Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
60
Countries
5
Sites
7
Amyotrophic Lateral Sclerosis (ALS)
To evaluate the safety and tolerability of ARGX-119 in participants with ALS
Key facts
- Sponsor
- Argenx
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 23 Oct 2024 → 26 Feb 2026
- Decision date (initial)
- 2024-09-30
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- argenx bv
External identifiers
- EU CT number
- 2024-511318-19-00
- ClinicalTrials.gov
- NCT06441682
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Efficacy, Safety
To evaluate the safety and tolerability of ARGX-119 in participants with ALS
Secondary objectives 2
- To assess the efficacy of ARGX-119 on electrophysiological measures of disease progression in participants with ALS
- To assess the PK and immunogenicity of ARGX-119 in participants with ALS
Conditions and MedDRA coding
Amyotrophic Lateral Sclerosis (ALS)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10002026 | Amyotrophic lateral sclerosis | 100000004852 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Double-blinded treatment, Active-Extension treatment Participants will receive ARGX-119 or placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
|
Randomised Controlled | Double | [{"id":175298,"code":4,"name":"Analyst"},{"id":175300,"code":1,"name":"Subject"},{"id":175296,"code":5,"name":"Carer"},{"id":175297,"code":3,"name":"Monitor"},{"id":175299,"code":2,"name":"Investigator"}] | ARGX-119: Participants will receive first dosage level of ARGX-119 intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period ARGX-119: Participants will receive second dosage level of ARGX-119 intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period ARGX-119: Participants will receive third dosage level of ARGX-119 intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period Placebo: Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period |
Regulatory references
- Scientific advice from competent authorities
- Medicines Evaluation Board, Federal Agency For Medicines And Health Products, Food And Drug Administration
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-509872-41-00 | A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7 Congenital Myasthenic Syndromes | Argenx |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- The participant is at least 18 and ≤80 years of age
- The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria
- The participant has a Treatment Research Initiative to Cure ALS (TRICALS) risk profile of ≥ −6.0 to < −2.0
- SVC of ≥ 60% of the predicted value according to Global Lung Function Initiative 2012
Exclusion criteria 3
- Use of noninvasive ventilation more than 10 hours a day or use of a tracheostomy for ventilatory support
- Any history of or current exposure to any gene or cell therapies (off-label use or investigational) for ALS
- Pregnant or lactating state or intention to become pregnant during the study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Assessment of adverse events (AEs)
- Assessment of clinical laboratory tests, ECGs and vital signs
Secondary endpoints 4
- Rate of change from baseline to week 24 in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)
- ARGX-119 pharmacokinetic (PK) parameters over time
- Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
- Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10388517 · Product
- Active substance
- Adimanebart
- Substance synonyms
- ARGX-119, Humanised IgG1 monoclonal antibody against muscle specific kinase
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 00 mg/kg milligram(s)/kilogram
- Max total dose
- 00 mg/kg milligram(s)/kilogram
- Max treatment duration
- 72 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ARGENX BV
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
Placebo to ARGX-119 concentrate for solution for infusion
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Argenx
- Sponsor organisation
- Argenx
- Address
- Industriepark-Zwijnaarde 7
- City
- Gent
- Postcode
- 9052
- Country
- Belgium
Scientific contact point
- Organisation
- Argenx
- Contact name
- Peter Ulrichts, PhD
Public contact point
- Organisation
- Argenx
- Contact name
- Sabine Coppieters, MD
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| Optimapharm Nordic Oy ORG-100009126
|
Espoo, Finland | On site monitoring |
| Julius Clinical International B.V. ORG-100028683
|
Zeist, Netherlands | On site monitoring, Code 12, Code 2, Code 5 |
Locations
5 EU/EEA countries · 7 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ended | 8 | 1 |
| Denmark | Ended | 6 | 2 |
| France | Ended | 7 | 2 |
| Netherlands | Ended | 18 | 1 |
| Sweden | Ended | 10 | 1 |
| Rest of world
Canada
|
— | 11 | — |
Investigational sites
Aarhus Universitetshospital
Department of Neurology, Palle Juul-Jensens Boulevard 99, 8200, Aarhus N
Copenhagen University Hospital
Department of Neurology, Bispebjerg Bakke 23, 2400, Copenhagen Nv
Centre Hospitalier Regional Universitaire De Tours
Neurology, 2 Boulevard Tonnelle, 37000, Tours
Hopitaux Universitaires Pitie Salpetriere
Neurology, 47 To 83 Boulevard De L Hopital, 75013, Paris
Universitair Medisch Centrum Utrecht
Neurology, Heidelberglaan 100, 3584 CX, Utrecht
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2024-11-05 | 2024-11-12 | |||
| Denmark | 2024-12-11 | 2024-12-19 | |||
| France | 2025-02-03 | 2025-02-06 | |||
| Netherlands | 2024-10-23 | 2024-10-29 | |||
| Sweden | 2024-12-12 | 2024-12-17 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Serious breaches 1 · Art. 52 CTR
Serious breach SB-82402
- Sponsor became aware
- 2025-05-06
- Date of breach
- 2024-11-21
- Submission date
- 2025-05-13
- Member states concerned
- Belgium, Denmark, France, Sweden, Netherlands
- Categories
- Protocol
- Areas impacted
- Subject rights
- Benefit-risk balance changed
- Yes
- Description
- 15 randomised patients in the Universitair Medisch Centrum Utrecht Site have been receiving the ARGX-119 or placebo (71 doses) without ancillary Inline IV infusion filter with luer-lock (required per argenx IP handling manual and as a standard safety measure).
This was reported to argenx by contracted CRO Julius Clinical on 06May2025 after the site identified the error themselves. As from 07May2025, site started to use the inline filters.
Inline filters are on the list of the ancillary supplies and per IP Handling Manual for this study, site should use their own ancillaries, or they can be provided by CRO/Sponsor upon request and approval. Site did not raise any concerns or requests until 06May2025 when this was identified by the site. This error was not identified during routine checks and monitoring. - Sponsor actions
- Please see attached supportive document
| Organisation | City | Country | Type |
|---|---|---|---|
| Universitair Medisch Centrum Utrecht | Utrecht | Netherlands | Clinical investigator |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 30 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-511318-19_For publication | 4.0 |
| Protocol (for publication) | D4_Patient Questionnaires Publication Statement_For publication | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1.2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1.1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangments | 1.2 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Website text_information letter | 1.2 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website text_Information letter | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website text_information letter | 1.1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website text_information letter | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website text_information letter | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_Dutch_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future Research | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Dutch_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_English_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_French_For publication | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_For publication | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_For publication | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_Dutch_2024-511318-19_For publication | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_English_2024-511318-19_For publication | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_French_2024-511318-19_For publication | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_German_2024-511318-19_For publication | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_Swedish_2024-511318-19_For publication | 4.0 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-06-07 | Denmark | Acceptable with conditions 2024-09-23
|
2024-09-23 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-10-25 | Denmark | Acceptable 2025-01-14
|
2025-01-14 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-03-31 | Denmark | Acceptable 2025-05-15
|
2025-05-15 |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-07-11 | Denmark | Acceptable 2025-08-29
|
2025-08-29 |
| 5 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-03-06 | Denmark | Acceptable | 2026-03-12 |