Early Access Program for ALXN1840 in Patients With Wilson Disease

NCT05686564 | Unknown

• 1 other identifier: D9430R00001
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.

Conditions

Wilson Disease

Keywords

Wilson Disease; ALXN1840

Interventions

ALXN1840 DRUG

bis-choline tetrathiomolybdate

Also known as: INN: tiomolibdic acid; USAN/JAN: tiomolibdate choline (WTX101)

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840.
• Confirmed diagnosis of WD.
• Has completed one of the following ALXN1840 clinical studies:
• ALXN1840-WD-205
• WTX101-301
• ALXN1840-WD-302
• Other ALXN1840 studies
• In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant.
• Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP.
• Female participants of childbearing potential and male participants must follow protocol-specified-contraception guidance.

You may not qualify if:

• Eligible for and able to participate in an Alexion-sponsored study of ALXN1840.
• Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.
• Has decompensated hepatic cirrhosis.
• Model for End-Stage Liver Disease (MELD) score \> 13.
• Modified Nazer Score \> 7.
• End-stage renal disease on dialysis (chronic kidney disease stage 5 \[CKD 5\]) or creatinine clearance \< 30 mL/min.
• Known hypersensitivity to ALXN1840, ALXN1840 excipients, or any of the ingredients contained in ALXN1840.
• Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with ALXN1840).

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Hepatolenticular Degeneration

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

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2023
• First Posted: January 17, 2023
• Last Updated: May 17, 2023

Record last verified: 2023-05