Trial Outcomes & Findings for Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda (NCT NCT01573754)

NCT ID: NCT01573754

Last Updated: 2023-02-17

Results Overview

Time to a decrease in plasma porphyrin concentration to less than 0.9 mcg/dL

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 48 participants
• Primary outcome timeframe: To end of study, an average of 3 years
• Results posted on: 2023-02-17

Participant Flow

Medical clinics and clinical research center

Diagnosis of porphyria cutanea tarda established before enrollment

Participant milestones

Measure	Hydroxychloroquine Low-dose hydroxychloroquine 100 mg by mouth twice weekly Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy Phlebotomy 450 mL biweekly Phlebotomy: 450 mL every 2 weeks
Overall Study STARTED	16	32
Overall Study COMPLETED	13	17
Overall Study NOT COMPLETED	3	15

Reasons for withdrawal

Measure	Hydroxychloroquine Low-dose hydroxychloroquine 100 mg by mouth twice weekly Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy Phlebotomy 450 mL biweekly Phlebotomy: 450 mL every 2 weeks
Overall Study Noncompliance or remission not achieved	3	15

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Hydroxychloroquine n=13 Participants Low-dose hydroxychloroquine 100 mg by mouth twice weekly Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=17 Participants Phlebotomy 450 mL biweekly Phlebotomy: 450 mL every 2 weeks	Total n=30 Participants Total of all reporting groups
Age, Continuous	50.3 years STANDARD_DEVIATION 6.0 • n=13 Participants	51.3 years STANDARD_DEVIATION 5.0 • n=17 Participants	50.9 years STANDARD_DEVIATION 5.4 • n=30 Participants
Sex: Female, Male Female	8 Participants n=13 Participants	2 Participants n=17 Participants	10 Participants n=30 Participants
Sex: Female, Male Male	5 Participants n=13 Participants	15 Participants n=17 Participants	20 Participants n=30 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: To end of study, an average of 3 years

Time to a decrease in plasma porphyrin concentration to less than 0.9 mcg/dL

Outcome measures

Measure	Hydroxychloroquine n=13 Participants Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=17 Participants Phlebotomy: 450 mL every 2 weeks
Remission	6.1 months Interval 0.9 to 10.7	6.9 months Interval 1.8 to 26.5

SECONDARY outcome

Timeframe: To end of study, an average of 3 years

Population: Participants missed some study visits.

50% reduction in plasma porphyrin level during treatment

Outcome measures

Measure	Hydroxychloroquine n=8 Participants Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=16 Participants Phlebotomy: 450 mL every 2 weeks
50% Reduction in Plasma Porphyrin Level	44 days Interval 13.0 to 77.0	65 days Interval 42.0 to 123.0

SECONDARY outcome

Timeframe: To end of study, an average of 3 years

Population: Participants missed some study visits.

Time to 75% reduction in plasma porphyrin level during treatment

Outcome measures

Measure	Hydroxychloroquine n=8 Participants Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=13 Participants Phlebotomy: 450 mL every 2 weeks
75% Reduction in Plasma Porphyrin Level	73 days Interval 72.0 to 123.0	44 days Interval 40.0 to 238.0

SECONDARY outcome

Timeframe: To end of study, an average of 3 years

Number of days to normal urinary porphyrin levels for participants treated for Porphyria Cutanea Tarda (PCT). Days are summed together for all participants for a single value in each Arm.

Outcome measures

Measure	Hydroxychloroquine n=6 Participants Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=12 Participants Phlebotomy: 450 mL every 2 weeks
Number of Days With Normal Urinary Porphyrin Levels	175 days	265 days

Adverse Events

Hydroxychloroquine

Serious events: 4 serious events

Other events: 3 other events

Deaths: 1 deaths

Phlebotomy

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Hydroxychloroquine n=13 participants at risk Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=17 participants at risk Phlebotomy: 450 mL every 2 weeks
General disorders Death	7.7% 1/13 • Number of events 1 • Up to 27 months	0.00% 0/17 • Up to 27 months
Cardiac disorders Coronary disease	15.4% 2/13 • Number of events 2 • Up to 27 months	0.00% 0/17 • Up to 27 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Prostate cancer	7.7% 1/13 • Number of events 1 • Up to 27 months	0.00% 0/17 • Up to 27 months

Other adverse events

Measure	Hydroxychloroquine n=13 participants at risk Hydroxychloroquine: 100 mg by mouth twice weekly	Phlebotomy n=17 participants at risk Phlebotomy: 450 mL every 2 weeks
Psychiatric disorders Suicidal thoughts	7.7% 1/13 • Number of events 1 • Up to 27 months	0.00% 0/17 • Up to 27 months
General disorders Chest pain	7.7% 1/13 • Number of events 1 • Up to 27 months	0.00% 0/17 • Up to 27 months
Skin and subcutaneous tissue disorders Pruritis	7.7% 1/13 • Number of events 1 • Up to 27 months	0.00% 0/17 • Up to 27 months

Additional Information

Dr. Karl Anderson

University of Texas Medical Branch Galveston

Phone: 409-772-4661

Email: kanderso@utmb.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place