Panhematin for Prevention of Acute Attacks of Porphyria

NCT02922413 | Terminated Phase 2 Results DMC

• 2 other identifiers: 14-0188FD-R-03720
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of porphyria. High quality studies have not been done previously for treating or preventing acute attacks with hemin. The lack of strong evidence for efficacy of hemin for treatment and prevention of attacks limits its availability for patients with acute porphyrias. Funding source: FDA Office of Orphan Product Development (FDA OOPD) FD-R-03720

Conditions

Hereditary Coproporphyria; Variegate Porphyria; Acute Intermittent Porphyria

Keywords

Porphyria; Heme

Outcome Measures

Primary Outcomes (2)

• Number of Participants Experiencing Acute Attacks of Porphyria After Treatment

  Participants were interviewed weekly by telephone to record the number of acute porphyria attacks after initial treatment with Panhematin or placebo.

  1-4 weeks

• Number of Participants With Treatment-Related Serious Adverse Events

  The number of participants who experience treatment-related serious adverse events (SAEs) as assessed and graded using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

  1-4 weeks

Other Outcomes (4)

• Effects on Levels of Porphobilinogen

  1-4 weeks

• Effects of Age

  1-4 weeks

• Effects of the Nature of the Porphobilinogen Deaminase (PBGD) Mutation

  1-4 weeks

Study Arms (2)

Hemin for injection

EXPERIMENTAL

Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour.

Biological: Hemin for injection

Placebo

PLACEBO COMPARATOR

A double blind dose of saline.

Other: Placebo

Interventions

Placebo OTHER

Saline infusion

Placebo

Hemin for injection BIOLOGICAL

Panhematin 4 mg/kg body weight reconstituted with 25% human albumin infused intravenously over at least 1 hour.

Also known as: Hematin

Hemin for injection

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female aged 18 years
• Willing to provide written informed consent

You may not qualify if:

• Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator
• Known or suspected allergy to Panhematin™ or related products
• A known or suspected allergy to human albumin
• 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
• Previous randomization in this trial

Sponsors & Collaborators

• The University of Texas Medical Branch, Galveston: lead

Study Sites (1)

University of Texas Medical Branch

Galveston, Texas, 77555, United States

Location

MeSH Terms

Conditions

Porphyria, Acute Intermittent; Coproporphyria, Hereditary; Porphyria, Variegate; Porphyrias

Interventions

Hemin; Injections

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Heme; Metalloporphyrins; Porphyrins; Tetrapyrroles; Pyrroles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 4 or More Rings; Heterocyclic Compounds, Fused-Ring; Macrocyclic Compounds; Polycyclic Compounds; Pigments, Biological; Biological Factors; Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Dr. Karl Anderson, MD
• Organization: University of Texas Medical Branch, Galveston

kanderso@utmb.edu

409-772-9092

Study Officials

• Karl E Anderson, MD

  University of Texas

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Administered doses are shielded from view. The pharmacy and one nurse who administers the drug intravenously will not be blinded.
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Two group parallel blinded study comparing active drug and placebo

Study Record Dates

• First Submitted: January 1, 2016
• First Posted: October 4, 2016
• Study Start: October 30, 2015
• Primary Completion: December 27, 2023
• Study Completion: December 27, 2023
• Last Updated: January 30, 2026
• Results First Posted: January 30, 2026

Record last verified: 2026-01

Locations

US (1)