Trial Outcomes & Findings for Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria (NCT NCT02180412)

Results Overview

The difference in the pre-infusion NRS pain scores and NRS pain score 12 hours from the infusion start time. To define this variable, all blinded study treatment infusion times were compared with the pain score survey times. The pain score closest to but prior to the infusion time was the pre-infusion pain score. Numeric rating scale for pain (0-10; 0=no pain, 10=most severe pain).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

Baseline and 12 hours

Results posted on

2025-04-11

Participant Flow

Participant milestones

Participant milestones
Measure	Panhematin Panhematin plus glucose Panhematin: Glucose loading Glucose: Glucose is administered to both groups as routine care.	Placebo Placebo (saline) plus glucose Glucose: Glucose is administered to both groups as routine care.
Overall Study STARTED	10	10
Overall Study COMPLETED	10	10
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Panhematin n=10 Participants Panhematin plus glucose Panhematin: Glucose loading Glucose: Glucose is administered to both groups as routine care.	Placebo n=10 Participants Placebo (saline) plus glucose Glucose: Glucose is administered to both groups as routine care.	Total n=20 Participants Total of all reporting groups
Age, Continuous	36.6 years STANDARD_DEVIATION 6.2 • n=5 Participants	36.7 years STANDARD_DEVIATION 11.2 • n=7 Participants	36.7 years STANDARD_DEVIATION 8.8 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	10 Participants n=7 Participants	20 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Non-Hispanic American Indian or Alaskan native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Non-Hispanic Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Non-Hispanic Black or African American	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Non-Hispanic White	9 Participants n=5 Participants	6 Participants n=7 Participants	15 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 12 hours

Population: One participant was randomized incorrectly and didn't meet inclusion criteria. Therefore, this analysis was done without one participant, making this analysis done under modified intent-to-treat protocol, not true intent-to-treat protocol.

The difference in the pre-infusion NRS pain scores and NRS pain score 12 hours from the infusion start time. To define this variable, all blinded study treatment infusion times were compared with the pain score survey times. The pain score closest to but prior to the infusion time was the pre-infusion pain score. Numeric rating scale for pain (0-10; 0=no pain, 10=most severe pain).

Outcome measures

Outcome measures
Measure	Panhematin n=9 Participants Panhematin plus glucose Panhematin: Glucose loading Glucose: Glucose is administered to both groups as routine care.	Placebo n=10 Participants Placebo (saline) plus glucose Glucose: Glucose is administered to both groups as routine care.
Difference in NRS Pain Score Between Baseline and 12 Hours	-0.2 score on a scale Standard Deviation 2.2	-0.2 score on a scale Standard Deviation 1.9

SECONDARY outcome

Timeframe: Day 1 (baseline), Day 2, Day 3 and Day 4

Population: One participant was randomized incorrectly and didn't meet inclusion criteria. Therefore, this analysis was done without one participant, making this analysis done under modified intent-to-treat protocol, not true intent-to-treat protocol.

Difference in the panhematin and placebo arm for the change in urinary ALA, (PBG), and total porphyrins between baseline and subsequent time points.

Outcome measures

Outcome measures
Measure	Panhematin n=9 Participants Panhematin plus glucose Panhematin: Glucose loading Glucose: Glucose is administered to both groups as routine care.	Placebo n=10 Participants Placebo (saline) plus glucose Glucose: Glucose is administered to both groups as routine care.
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria ALA Day 1 vs Day 2	8.47 mean difference of mg/g creatinine Standard Deviation 8.97	2.31 mean difference of mg/g creatinine Standard Deviation 5.5
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria ALA Day 1 vs Day 3	13.27 mean difference of mg/g creatinine Standard Deviation 11.45	-0.57 mean difference of mg/g creatinine Standard Deviation 6.01
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria ALA Day 1 vs Day 4	14.90 mean difference of mg/g creatinine Standard Deviation 11.85	1.99 mean difference of mg/g creatinine Standard Deviation 10.73
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria PBG Day 1 vs Day 3	15.56 mean difference of mg/g creatinine Standard Deviation 17.88	-7.08 mean difference of mg/g creatinine Standard Deviation 10.76
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria PBG Day 1 vs Day 4	20.16 mean difference of mg/g creatinine Standard Deviation 18.63	-5.83 mean difference of mg/g creatinine Standard Deviation 20.00
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria Total Porphyrins Day 1 vs Day 2	297.60 mean difference of mg/g creatinine Standard Deviation 948.90	317.00 mean difference of mg/g creatinine Standard Deviation 682.93
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria Total Porphyrins Day 1 vs Day 3	594.00 mean difference of mg/g creatinine Standard Deviation 1173.48	165.00 mean difference of mg/g creatinine Standard Deviation 678.41
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria Total Porphyrins Day 1 vs Day 4	606.11 mean difference of mg/g creatinine Standard Deviation 1238.87	331.33 mean difference of mg/g creatinine Standard Deviation 836.59
Biochemical Effects of Panhematin In Participants Treated Early For Attacks of Porphyria PBG Day 1 vs Day 2	6.56 mean difference of mg/g creatinine Standard Deviation 11.69	-2.94 mean difference of mg/g creatinine Standard Deviation 12.85

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 days

Age, sex, exacerbating factors

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 days

Types of mutations

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 days

Frequency of side effects or adverse events

Outcome measures

Outcome data not reported

Adverse Events

Panhematin

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