Trial Outcomes & Findings for Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria (NCT NCT01550705)

NCT ID: NCT01550705

Last Updated: 2017-01-16

Results Overview

Plasma Protoporphyrin IX will be measured at baseline and at 3 months

Recruitment status

TERMINATED

Study phase

Target enrollment

11 participants

Primary outcome timeframe

Baseline and 3 Months

Results posted on

2017-01-16

Participant Flow

Participant milestones

Participant milestones
Measure	Isoniazid Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Isoniazid n=11 Participants Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Gender Female	6 Participants n=99 Participants
Gender Male	5 Participants n=99 Participants
Protoporphyria Type Erythropoietic Protoporphyria (EPP)	9 Participants n=99 Participants
Protoporphyria Type X-linked Erythropoietic Protoporphyria (XLEPP)	2 Participants n=99 Participants
Baseline Protoporphyrin IX level	13.3 micrograms per deciliter (µg/dL) STANDARD_DEVIATION 14.4 • n=99 Participants

PRIMARY outcome

Timeframe: Baseline and 3 Months

Plasma Protoporphyrin IX will be measured at baseline and at 3 months

Outcome measures

Outcome measures
Measure	Isoniazid n=10 Participants Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
Change in Plasma Protoporphyrin IX Level	0.36 µg/dL Standard Deviation 9.1

SECONDARY outcome

Timeframe: Baseline and 3 Months

Study participants were asked to report after 3 months if they had experienced an increase in subjective measures of sun sensitivity during the trial. Reported outcome is the number of study participants who reported increased sun sensitivity

Outcome measures

Outcome measures
Measure	Isoniazid n=10 Participants Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
Participants With Increased Sun Sensitivity	0 participants

Adverse Events

Isoniazid

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Isoniazid n=11 participants at risk Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
General disorders Flu-like Symptoms	9.1% 1/11 • Number of events 1 • 3 Months

Other adverse events

Other adverse events
Measure	Isoniazid n=11 participants at risk Subjects will receive isoniazid daily for 2 months. Subjects will be seen every 2 weeks to obtain lab samples and health check. Isoniazid: Isoniazid 5 mg/Kg up to 300 mg per day. Oral tablets. 2 months.
Gastrointestinal disorders Constipation	9.1% 1/11 • Number of events 1 • 3 Months
Respiratory, thoracic and mediastinal disorders Productive Cough	9.1% 1/11 • Number of events 1 • 3 Months
Renal and urinary disorders Urinary Tract Pain	9.1% 1/11 • Number of events 1 • 3 Months
Respiratory, thoracic and mediastinal disorders Nasal Congestion	9.1% 1/11 • Number of events 2 • 3 Months
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • 3 Months
Cardiac disorders Atrial Flutter	9.1% 1/11 • Number of events 1 • 3 Months
Reproductive system and breast disorders Dysmenorrhea	9.1% 1/11 • Number of events 1 • 3 Months
Ear and labyrinth disorders Vertigo	9.1% 1/11 • Number of events 1 • 3 Months

Additional Information

John D. Phillips, Ph.D.

University of Utah

Phone: 801-581-6650

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place