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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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

Other trials in United States

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.

Sep 1997?

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

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Enrolling by invitationPhase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingObservational study
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Acute Hepatic Porphyria
Active, not recruitingObservational study
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
RecruitingObservational study
Identification of Acute Intermittent Porphyria Modifying Genes
Acute Intermittent Porphyria (AIP)

All Porphyria trials

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 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.

Related trials for Porphyria

Enrolling by invitationPhase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingObservational study
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Acute Hepatic Porphyria
Active, not recruitingObservational study
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
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?

On the ClinicalTrials.gov record for NCT00004396.

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.