Trial Outcomes & Findings for Harvoni Treatment Porphyria Cutanea Tarda (NCT NCT03118674)

NCT ID: NCT03118674

Last Updated: 2023-07-03

Results Overview

Resolution of active PCT, defined as normalization of plasma porphyrins (less than 0.9 mcg/dL) by 7 months after start of therapy

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 23 participants
• Primary outcome timeframe: 7 months
• Results posted on: 2023-07-03

Participant Flow

Participant milestones

Measure	Harvoni 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Overall Study STARTED	23
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	15

Reasons for withdrawal

Measure	Harvoni 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Overall Study Lost to Follow-up	2
Overall Study Screen Faliure	8
Overall Study Withdrawal by Subject	5

Baseline Characteristics

Harvoni Treatment Porphyria Cutanea Tarda

Baseline characteristics by cohort

Measure	Harvoni n=15 Participants 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Age, Continuous	58.9 years STANDARD_DEVIATION 4.8 • n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	13 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	15 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	14 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: 7 months

Population: 9 observations collected at 7 months

Resolution of active PCT, defined as normalization of plasma porphyrins (less than 0.9 mcg/dL) by 7 months after start of therapy

Outcome measures

Measure	Harvoni n=9 Participants 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Number of Participants With Resolution of Active PCT by 7 Months After Start of Therapy	9 Participants

SECONDARY outcome

Timeframe: through study completion, an average of 1 year

Population: Observations for 6 participants collected at through study completion, an average of 1 year

Time to resolution of active PCT, defined as cessation of any new blisters or bullae and normalization of plasma porphyrins

Outcome measures

Measure	Harvoni n=6 Participants 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Time to Resolution of Active PCT	240 days Interval 100.0 to 615.0

SECONDARY outcome

Timeframe: 12 Months

Population: 6 Observations collected at 12 Months

Defined as a decrease of the sum of urinary uro- and hepta-carboxyl porphyrins to less than 100 mcg/g creatinine and a normal urine porphyrin HPLC pattern defined as the total of highly carboxylated porphyrins (uro- and heptacarboxyl-porphyrins) being less than that of coproporphyrins, and the absence of a plasma fluorescence peak by fluorescence scanning

Outcome measures

Measure	Harvoni n=6 Participants 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Number of Participants With Complete Biochemical Remission of PCT	5 Participants

SECONDARY outcome

Timeframe: Up to 15 months

Population: 2 participants lost of follow up, 1 participant left the study after 4 weeks of dosing

Defined as no detectable HCV RNA at end of treatment and persisting for at least 12 weeks after end of treatment.

Outcome measures

Measure	Harvoni n=12 Participants 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Number of Participants With Cure of CHC	11 Participants

Adverse Events

Harvoni

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Harvoni n=15 participants at risk 1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis Harvoni: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Vascular disorders Hypertension	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Psychiatric disorders Anxiety	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Musculoskeletal and connective tissue disorders Back pain	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
General disorders Sinus pressure	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Infections and infestations Infection	6.7% 1/15 • Number of events 2 • Baseline through study completion, an average of 1 year
Musculoskeletal and connective tissue disorders Muscle weakness left-sided	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
General disorders Nasal congestion	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Musculoskeletal and connective tissue disorders Neck pain	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Musculoskeletal and connective tissue disorders pain in both hands	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Infections and infestations Skin infection	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Psychiatric disorders Suicidal ideation	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year
Gastrointestinal disorders Vomiting	6.7% 1/15 • Number of events 1 • Baseline through study completion, an average of 1 year

Additional Information

Herbert L. Bonkovsky, MD

Wake Forest School of Medicine

Phone: 3367137341

Email: hbonkovs@wakehealth.edu

Results disclosure agreements

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