Condition or disease | Intervention/treatment | Phase |
---|---|---|
Wilson Disease | Drug: WTX101 Drug: SOC Therapy | Phase 3 |
This study is a randomized, rater-blinded, multi-center study assessing the efficacy and safety of an individualized WTX101 dosing regimen administered for 48 weeks, compared to SoC, in WD subjects aged 18 and older. Approximately 100 subjects will be enrolled at approximately 5 to 10 North American sites and 15 to 25 sites in the rest of the world. Subjects who complete the 48-week treatment period will be offered to participate in an Extension Phase of the study to evaluate the long-term safety and durability of treatment effect of WTX101.
The primary objective is to evaluate the efficacy of WTX101 administered for 48 weeks, compared to SOC, on Cu control in WD subjects aged 18 and older. Copper control will be assessed in terms of the percentage change from baseline (Day 1) to 48 weeks in non-ceruloplasmin-bound copper (NCC) levels. For WTX101-treated subjects, the NCC level will be corrected for the amount of Cu bound to the WTX101 tripartite complex (TPC) (NCCcorrected).
Study Type : | Interventional (Clinical Trial) |
EstimatedEnrollment : | 102 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Single (Outcomes Assessor) |
Masking Description: | This study is rater-blinded for the Unified Wilson Disease Rating Scale (UWDRS) assessment only. The rater will be blinded and will have no knowledge of the subject's treatment assignment and no access to systems that could result in potential unblinding of treatment assignment. Both raters and subjects will be instructed to avoid lines of inquiry, questions, and responses that could potentially lead to unblinding of the subject's treatment. The blinding will be documented by the rater after each UWDRS assessment. The rater assessments will be strictly limited to administration of the protocol specified instruments and assessments. |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older With an Extension Phase of up to 60 Months |
Actual Study Start Date : | February 15, 2018 |
Estimated Primary Completion Date : | December 18, 2019 |
Estimated Study Completion Date : | February 14, 2024 |
Arm | Intervention/treatment |
---|---|
Experimental: 15-60 mg WTX101 to subjects treated >28 days with SOC Subjects treated for >28 days with chelation or zinc (Zn) therapy or a combination of both chelation and Zn therapy will be treated with 15 mg every other day (QOD) to 60 mg once daily (QD) of WTX101 by oral administration. | Drug: WTX101 WTX101 administered orally in 15 mg tablets |
Active Comparator: SOC for subjects treated with SOC >28 days prior Subjects treated for >28 days with chelation or Zn therapy or a combination of both chelation and Zn therapy will continue to receive the SOC therapy. | Drug: SOC Therapy Subjects randomized to receive SOC treatment will continue their current therapy or initiate therapy with chelation, Zn, or a combination of both chelation and Zn therapy. Depending on the site/region enrolling the subject, this treatment will be trientine hydrochloride, penicillamine, or Zn, or a combination of both chelation and Zn therapy, administered according to standard regimens. |
Experimental: 15-60 mg WTX101 to subjects treated ≤28 days with SOC Subjects who are treatment naïve or have been previously treated with chelation or Zn therapy or a combination of both chelation and Zn therapy for ≤28 days will be treated with 15 mg QOD to 60 mg QD of WTX101 by oral administration. | Drug: WTX101 WTX101 administered orally in 15 mg tablets |
Active Comparator: SOC for subjects treated with SOC ≤28 days prior Subjects who are treatment naïve or have been previously treated with chelation or Zn therapy or a combination of both chelation and Zn therapy for ≤28 days will initiate or continue SOC therapy. | Drug: SOC Therapy Subjects randomized to receive SOC treatment will continue their current therapy or initiate therapy with chelation, Zn, or a combination of both chelation and Zn therapy. Depending on the site/region enrolling the subject, this treatment will be trientine hydrochloride, penicillamine, or Zn, or a combination of both chelation and Zn therapy, administered according to standard regimens. |
