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Gene Therapy
UX701 Gene Therapy for Wilson Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by Ultragenyx Pharmaceutical Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Confirmed diagnosis of Wilson disease
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 312
Awards & highlights
Study Summary
This trial is testing a new drug, UX701, to see if it is safe for people with Wilson Disease and if it helps regulate copper levels.
Who is the study for?
This trial is for individuals with confirmed Wilson disease who have been avoiding high copper foods and taking copper chelators or zinc therapy consistently for at least 6 months. They should be able to follow the study's procedures, including blood and urine tests. People with significant liver inflammation, pre-existing antibodies to AAV9 capsid, severe neurological issues, a MELD score over 13, or history of liver transplant cannot participate.Check my eligibility
What is being tested?
The study is testing UX701 gene transfer against a placebo in patients with Wilson disease. It aims to assess the safety of single IV doses of UX701 and its effectiveness on regulating copper levels in the body. The best dose will be chosen based on safety and efficacy data collected during the trial.See study design
What are the potential side effects?
Potential side effects may include reactions related to immune response against the AAV9 vector used in gene transfer such as mild flu-like symptoms, muscle pain or discomfort at injection site. Long-term side effects are unknown due to the novelty of this treatment.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with Wilson disease.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to week 312
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to week 312
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52
Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52
Stage 1: Change in Ceruloplasmin from Baseline at Week 52
+9 moreSecondary outcome measures
Stage 2: Change in Ceruloplasmin Activity Levels from Baseline at Week 52, Evaluated for Superiority
Stage 2: Change in FACIT-Fatigue Scale Score from Baseline at Week 52
Stage 2: Change in Liver Copper Concentration Assessed by Liver Biopsy from Baseline at Week 52
+1 moreOther outcome measures
Stage 2: Development of Anti-ATP7B Antibodies
Therapeutic procedure
Trial Design
5Treatment groups
Experimental Treatment
Group I: Stage 3: Placebo or UX701Experimental Treatment2 Interventions
Participants randomized in Stage 2 to UX701 will receive a single, peripheral IV infusion of normal saline (placebo). Participants randomized in Stage 2 to placebo will be eligible for a single, peripheral IV infusion of UX701 at the selected dose.
Group II: Stage 2: UX701 or PlaceboExperimental Treatment2 Interventions
Participants randomized 2:1 to receive UX701 or Placebo. Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at a dose selected in Stage 1. Participants randomized to placebo will receive a single, peripheral IV infusion of normal saline (placebo).
Group III: Stage 1: UX701 Dose Level 3Experimental Treatment1 Intervention
Participants receive a single, peripheral IV infusion of UX701 at dose level 3.
Group IV: Stage 1: UX701 Dose Level 2Experimental Treatment1 Intervention
Participants receive a single, peripheral IV infusion of UX701 at dose level 2.
Group V: Stage 1: UX701 Dose Level 1Experimental Treatment1 Intervention
Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1.
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Who is running the clinical trial?
Ultragenyx Pharmaceutical IncLead Sponsor
89 Previous Clinical Trials
178,996 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,777 Previous Clinical Trials
8,063,402 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your MELD score is higher than 13.I have been on a stable Wilson disease treatment for at least 6 months.I am willing and able to follow all study requirements, including frequent tests and long-term follow-up.I have been diagnosed with Wilson disease.I have had a liver transplant.I have advanced liver disease with symptoms like swelling, enlarged spleen, or confusion.My kidney function is reduced, with a filtration rate below 60 mL/min.I have liver inflammation shown by lab tests.I have moderate to severe depression or recent thoughts of harming myself.You are allergic to certain types of local anesthetics.Your hemoglobin level is lower than 9 grams per deciliter.You must avoid eating foods that contain a lot of copper for at least 6 months before the study and during the study.You have antibodies that can be found in your body before starting the study.
Research Study Groups:
This trial has the following groups:- Group 1: Stage 1: UX701 Dose Level 2
- Group 2: Stage 1: UX701 Dose Level 3
- Group 3: Stage 2: UX701 or Placebo
- Group 4: Stage 1: UX701 Dose Level 1
- Group 5: Stage 3: Placebo or UX701
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What are the prominent conditions that UX701 is utilized to address?
"UX701 is often used to manage allergic contact dermatitis, but its efficacy also extends to treating nephrotic syndrome, vasculitis, and synovitis."
Answered by AI
What is the current scope of this trial's presence in clinics?
"This medical experiment is being conducted at Indiana University in Indianapolis, the University of Miami in Florida, and UCLA in California. Additionally, there are 10 other sites participating as well."
Answered by AI
How many participants are in this clinical trial?
"Affirmative. Clinicaltrials.gov's data reveals that this study, first posted on September 27th 2021 is currently recruiting participants. Approximately 78 volunteers must be registered from 10 clinical sites."
Answered by AI
Has there been previous research examining UX701?
"UX701 was initially investigated by Karolinska Institutet in 2002, with a total of 18518 trials completed to date. There are 87 clinical experiments presently taking place, predominantly centered around Indianapolis, Indiana."
Answered by AI
Are there any vacancies within this clinical investigation?
"As confirmed on clinicaltrials.gov, this trial is currently seeking participants and has been since it was published on September 27th 2021 with the most recent update being issued November 2nd 2022."
Answered by AI
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