Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

• Register: ClinicalTrials.gov
• Last refreshed on: 10 April 2023
• Main ID: NCT02180412
• Date of registration: 25/06/2014
• Prospective Registration: No
• Primary sponsor: The University of Texas Medical Branch, Galveston
• Public title: Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria
• Scientific title: A Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of Porphyria
• Date of first enrolment: April 28, 2014
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02180412
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Karl E Anderson, MD
• Affiliation: UT, Galveston

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female aged 18 years

- Willing to provide written informed consent

- Acute symptoms (7 days duration or less to time of enrollment) such as abdominal, back
and/or limb pain, diagnosed by the investigator as caused by porphyria after initial
evaluation has excluded other causes.

- Diagnosis of acute porphyria documented by a substantial increase in urinary or serum
porphobilinogen (PBG).

- Type of acute porphyria confirmed by additional testing (in addition to increased
PBG), which may be completed before or after treatment begins using pretreatment
samples:

- For acute intermittent porphyria (AIP): Normal or only slight increases in plasma and
fecal porphyrins. Most (~90 percent) will have deficient activity of erythrocyte
porphobilinogen deaminase (PBGD), and almost all (>95 percent) will have a
demonstrable disease-causing PBGD mutation.

- For hereditary coproporphyria (HCP): Substantial increases in fecal porphyrins (almost
entirely coproporphyrin III). In the absence of skin photosensitivity, most will have
normal or only slight increases in plasma porphyrins. Almost all (>95 percent) will
have a demonstrable disease-causing coproporphyrinogen oxidase (CPO) mutation.

- For variegate porphyria (VP): Substantial increases in fecal porphyrins (mostly
coproporphyrin III and protoporphyrin), increased plasma total porphyrins and a
fluorescence emission maximum of diluted plasma at neutral pH near 626 nm. Almost all
(~95 percent) will have a demonstrable disease-causing protoporphyrinogen oxidase
(PPO) mutation.

Exclusion Criteria:

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

- Therapy with hemin within 7 days prior to enrollment in this study

- Known or suspected allergy to Panhematin™ or related products

- Preexisting coagulation defect or concurrent treatment with an anticoagulant

- Previously documented renal impairment defined as a serum creatinine above 1.7 mg/dL
or 150 mmol/L.

- A diagnosis of diabetes mellitus, which might increase the risk of glucose infusion.

- Heart failure, significant chronic anemia or any disease or condition that the
investigator judges would lead to an unacceptable risk to the patient or interfere
with the successful collection of date for the trial

- Previous randomization in this trial



Age minimum: 18 Years
Age maximum: 100 Years
Gender: All

Health Condition(s) or Problem(s) studied

Acute Porphyrias

Intervention(s)

Biological: Panhematin

Other: Glucose

Primary Outcome(s)

Pain scale [Time Frame: 4 days]

Secondary Outcome(s)

Biochemical effects of Panhematin [Time Frame: 4 days]

Secondary ID(s)

10-203

FD-R-03720

Source(s) of Monetary Support

Please refer to primary and secondary sponsors