Trial Outcomes & Findings for A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (NCT NCT04537377)
NCT ID: NCT04537377
Last Updated: 2026-01-30
Results Overview
AEs will be summarized based on the date of onset for the event. Number of treatment-emergent AEs will be provided by SOC and PT, by dose cohort and overall.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE1/PHASE2
Target enrollment
4 participants
Primary outcome timeframe
through primary completion visit, an average of 1 year
Results posted on
2026-01-30
Participant Flow
The study was prematurely terminated by the Sponsor for futility reasons, after insufficient pharmacodynamic response in cohorts 1 and 2. No further patient enrollment occurred after this date. The 4 treated patients entered the long-term follow-up (LTFU) up to 5 years post injection, thus 2 sites remain active. An abbreviated CSR was issued including all data until early termination (an average of 1 year). The LTFU results will be provided in a separate addendum at the end of 2029.
Participant milestones
| Measure |
VTX-801 5E12 VG/kg
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
End of Primary Completion Period
|
2
|
2
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
VTX-801 5E12 VG/kg
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Overall Study
Long term follow-up
|
2
|
2
|
Baseline Characteristics
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Baseline characteristics by cohort
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
36.5 years
STANDARD_DEVIATION 17.68 • n=41 Participants
|
32.0 years
STANDARD_DEVIATION 18.38 • n=1581 Participants
|
34.3 years
STANDARD_DEVIATION 14.95 • n=4626 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
2 Participants
n=4626 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
2 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
1 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
3 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
3 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
1 Participants
n=1581 Participants
|
1 Participants
n=4626 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=41 Participants
|
2 participants
n=1581 Participants
|
4 participants
n=4626 Participants
|
|
Smoking History
Yes
|
0 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
0 Participants
n=4626 Participants
|
|
Smoking History
No
|
2 Participants
n=41 Participants
|
2 Participants
n=1581 Participants
|
4 Participants
n=4626 Participants
|
|
Alcohol Consumption
Yes current
|
1 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
1 Participants
n=4626 Participants
|
|
Alcohol Consumption
Yes former
|
1 Participants
n=41 Participants
|
0 Participants
n=1581 Participants
|
1 Participants
n=4626 Participants
|
|
Alcohol Consumption
No
|
0 Participants
n=41 Participants
|
2 Participants
n=1581 Participants
|
2 Participants
n=4626 Participants
|
|
BMI
|
25.4 kg/m^2
STANDARD_DEVIATION 2.0 • n=41 Participants
|
29.7 kg/m^2
STANDARD_DEVIATION 4.64 • n=1581 Participants
|
27.6 kg/m^2
STANDARD_DEVIATION 3.83 • n=4626 Participants
|
PRIMARY outcome
Timeframe: through primary completion visit, an average of 1 yearPopulation: Safety Analysis Set
AEs will be summarized based on the date of onset for the event. Number of treatment-emergent AEs will be provided by SOC and PT, by dose cohort and overall.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
Treatment-emergent AEs
|
2 participants
|
2 participants
|
|
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
Deaths
|
0 participants
|
0 participants
|
|
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
SAEs
|
0 participants
|
1 participants
|
|
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
AEs leading to discontinuation
|
0 participants
|
0 participants
|
|
Safety and Tolerability Profile (Including Treatment-emergent Adverse Events (TEAE)) - Number of Participants
TEAE meeting any stopping rule
|
2 participants
|
2 participants
|
SECONDARY outcome
Timeframe: through primary completion visit, an average of 1 yearPopulation: Safety Analysis Set
Free serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Free Serum Cu
Absolut value
|
1.080 umol/L
Standard Deviation 0.6930
|
0.620 umol/L
Standard Deviation 0.1556
|
|
Free Serum Cu
CFB
|
0.115 umol/L
Standard Deviation 0.3465
|
-0.225 umol/L
Standard Deviation 0.3182
|
SECONDARY outcome
Timeframe: through primary completion visit, an average of 1 yearPopulation: Safety Analysis Set
Total serum Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Total Serum Cu
CFB
|
0.700 umol/L
Standard Deviation 0.5091
|
1.185 umol/L
Standard Deviation 1.4354
|
|
Total Serum Cu
Absolut value
|
2.595 umol/L
Standard Deviation 0.3889
|
5.185 umol/L
Standard Deviation 5.4094
|
SECONDARY outcome
Timeframe: through primary completion visit, an average of 1 yearPopulation: Safety Analysis Set
24-hour urinary Cu will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
24-hour Urinary Cu
Absolut value
|
78.0 mcg/24hr
Standard Deviation 5.66
|
85.5 mcg/24hr
Standard Deviation 61.52
|
|
24-hour Urinary Cu
CFB
|
-7.0 mcg/24hr
Standard Deviation 1.41
