Heme arginate is administered as a single intravenous infusion.
Patients are entered in cohorts of 4. Subsequent cohorts of 4 patients each receive escalating doses of tin mesoporphyrin in combination with 1 of 2 different dosages of heme arginate. Subjects must maintain a constant diet.
Patients experiencing adverse reactions are followed as clinically indicated.
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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ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
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Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
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A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
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RecruitingObservational study
Identification of Acute Intermittent Porphyria Modifying Genes
Acute Intermittent Porphyria (AIP)
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
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing heme arginate and tin mesoporphyrin in people with Porphyria, run by National Center for Research Resources (NCRR) at 2 sites 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?
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.
Where is the study taking place?
The record lists 2 sites 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 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.
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.