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UX701

Phase 1

Wilson Disease | Gene therapy | Rare Disease |Ultragenyx Pharmaceutical Inc.|Last Updated: Mar 13, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment82
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04884815A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseasePHASE1 ACTIVE NOT_RECRUITING 82 — — Sep 27, 2021Mar 1, 2034Mar 13, 202616 United States, Canada +3
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Study Endpoints
Primary Endpoints
Stage 1: Incidence of Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Adverse Events of Special Interest (AESIs), Treatment-Related TEAEs, and Treatment-Related TESAEs
Up to Week 52
Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Total Copper from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Ceruloplasmin-bound Copper from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Ceruloplasmin from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Free Copper from Baseline at Week 52
Baseline, Week 52
Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52
Baseline, Week 52
Stage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52
Week 52
Stage 1: Number of Participants Who Discontinue SOC Medication by Week 52
Week 52
Stage 1: Number of Consecutive Weeks off SOC Medication at Week 52
Week 52
Stage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, Evaluated for Superiority
Baseline, Week 52
Stage 2: Percent Reduction in SOC Medication by Week 52, Evaluated for Superiority
Week 52
Secondary Endpoints
Stage 2: Change in Ceruloplasmin Activity Levels from Baseline at Week 52, Evaluated for Superiority
Baseline, Week 52
Stage 2: Number of Participants who Discontinue SOC Medication by Week 52
Week 52
Stage 2: Change in FACIT-Fatigue Scale Score from Baseline at Week 52
Baseline, Week 52
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Stage 1: UX701 Dose Level 1EXPERIMENTALParticipants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1.
Stage 1: UX701 Dose Level 2EXPERIMENTALParticipants receive a single, peripheral IV infusion of UX701 at dose level 2.
Stage 1: UX701 Dose Level 3EXPERIMENTALParticipants receive a single, peripheral IV infusion of UX701 at dose level 3.
Stage 1: UX701 Dose Level 4EXPERIMENTALParticipants receive a single, peripheral IV infusion of UX701 at dose level 4.
Stage 2: UX701 at Selected DoseEXPERIMENTALParticipants randomized to UX701 receive a single, peripheral IV infusion of UX701 at the selected dose.
Stage 2: Standard of Care (SOC) to UX701EXPERIMENTALParticipants randomized to SOC will continue their baseline SOC medications for 52 weeks, followed by a single, peripheral IV infusion of UX701 at the selected dose. Following UX701 administration, participants will be evaluated for modification of their SOC medications.
Interventions
NameTypeDescription
UX701GENETICNonreplicating, recombinant gene transfer vector
Standard of Care (SOC)DRUGSOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites16

Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening...

Countries:United StatesCanadaPortugalSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04884815primaryCompletionDate: changed
LOWMay 24, 2026NCT04884815studyFirstPostDate: changed