One-a-day trientine for newly diagnosed Wilson's disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 trial compares a once-daily trientine tetrahydrochloride (TETA 4HCl) to d-Penicillamine (DPA) as initial therapy for Wilson's disease. Eligible participants are treatment-naïve or chelator-naïve patients aged 8 years and above (weight ≥25 kg), diagnosed by Leipzig score ≥4. The study includes up to 28 days of screening followed by 48 weeks of post-randomization treatment with up to 12 visits for safety labs and clinical assessments, plus optional questionnaires on treatment satisfaction and well-being. The trial aims to determine if the new once-daily formulation is safe and effective as a first-line option for managing copper overload in WD. Lead sponsor is Orphalan, with sites at three U.S. academic medical centers.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Participant is aged 8 years or older and is willing and able to give informed consent for participation in the study, or by a parent/legally authorized representative (LAR) and assent obtained (in accordance with local regulations) for any participant less than the age of majority (e.g. less than 18 years of age, depending on local requirements).
2. Participant has a body weight of at least 25 kg at screening.
3. Participant has a diagnosis of WD, as defined by a Leipzig score of greater than or equal to 4. Note that historical test results can be used for the diagnosis.
4. Participant has either:

   1. Received no prior prescribed therapy \[a\] for the treatment of WD (treatment-naïve), or
   2. Received no prescribed chelator therapy \[a\] for the treatment of WD (chelator-naïve); zinc salts are permitted for no more than 28 days prior to the start of screening assessments, and these participants must be symptomatic.

   \[a\] prescribed therapy for WD refers to the authorized chelator treatments of trientine (TETA 2HCl or TETA 4HCl) and DPA, or zinc salts.
5. Able and willing to comply with study procedures and requirements, as described in the informed consent.
6. Adequate venous access to allow collection of required blood samples.
7. Willing to comply with low copper diet for the duration of the study.
8. Participant requires treatment for WD, in the opinion of the Investigator.
9. Participant is able to take the study medication as prescribed, in the opinion of the Investigator.

Exclusion Criteria:

1. Any known contraindications for treatment with DPA.
2. Any known contraindications for treatment with TETA 4HCl.
3. Unable to swallow tablets/capsules independently or considered high risk for aspiration, in the opinion of the Investigator
4. Acute liver failure (ALF) or at high risk of ALF, in the opinion of the Investigator.
5. Decompensated hepatic cirrhosis, in the opinion of the Investigator.
6. Participants 12 years or older at screening, Model for End stage Liver Disease (MELD) score of greater than or equal to 12.
7. Participants 8 to 11 years at screening, Model for Pediatric End stage Liver Disease (PELD) of greater than or equal to 10
8. Hemoglobin of less than or equal to 9 g/dL.
9. Estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73m²
10. Nephritis or nephrotic syndrome, in the opinion of the Investigator.
11. Alanine aminotransferase greater than 5 times upper limit of normal (ULN).
12. Severe pulmonary disease requiring home nebulization and/or home oxygen therapy.
13. Clinically significant gastrointestinal bleed within past 6-months.
14. Neurological disease requiring either nasogastric feeding or intensive inpatient medical care.
15. Active or history of seizures requiring anti-epileptics within 6 months prior to informed consent.
16. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV).
17. Major systemic disease or other illness that would, in the opinion of the Investigator, compromise patient safety or interfere with the collection or interpretation of the study results.
18. Female participants of childbearing potential, currently pregnant, currently nursing, or planning a pregnancy during study period.
19. Female participants of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study.
20. Male participants, unable or unwilling to use a reliable form of contraceptive throughout the study.
21. Participant is not willing to comply with the prohibited medication requirements for the study.
22. In the opinion of the Investigator, the participant is likely to be non-compliant or uncooperative for the required study visits or study assessments, or has any disease, disability, illness or abnormal laboratory values that could compromise patient safety or interfere with the collection or interpretation of study results.

Where it's running

• University of Colorado Anschutz School of Medicine
  Denver, Colorado, United States Recruiting
• University of Michigan Medical Centre
  Ann Arbor, Michigan, United States Recruiting
• Beijing YouAn Hospital, Capital Medical University
  Beijing, Beijing Municipality, China Recruiting
• Beijing Friendship Hospital,Capital Medical University
  Beijing, Beijing Municipality, China Recruiting
• The Second Affiliated Hospital Zhejiang University School of Medicine
  Hangzhou, Zhengjiang Province, China Recruiting
• Yale University School of Medicine
  New Haven, Connecticut, United States Not yet recruiting

Study contacts

• Study coordinator: Carla Bennett, Bsc. Hons
• Email: clinicaloperations@orphalan.com
• Phone: +44 (0) 7918380893

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• PM577a: a one-time gene-editing therapy for Wilson disease

  This study tests PM577a, a gene-editing treatment, to see if it is safe and may help people with Wilson disease who have a p.H1069Q ATP7B mutation.

  Recruiting at 2 sites Phase 1/2 Prime Medicine, Inc. · New Haven, Connecticut and 3 other locations · NCT07748403

• ATP7B mRNA (DSL101) therapy for Wilson disease

  This treatment will test whether one or more IV infusions of ATP7B mRNA (DSL101) are safe and help control copper in adults with genetically confirmed Wilson disease.

  Recruiting Early Phase 1 DSciLab Co., Ltd. · Hefei, Anhui · NCT07240896

• Investigating heart issues in Wilson's Disease

  This study is testing how Wilson's Disease affects the heart in adults by using long-term heart monitoring to see if copper buildup causes any problems.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT05493605

• International registry for Wilson's Disease patients

  This study is gathering information from people with Wilson's Disease during their regular doctor visits to better understand the disease and how patients are managing it.

  Recruiting at 16 sites Orphalan · Leuven and 15 other locations · NCT05239858

• Examining copper levels in breast milk of pregnant women with Wilson's disease

  This study tests how treatment for Wilson's disease affects copper levels in the breast milk of pregnant women to ensure the health of both mom and baby during breastfeeding.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT05183165

• Observational study on Wilson's Disease

  This study looks at how Wilson's Disease affects people in different ways to help improve care for patients and their families.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou · NCT04012658

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.