Evaluate the effects of WTX101 on hepatic status assessed by the Model for End-Stage Liver Disease (MELD) score. The MELD uses the subject's values for serum bilirubin, serum creatinine, and the international normalized ratio for prothrombin time (INR) to predict survival. It is calculated according to the following formula:
MELD = 3.78×ln[serum bilirubin (mg/dL)] + 11.2×ln[INR] + 9.57×ln[serum creatinine (mg/dL)] + 6.43.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Subjects who meet all of the following criteria will be eligible to participate in the study:
Females of childbearing potential will be included if they are either sexually inactive (abstinent) for 14 days prior to the first WTX101 dose and continuing through 28 days after the last WTX101 dose, or using 1 of the following highly effective birth control methods (ie, results in <1% failure rate when used consistently and correctly):
Bilateral tubal occlusion for at least 6 months prior to the first WTX101 dose;
A female of non-childbearing potential must have undergone 1 of the following sterilization procedures at least 6 months prior to the first WTX101 dose:
A non-vasectomized male subject agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study drug. For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non-vasectomized male;
Exclusion Criteria:
Subjects who meet any of the following criteria will be excluded from participation in the study:
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03403205
Contact: Alexion Pharmaceuticals Inc. | +1-855-752-2356 | clinicaltrials@alexion.com |
United States, California | |
Clinical Trial Site | Recruiting |
Los Angeles, California, United States, 90095 | |
United States, Connecticut | |
Clinical Trial Site | Recruiting |
New Haven, Connecticut, United States, 06510 | |
United States, Illinois | |
Clinical Trial Site | Recruiting |
Chicago, Illinois, United States, 60611 | |
United States, Michigan | |
Clinical Trial Site | Recruiting |
Ann Arbor, Michigan, United States, 48109 | |
United States, Tennessee | |
Clinical Trial Site | Recruiting |
Nashville, Tennessee, United States, 37240-7915 | |
United States, Texas | |
Clinical Trial Site | Recruiting |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Clinical Trial Site | Recruiting |
Seattle, Washington, United States, 98105 | |
Austria | |
Clinical Trial Site | Recruiting |
Graz, Austria, 8036 | |
Clinical Trial Site | Recruiting |
Innsbruck, Austria, 6020 | |
Clinical Trial Site | Recruiting |
Wien, Austria, 1090 | |
Germany | |
Clinical Trial Site | Recruiting |
Dresden, Germany, 01307 | |
Clinical Trial Site | Recruiting |
Hamburg, Germany, 20099 | |
Clinical Trial Site | Recruiting |
Heidelberg, Germany, 69120 | |
Clinical Trial Site | Recruiting |
Leipzig, Germany, 04103 | |
Poland | |
Clinical Trial Site | Recruiting |
Warszawa, Poland, 02-957 | |
Spain | |
Clinical Trial Site | Recruiting |
Majadahonda, Spain, 28222 | |
Clinical Trial Site | Recruiting |
Sabadell, Spain, E-08208 | |
United Kingdom | |
Clinical Trial Site | Recruiting |
Birmingham, United Kingdom, B15 2PR | |
Clinical Trial Site | Recruiting |
Cambridge, United Kingdom, CB2 0OQ | |
Clinical Trial Site | Recruiting |
Guildford, United Kingdom, GU2 7XX |
Study Director: | Eugene Swenson, MD, PhD | Alexion Pharmaceuticals |
Responsible Party: | Wilson Therapeutics AB |
ClinicalTrials.gov Identifier: | NCT03403205 History of Changes |
Other Study ID Numbers: | WTX101-301 |
First Posted: | January 18, 2018 Key Record Dates |
Last Update Posted: | December 7, 2018 |
Last Verified: | August 2018 |
Studies a U.S. FDA-regulated Drug Product: | Yes | |
Studies a U.S. FDA-regulated Device Product: | No |
Hepatolenticular Degeneration Liver Diseases Digestive System Diseases Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Brain Diseases, Metabolic, Inborn | Brain Diseases, Metabolic Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors |
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