Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

NCT07465718 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ORPH-131-017
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 3

Brief Summary

The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.

Conditions

Wilson's Disease

Keywords

trientine; randomized controlled trial; d-Penicillamine; Phase 3

Outcome Measures

Primary Outcomes (1)

• Absolute value of serum NCC at Week 48 assessed using the NCC-speciation assay (serum NCC-Sp)

  Week 48

Secondary Outcomes (5)

• Serum NCC-Sp

  Week 48

• 24-hour UCE

  Week 48

• Investigator's assessment of signs and symptoms

  Week 48

• Clinical Global Impression of Change

  Week 48

• Clinical stability

  Week 48

Study Arms (2)

TETA 4HCl formulation

EXPERIMENTAL

Participants are planned to receive TETA 4HCl for the 48-week post-randomization period.

Drug: TETA 4HCl formulation

Standard of care d-Penicillamine (DPA)

ACTIVE COMPARATOR

Participants are planned to receive DPA for the 48-week post-randomization period.

Drug: D-Penicillamine

Interventions

TETA 4HCl formulation DRUG

The new formulation of TETA 4HCl will be administered once a day. Each film-coated tablet is scored to enable halving, if required. Randomized participants are planned to receive TETA 4HCl for the 48-week post-randomization period.

TETA 4HCl formulation

D-Penicillamine DRUG

Standard of care DPA is to be used, per the sites and treating physician's usual practice. To be administered in accordance with the product labelling and/or the institutions treatment practice guidelines. Randomised participants are planned to receive DPA for the 48-week post-randomization period.

Standard of care d-Penicillamine (DPA)

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• Participant has a body weight of at least 25 kg at screening.
• 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.
• Participant has either:
• Received no prior prescribed therapy \[a\] for the treatment of WD (treatment-naïve), or
• 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.
• Able and willing to comply with study procedures and requirements, as described in the informed consent.
• Adequate venous access to allow collection of required blood samples.
• Willing to comply with low copper diet for the duration of the study.
• Participant requires treatment for WD, in the opinion of the Investigator.
• Participant is able to take the study medication as prescribed, in the opinion of the Investigator.

You may not qualify if:

• Any known contraindications for treatment with DPA.
• Any known contraindications for treatment with TETA 4HCl.
• Unable to swallow tablets/capsules independently or considered high risk for aspiration, in the opinion of the Investigator
• Acute liver failure (ALF) or at high risk of ALF, in the opinion of the Investigator.
• Decompensated hepatic cirrhosis, in the opinion of the Investigator.
• Participants 12 years or older at screening, Model for End stage Liver Disease (MELD) score of greater than or equal to 12.
• Participants 8 to 11 years at screening, Model for Pediatric End stage Liver Disease (PELD) of greater than or equal to 10
• Hemoglobin of less than or equal to 9 g/dL.
• Estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73m²
• Nephritis or nephrotic syndrome, in the opinion of the Investigator.
• Alanine aminotransferase greater than 5 times upper limit of normal (ULN).
• Severe pulmonary disease requiring home nebulization and/or home oxygen therapy.
• Clinically significant gastrointestinal bleed within past 6-months.
• Neurological disease requiring either nasogastric feeding or intensive inpatient medical care.
• Active or history of seizures requiring anti-epileptics within 6 months prior to informed consent.

Sponsors & Collaborators

• Orphalan: lead

Study Sites (3)

University of Colorado Anschutz School of Medicine

Denver, Colorado, 80045, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of Michigan Medical Centre

Ann Arbor, Michigan, 48109-2029, United States

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Penicillamine

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Central Study Contacts

Carla Bennett, Bsc. Hons

CONTACT

+44 (0) 7918380893; clinicaloperations@orphalan.com

Omar Kamlin

CONTACT

clinicaloperations@orphalan.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomised, parallel group, open label.

Study Record Dates

• First Submitted: March 6, 2026
• First Posted: March 12, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2028
• Last Updated: May 28, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)