Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Wilson Disease Clinical Trial

Official title:

A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05047523
• Other study ID #: ALXN1840-WD-302
• Secondary ID: 2021-001015-82
• Status: Terminated
• Phase: Phase 3
• Start date: October 6, 2021
• Est. completion date: June 26, 2023

Study information

• Verified date: December 2023
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).

Description:

Participants who complete the 48 weeks of treatment in Period 1 will have the option to receive ALXN1840 for 24 weeks in Period 2 (open-label extension).

Safety will be monitored throughout the study.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 40
• Est. completion date: June 26, 2023
• Est. primary completion date: June 26, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 17 Years

Eligibility

Key Inclusion Criteria:

1. Diagnosis of Wilson Disease by Leipzig Score = 4.

2. Adequate venous access to allow collection of required blood samples.

3. Able to swallow intact ALXN1840 tablets or mini-tablets.

4. Willing to avoid intake of foods and drinks with high contents of copper.

5. Willing and able to follow protocol-specified contraception requirements.

Key Exclusion Criteria:

1. Decompensated hepatic cirrhosis or MELD score > 13 (ages 12 to <18) or PELD score > 13 (ages 3 to < 12).

2. Modified Nazer score > 7.

3. Clinically significant gastrointestinal bleed within past 3 months.

4. Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) for participants treated for > 28 days with WD therapy or ALT > 5 × ULN for treatment-naïve participants or participants who have been treated for = 28 days.

5. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.

6. Hemoglobin less than lower limit of the reference range for age and sex.

7. History of seizure activity within 6 months prior to informed consent/assent.

8. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or estimated glomerular filtration rate < 30 milliliters/minute/1.73 meter squared.

Related Conditions & MeSH terms

• Wilson Disease

Intervention

Drug:
• ALXN1840
Administered as an oral tablet.
• Standard of Care
Depending on the site/region, participants randomized to receive Standard of Care treatment will receive trientine, penicillamine, zinc, or a combination of these medicines, administered according to standard regimens.

Locations

Country	Name	City	State
Australia	Clinical Trial Site	Parkville
Australia	Clinical Trial Site	South Brisbane
France	Clinical Trial Site	Bron
France	Clinical Trial Site	Lille
France	Clinical Trial Site	Paris
France	Clinical Trial Site	Paris
France	Clinical Trial Site	Toulouse
Germany	Clinical Trial Site	Hamburg
Germany	Clinical Trial Site	Hanover
Germany	Clinical Trial Site	Tuebingen
Japan	Clinical Trial Site	Chiba
Japan	Clinical Trial Site	Kumamoto
Japan	Clinical Trial Site	Kurume
Japan	Clinical Trial Site	Meguro-Ku
Japan	Clinical Trial Site	Sapporo
Korea, Republic of	Clinical Trial Site	Seoul
Korea, Republic of	Clinical Trial Site 2	Seoul
Korea, Republic of	Clinical Trial Site3	Seoul
Poland	Clinical Trial Site	Warsaw
Spain	Clinical Trial Site	Barcelona
Spain	Clinical Trial Site	Barcelona
Spain	Clinical Trial Site	Las Palmas de Gran Canaria
Spain	Clinical Trial Site	Madrid
Spain	Clinical Trial Site	Malaga
Spain	Clinical Trial Site	Pamplona
United Kingdom	Clinical Trial Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

Australia,  France,  Germany,  Japan,  Korea, Republic of,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage Change From Baseline To Week 48 In Non-ceruloplasmin-bound Copper (NCC) In Plasma		Baseline, Week 48
Secondary	Area Under The Effect Versus Time Curve (AUEC) For NCC And Plasma Total Copper		Week 48
Secondary	Observed Change From Baseline To Week 48 Of Ceruloplasmin-bound Copper And Ceruloplasmin		Baseline, Week 48
Secondary	NCC Responder Rate		Week 48
Secondary	Change From Baseline To Week 48 In The UWDRS Part II Total Score		Baseline, Week 48
Secondary	Change From Baseline To Week 48 In The UWDRS Part III Total Score		Baseline, Week 48
Secondary	PK: Maximum Observed Concentration (Cmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations		Up to Week 48
Secondary	PK: Time To Maximum Concentration (Tmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations		Up to Week 48
Secondary	PK: Area Under The Plasma Concentration Versus Time Curve From Time 0 To The End Of The Dosing Interval (AUCtau) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations		Up to Week 48
Secondary	Clinical Global Impression-improvement (CGI-I), As Assessed By The Investigator		Week 48
Secondary	Change From Baseline To Week 48 In Clinical Global Impression-severity (CGI-S), As Assessed By The Investigator		Baseline, Week 48
Secondary	Change From Baseline To Week 48 In Model For End-stage Liver Disease (MELD) Score (Ages 12 Years And Older) Or Pediatric End-stage Liver Disease (PELD) Score (Ages 3 To < 12 Years)		Baseline, Week 48
Secondary	Change From Baseline To Week 48 In Modified Nazer Score		Baseline, Week 48