Trial Outcomes & Findings for A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype (NCT NCT05643495)
NCT ID: NCT05643495
Last Updated: 2025-09-08
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
From Baseline at Week 48
Results posted on
2025-09-08
Participant Flow
This study had 2 Groups: Group A participants did not have a liver biopsy and Group B participants have 2 liver biopsies performed over the course of the study.
A total of 14 participants were enrolled from 23 February 2023 to 03 November 2023 in this study. Study drug dosing and efficacy assessments were terminated early due to Sponsor decision, therefore evaluation of efficacy outcome measure was not completed.
Participant milestones
| Measure |
Group A VX-864 500 mg
Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
4
|
|
Overall Study
COMPLETED
|
9
|
2
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
Group A VX-864 500 mg
Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Withdrawal of consent (not due to AE)
|
1
|
1
|
Baseline Characteristics
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
Baseline characteristics by cohort
| Measure |
Group A VX-864 500 mg
n=10 Participants
Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
n=4 Participants
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
52.8 years
STANDARD_DEVIATION 14.2 • n=5 Participants
|
50.8 years
STANDARD_DEVIATION 10.4 • n=7 Participants
|
52.2 years
STANDARD_DEVIATION 12.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline at Week 48Population: Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 48Population: Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 48Population: Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 48Population: Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From Baseline up to Week 48Population: No participants in Group B underwent a second liver biopsy at week 24 or week 48 as the study drug dosing was terminated prior to any participant reaching week 24 or week 48. Therefore, data was not collected for this Outcome Measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 up to Week 52Population: Safety set included all participants who had received at least 1 dose of study drug in this study.
Outcome measures
| Measure |
Group A VX-864 500 mg
n=10 Participants
Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
n=4 Participants
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
|---|---|---|
|
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with TEAEs
|
10 Participants
|
4 Participants
|
|
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
1 Participants
|
0 Participants
|
Adverse Events
Group A VX-864 500 mg
Serious events: 1 serious events
Other events: 10 other events
Deaths: 0 deaths
Group B VX-864 500 mg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group A VX-864 500 mg
n=10 participants at risk
Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
n=4 participants at risk
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
|---|---|---|
|
Infections and infestations
Diverticulitis
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
Other adverse events
| Measure |
Group A VX-864 500 mg
n=10 participants at risk
Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated.
|
Group B VX-864 500 mg
n=4 participants at risk
Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48.
|
|---|---|---|
|
Congenital, familial and genetic disorders
Porphyria non-acute
|
0.00%
0/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Abdominal distension
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
2/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
50.0%
2/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Mouth ulceration
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Nausea
|
30.0%
3/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
50.0%
2/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Paraesthesia oral
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Infections and infestations
Bronchitis
|
0.00%
0/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Infections and infestations
Diverticulitis
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Investigations
Blood creatine phosphokinase increased
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Nervous system disorders
Dizziness
|
20.0%
2/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Nervous system disorders
Dysgeusia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Nervous system disorders
Headache
|
0.00%
0/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Nervous system disorders
Hyperaesthesia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Psychiatric disorders
Irritability
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Blister
|
20.0%
2/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Drug eruption
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Dyshidrotic eczema
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Photosensitivity reaction
|
30.0%
3/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Post inflammatory pigmentation change
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Pseudoporphyria
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
25.0%
1/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.0%
1/10 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
0.00%
0/4 • Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place