|
7.0 mcg/24hr
Standard Deviation 22.63
|
SECONDARY outcome
Timeframe: through primary completion visit, an average of 1 yearPopulation: Safety Analysis Set
Serum ceruloplasmin will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values and changes from baseline.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Serum Ceruloplasmin Activity (Enzymatic Assay)
Absolut value
|
10.75 nmol Fe2+/min/mL
Standard Deviation 0.495
|
42.80 nmol Fe2+/min/mL
Standard Deviation 46.810
|
|
Serum Ceruloplasmin Activity (Enzymatic Assay)
CFB
|
1.05 nmol Fe2+/min/mL
Standard Deviation 1.344
|
-27.40 nmol Fe2+/min/mL
Standard Deviation 39.174
|
SECONDARY outcome
Timeframe: At Week 12 and Week 36Population: Safety Analysis Set
The number of Responders and Insufficient-Responders will be summarized by dose cohort and planned visit, with response to treatment. Responder status was assessed using radiocopper blood PK results and other cold copper parameters if needed.
Outcome measures
| Measure |
VTX-801 5E12 VG/kg
n=2 Participants
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 Participants
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
VTX-801 Responder Status
W12 · Responder
|
0 Participants
|
0 Participants
|
|
VTX-801 Responder Status
W12 · Insufficient Responder
|
2 Participants
|
2 Participants
|
|
VTX-801 Responder Status
W12 · Not assessed
|
0 Participants
|
0 Participants
|
|
VTX-801 Responder Status
W36 · Responder
|
0 Participants
|
0 Participants
|
|
VTX-801 Responder Status
W36 · Insufficient Responder
|
2 Participants
|
0 Participants
|
|
VTX-801 Responder Status
W36 · Not assessed
|
0 Participants
|
2 Participants
|
Adverse Events
VTX-801 5E12 VG/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
VTX-801 1.5E13 VG/kg
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
VTX-801 5E12 VG/kg
n=2 participants at risk
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 participants at risk
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Hepatobiliary disorders
Drug-Induced Liver Injury
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
Other adverse events
| Measure |
VTX-801 5E12 VG/kg
n=2 participants at risk
Cohort 1: Patients received a single IV infusion of VTX-801 at 5E12 VG/kg and with a 5-year follow-up.
|
VTX-801 1.5E13 VG/kg
n=2 participants at risk
Cohort 2: Patients received a single IV infusion of VTX-801 at 1.5E13 VG/kg and with a 5-year follow-up.
|
|---|---|---|
|
Infections and infestations
Gastroenteritis Viral
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Injury, poisoning and procedural complications
Infusion Related Reaction
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Injury, poisoning and procedural complications
Limb Injury
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Injury, poisoning and procedural complications
Meniscus Injury
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Nervous system disorders
Tremor
|
100.0%
2/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Nervous system disorders
Brain Fog
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Eye disorders
Kayser-Fleischer Ring
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Hepatobiliary disorders
Drug-Induced Liver Injury
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Immune system disorders
Seasonal Allergy
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Psychiatric disorders
Insomnia
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Infections and infestations
Covid-19
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
100.0%
2/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Rectal Haemorrhage
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
General disorders
Fatigue
|
100.0%
2/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
100.0%
2/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
|
General disorders
Non-Cardiac Chest Pain
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Alanine Aminotransferase Increased
|
100.0%
2/2 • Number of events 7 • From Baseline through primary completion visit (an average of 1 year)
|
100.0%
2/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Lymphocyte Count Decreased
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Blood Creatine Phosphokinase Increased
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 3 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Body Temperature Increased
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Drug Level Increased
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Neutrophil Count Decreased
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
|
Investigations
Urine Copper Increased
|
0.00%
0/2 • From Baseline through primary completion visit (an average of 1 year)
|
50.0%
1/2 • Number of events 1 • From Baseline through primary completion visit (an average of 1 year)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor can request deletions to Confidential Information to the extent such deletion does not preclude the complete and accurate presentation and interpretation of the Study results.
- Publication restrictions are in place
Restriction type: OTHER