Overview
Sponsor-declared trial summary
Amyotrophic Lateral Sclerosis (ALS)
To evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in Participants with SOD1-ALS
Key facts
- Sponsor
- uniQure biopharma B.V.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 3 Mar 2025 → ongoing
- Decision date (initial)
- 2025-01-28
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-517244-57-00
- ClinicalTrials.gov
- NCT06100276
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Efficacy, Safety
To evaluate the safety and tolerability of ascending
doses of intrathecally administered AMT-162 in
Participants with SOD1-ALS
Secondary objectives 2
- To characterize the immune response to AMT-162 and shedding of intrathecally administered AMT162
- To characterize the effect of intrathecally administered AMT-162
Conditions and MedDRA coding
Amyotrophic Lateral Sclerosis (ALS)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10002026 | Amyotrophic lateral sclerosis | 100000004852 |
Regulatory references
- Scientific advice from competent authorities
- uniQure biopharma B.V.
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Previously received investigational AMT-162 on the EPISOD1 (AMT-162-001) study at least 6 months prior to consent.
- Willing and able to provide informed consent
Exclusion criteria 1
- Presence of any condition which, in the opinion of the Investigator, might put the Participant at unacceptable risk due to participation in the stud
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 4
- Incidence of treatment-emergent adverse events (TEAEs)
- Mean change from baseline (BL) in clinical safety laboratory tests
- Mean change from BL in vital sign parameters
- Mean change from BL in electrocardiogram (ECG) parameters
Secondary endpoints 5
- AMT-162 vector shedding
- B-cell and T-cell immunological response (neutralizing antibodies to AAVrh10 viral capsid, anti-AAV total IgG/IgM antibodies, and T cell response)
- Change from BL in slow vital capacity (SVC) percent of predicted value
- Hand-held dynamometry (HHD) scores
- Neurofilament light chain (NfL) protein levels in serum up to 5 years post-treatment
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Adeno-Associated Viral Vector Serotype RH10 Encoding Mirna Against SOD1 Mrna
PRD11664530 · Product
- Active substance
- Adeno-Associated Viral Vector Serotype RH10 Encoding Mirna Against SOD1 Mrna
- Substance synonyms
- AAVrh10-H1-amiR-SOD1, AMT-162, APB-102
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Authorisation status
- Not Authorised
- MA holder
- UNIQURE BIOPHARMA B.V
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- ODD EU/3/23/2859
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
uniQure biopharma B.V.
- Sponsor organisation
- uniQure biopharma B.V.
- Address
- Paasheuvelweg 25
- City
- Amsterdam
- Postcode
- 1105 BP
- Country
- Netherlands
Scientific contact point
- Organisation
- uniQure biopharma B.V.
- Contact name
- Natascha Schillemans
Public contact point
- Organisation
- uniQure biopharma B.V.
- Contact name
- Natascha Schillemans
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Sweden | Ongoing, recruitment ended | 5 | 1 |
| Rest of world
United States
|
— | 15 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| Sweden | 2025-03-03 | 2025-03-03 | 2026-04-07 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 6 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | AMT-162-001-Protocol-V6-1-LTFU-28Aug2024-FINAL-Redacted | 6-2 |
| Recruitment arrangements (for publication) | informedconsent-patientrecruitmentprocedure-SV clean | 1 |
| Subject information and informed consent form (for publication) | AMT-162_001_MasterICF_LTFU_v6_2perProtAmend_03Jan2025 clean SE | 2 |
| Subject information and informed consent form (for publication) | AMT-162-001_MasterICF_LTFU_28Aug2024_ReadyforSignoff_sv_SE | 1 |
| Synopsis of the protocol (for publication) | AMT-162-001_Protocol Synopsis _V6_2_LTFU_sv_SE | 6-2 |
| Synopsis of the protocol (for publication) | AMT-162-001-Protocol-V6-1-LTFU-Synopsis-English-Swedish | 1 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-16 | Sweden | Acceptable 2025-01-27
|
2025-01-28 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-04-16 | Sweden | Acceptable 2025-01-27
|
2025-04-16 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-06-13 | Sweden | