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

Primary Purpose

Wilson Disease

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

ALXN1840

Standard of Care

About this trial

This is an interventional treatment trial for Wilson Disease focused on measuring Wilson Disease, Pediatric

Eligibility Criteria

3 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Diagnosis of Wilson Disease by Leipzig Score ≥ 4.
Adequate venous access to allow collection of required blood samples.
Able to swallow intact ALXN1840 tablets or mini-tablets.
Willing to avoid intake of foods and drinks with high contents of copper.
Willing and able to follow protocol-specified contraception requirements.

Key Exclusion Criteria:

Decompensated hepatic cirrhosis or MELD score > 13 (ages 12 to <18) or PELD score > 13 (ages 3 to < 12).
Modified Nazer score > 7.
Clinically significant gastrointestinal bleed within past 3 months.
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.
Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
Hemoglobin less than lower limit of the reference range for age and sex.
History of seizure activity within 6 months prior to informed consent/assent.
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.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

ALXN1840

Standard of Care

Arm Description

ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted.

Participants will receive their current therapy or initiate Standard of Care therapy.

Outcomes

Primary Outcome Measures

Percentage Change From Baseline To Week 48 In Non-ceruloplasmin-bound Copper (NCC) In Plasma

Secondary Outcome Measures

Area Under The Effect Versus Time Curve (AUEC) For NCC And Plasma Total Copper

Observed Change From Baseline To Week 48 Of Ceruloplasmin-bound Copper And Ceruloplasmin

NCC Responder Rate

Change From Baseline To Week 48 In The UWDRS Part II Total Score

Change From Baseline To Week 48 In The UWDRS Part III Total Score

PK: Maximum Observed Concentration (Cmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations

PK: Time To Maximum Concentration (Tmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations

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

Clinical Global Impression-improvement (CGI-I), As Assessed By The Investigator

Change From Baseline To Week 48 In Clinical Global Impression-severity (CGI-S), As Assessed By The Investigator

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)

Change From Baseline To Week 48 In Modified Nazer Score

Full Information

NCT ID

NCT05047523

First Posted

September 9, 2021

Last Updated

July 11, 2023

Sponsor

Alexion

1. Study Identification

Unique Protocol Identification Number

NCT05047523

Brief Title

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

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Terminated

Why Stopped

Sponsor decision to terminate the program

Study Start Date

October 6, 2021 (Actual)

Primary Completion Date

June 26, 2023 (Actual)

Study Completion Date

June 26, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Wilson Disease

Keywords

Wilson Disease, Pediatric

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

Outcomes Assessor

Masking Description

This study is rater-blinded for the Unified Wilson Disease Rating Scale (UWDRS) assessment only.

Allocation

Randomized

Enrollment

40 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ALXN1840

Arm Type

Experimental

Arm Description

ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted.

Arm Title

Standard of Care

Arm Type

Active Comparator

Arm Description

Participants will receive their current therapy or initiate Standard of Care therapy.

Intervention Type

Drug

Intervention Name(s)

ALXN1840

Other Intervention Name(s)

Formerly WTX101

Intervention Description

Administered as an oral tablet.

Intervention Type

Drug

Intervention Name(s)

Standard of Care

Intervention Description

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.

Primary Outcome Measure Information:

Title

Percentage Change From Baseline To Week 48 In Non-ceruloplasmin-bound Copper (NCC) In Plasma

Time Frame

Baseline, Week 48

Secondary Outcome Measure Information:

Title

Area Under The Effect Versus Time Curve (AUEC) For NCC And Plasma Total Copper

Time Frame

Week 48

Title

Observed Change From Baseline To Week 48 Of Ceruloplasmin-bound Copper And Ceruloplasmin

Time Frame

Baseline, Week 48

Title

NCC Responder Rate

Time Frame

Week 48

Title

Change From Baseline To Week 48 In The UWDRS Part II Total Score

Time Frame

Baseline, Week 48

Title

Change From Baseline To Week 48 In The UWDRS Part III Total Score

Time Frame

Baseline, Week 48

Title

PK: Maximum Observed Concentration (Cmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations

Time Frame

Up to Week 48

Title

PK: Time To Maximum Concentration (Tmax) Of ALXN1840 For Plasma Total Molybdenum And Plasma Ultrafiltrate Molybdenum Concentrations

Time Frame

Up to Week 48

Title

Time Frame

Up to Week 48

Title

Clinical Global Impression-improvement (CGI-I), As Assessed By The Investigator

Time Frame

Week 48

Title

Change From Baseline To Week 48 In Clinical Global Impression-severity (CGI-S), As Assessed By The Investigator

Time Frame

Baseline, Week 48

Title

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)

Time Frame

Baseline, Week 48

Title

Change From Baseline To Week 48 In Modified Nazer Score

Time Frame

Baseline, Week 48

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Diagnosis of Wilson Disease by Leipzig Score ≥ 4. Adequate venous access to allow collection of required blood samples. Able to swallow intact ALXN1840 tablets or mini-tablets. Willing to avoid intake of foods and drinks with high contents of copper. Willing and able to follow protocol-specified contraception requirements. Key Exclusion Criteria: Decompensated hepatic cirrhosis or MELD score > 13 (ages 12 to <18) or PELD score > 13 (ages 3 to < 12). Modified Nazer score > 7. Clinically significant gastrointestinal bleed within past 3 months. 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. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care. Hemoglobin less than lower limit of the reference range for age and sex. History of seizure activity within 6 months prior to informed consent/assent. 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Eugene S. Swenson, MD, PhD

Organizational Affiliation

Alexion

Official's Role

Study Director

Facility Information:

Facility Name

Clinical Trial Site

City

Parkville

ZIP/Postal Code

VIC 3052

Country

Australia

Facility Name

Clinical Trial Site

City

South Brisbane

ZIP/Postal Code

4101

Country

Australia

Facility Name

Clinical Trial Site

City

Bron

ZIP/Postal Code

69667

Country

France

Facility Name

Clinical Trial Site

City

Lille

ZIP/Postal Code

59037

Country

France

Facility Name

Clinical Trial Site

City

Paris

ZIP/Postal Code

7515

Country

France

Facility Name

Clinical Trial Site

City

Paris

ZIP/Postal Code

94270

Country

France

Facility Name

Clinical Trial Site

City

Toulouse

ZIP/Postal Code

31059

Country

France

Facility Name

Clinical Trial Site

City

Hamburg

ZIP/Postal Code

20246

Country

Germany

Facility Name

Clinical Trial Site

City

Hanover

ZIP/Postal Code

30625

Country

Germany

Facility Name

Clinical Trial Site

City

Tuebingen

ZIP/Postal Code

72076

Country

Germany

Facility Name

Clinical Trial Site

City

Chiba

ZIP/Postal Code

266-0007

Country

Japan

Facility Name

Clinical Trial Site

City

Kumamoto

ZIP/Postal Code

860-8556

Country

Japan

Facility Name

Clinical Trial Site

City

Kurume

ZIP/Postal Code

830-0011

Country

Japan

Facility Name

Clinical Trial Site

City

Meguro-Ku

ZIP/Postal Code

153-8515

Country

Japan

Facility Name

Clinical Trial Site

City

Sapporo

ZIP/Postal Code

063-0005

Country

Japan

Facility Name

Clinical Trial Site3

City

Seoul

ZIP/Postal Code

03080

Country

Korea, Republic of

Facility Name

Clinical Trial Site

City

Seoul

ZIP/Postal Code

03722

Country

Korea, Republic of

Facility Name

Clinical Trial Site 2

City

Seoul

ZIP/Postal Code

06351

Country

Korea, Republic of

Facility Name

Clinical Trial Site

City

Warsaw

ZIP/Postal Code

04-730

Country

Poland

Facility Name

Clinical Trial Site

City

Barcelona

ZIP/Postal Code

08035

Country

Spain

Facility Name

Clinical Trial Site

City

Barcelona

ZIP/Postal Code

08950

Country

Spain

Facility Name

Clinical Trial Site

City

Las Palmas de Gran Canaria

ZIP/Postal Code

35016

Country

Spain

Facility Name

Clinical Trial Site

City

Madrid

ZIP/Postal Code

28041

Country

Spain

Facility Name

Clinical Trial Site

City

Malaga

ZIP/Postal Code

29010

Country

Spain

Facility Name

Clinical Trial Site

City

Pamplona

ZIP/Postal Code

31008

Country

Spain

Facility Name

Clinical Trial Site

City

London

ZIP/Postal Code

SE59RS

Country

United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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

Wilson Disease

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial