Clinical trial NCT05047523: Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Wilson Disease
Keywords: Wilson Disease
Pediatric
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-09-17
Enrollment: 40
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
timeFrame: Baseline, Week 48
Secondary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
timeFrame: Baseline up to Week 48
measure: Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
timeFrame: Week 48
measure: Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
timeFrame: Week 48
measure: 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
timeFrame: Week 48
Other outcomes:
Locations:
city: Parkville
country: Australia
facility: Research Site
city: South Brisbane
country: Australia
facility: Research Site
city: Lille
country: France
facility: Research Site
city: Toulouse
country: France
facility: Research Site
city: Hannover
country: Germany
facility: Research Site
city: Tübingen
country: Germany
facility: Research Site
city: Kumamoto-shi
country: Japan
facility: Research Site
city: Kurume-shi
country: Japan
facility: Research Site
city: Meguro-ku
country: Japan
facility: Research Site
city: Sapporo-shi
country: Japan
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Warsaw
country: Poland
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Esplugues de Llobregat
country: Spain
facility: Research Site
city: Las Palmas de Gran Canaria
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Málaga
country: Spain
facility: Research Site
city: Pamplona
country: Spain
facility: Research Site
Study officials:
affiliation: Alexion Pharmaceuticals, Inc.
name: Eugene S. Swenson, MD, PhD
role: STUDY_DIRECTOR
References: