Panhematin for Prevention of Acute Attacks of Porphyria

• Register: ClinicalTrials.gov
• Last refreshed on: 20 February 2023
• Main ID: NCT02922413
• Date of registration: 01/01/2016
• Prospective Registration: No
• Primary sponsor: The University of Texas Medical Branch, Galveston
• Public title: Panhematin for Prevention of Acute Attacks of Porphyria
• Scientific title: Safety and Efficacy of Panhematin™ for Prevention of Acute Attacks of Porphyria
• Date of first enrolment: October 30, 2015
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02922413
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Prevention. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Karl E Anderson, MD
• Affiliation: University of Texas

• Name: Karl E Anderson, MD

• Name: Karl E Anderson, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female aged 18 years

2. Willing to provide written informed consent

3. A diagnosis of acute intermittent porphyria, hereditary coproporphyria or variegate
porphyria confirmed by the following criteria, which are based on the criteria for
enrollment in the Longitudinal Study of the Porphyrias Consortium. For each type of
porphyria, the inclusion criteria are based on 1) clinical features, 2) biochemical
findings, as documented by laboratory reports (or copies) of porphyria-specific
testing, and 3) molecular studies to identify a mutation in a porphyria-related gene.
Equivocal biochemical measurements may require confirmatory testing. Testing for a
disease-causing mutation must be attempted, but an identified mutation is not
essential for enrollment, since it is known that a mutation cannot be found in a small
fraction (<5%) of biochemically proven cases of porphyria. Subjects will (1.) have had
frequent attacks in the past, with symptoms such as abdominal, back and/or limb pain
and diagnosed after exclusion of other causes, and (2.) be on hemin prophylaxis for
prevention of frequent attacks. It is expected that they will have had 6 or more
attacks in one year before starting hemin prophylaxis.

Exclusion Criteria:

1. Symptoms such as abdominal, back or limb pain are explained by another condition, as
judged by the investigator

2. Known or suspected allergy to Panhematin™ or related products

3. A known or suspected allergy to human albumin

4. Any disease or condition that the investigator judges would lead to an unacceptable
risk to the patient or interfere with the successful collection of data for the trial

5. Previous randomization in this trial



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Acute Intermittent Porphyria

Variegate Porphyria

Hereditary Coproporphyria

Intervention(s)

Other: Placebo

Biological: Hemin for injection

Primary Outcome(s)

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [Time Frame: 1-4 weeks]

Occurrence of an acute attack of porphyria after treatment [Time Frame: 1-4 weeks]

Secondary Outcome(s)

Effects on levels of porphobilinogen [Time Frame: 1-4 weeks]

Secondary ID(s)

FD-R-03720

14-0188

Source(s) of Monetary Support

Please refer to primary and secondary sponsors