Trial Outcomes & Findings for Panhematin for Prevention of Acute Attacks of Porphyria (NCT NCT02922413)

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

1-4 weeks

Results posted on

2026-01-30

Participant Flow

Participant milestones

Participant milestones
Measure	Hemin for Injection Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour. Hemin for injection: Panhematin 4 mg/kg body weight reconstituted with 25% human albumin infused intravenously over at least 1 hour.	Placebo A double blind dose of saline. Placebo: Saline infusion
Overall Study STARTED	7	6
Overall Study COMPLETED	7	6
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Panhematin for Prevention of Acute Attacks of Porphyria

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hemin for Injection n=7 Participants Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour. Hemin for injection: Panhematin 4 mg/kg body weight reconstituted with 25% human albumin infused intravenously over at least 1 hour.	Placebo n=6 Participants A double blind dose of saline. Placebo: Saline infusion	Total n=13 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=35 Participants	0 Participants n=4328 Participants	0 Participants n=8687 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=35 Participants	6 Participants n=4328 Participants	13 Participants n=8687 Participants
Age, Categorical >=65 years	0 Participants n=35 Participants	0 Participants n=4328 Participants	0 Participants n=8687 Participants
Sex: Female, Male Female	6 Participants n=35 Participants	6 Participants n=4328 Participants	12 Participants n=8687 Participants
Sex: Female, Male Male	1 Participants n=35 Participants	0 Participants n=4328 Participants	1 Participants n=8687 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=35 Participants	0 Participants n=4328 Participants	0 Participants n=8687 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=35 Participants	6 Participants n=4328 Participants	13 Participants n=8687 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=35 Participants	0 Participants n=4328 Participants	0 Participants n=8687 Participants
Region of Enrollment United States	7 participants n=35 Participants	6 participants n=4328 Participants	13 participants n=8687 Participants

PRIMARY outcome

Timeframe: 1-4 weeks

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

Outcome measures

Outcome measures
Measure	Hemin for Injection n=7 Participants Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour. Hemin for injection: Panhematin 4 mg/kg body weight reconstituted with 25% human albumin infused intravenously over at least 1 hour.	Placebo n=6 Participants A double blind dose of saline. Placebo: Saline infusion
Number of Participants Experiencing Acute Attacks of Porphyria After Treatment	1 Participants	5 Participants

PRIMARY outcome

Timeframe: 1-4 weeks

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.

Outcome measures

Outcome measures
Measure	Hemin for Injection n=7 Participants Double blind doses of Panhematin 4 mg/kg body weight reconstituted with 25% human albumin and infused over at least one hour. Hemin for injection: Panhematin 4 mg/kg body weight reconstituted with 25% human albumin infused intravenously over at least 1 hour.	Placebo n=6 Participants A double blind dose of saline. Placebo: Saline infusion
Number of Participants With Treatment-Related Serious Adverse Events	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 1-4 weeks

To evaluate the biochemical effects of Panhematin™ in patients treated with Panhematin™ to prevent attacks of acute porphyria by measuring urinary porphobilinogen and serum porphobilinogen. This will determine whether biochemical measurements are predictive of efficacy in preventing an attack.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1-4 weeks

To evaluate effects of age, as an example of clinical features, on response to preventive administration of Panhematin™.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1-4 weeks

To evaluate effects of the nature or the PBGD mutation on response to preventive Panhematin™

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1-4 weeks

To evaluate the use of Panhematin™ reconstituted with 25% human albumin in patients treated to prevent acute attacks of porphyria in terms of the frequency of injection site complications, which may include thrombosis or inflammation.

Outcome measures

Outcome data not reported

Adverse Events

Hemin for Injection

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Karl Anderson, MD

University of Texas Medical Branch, Galveston

Phone: 409-772-9092

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place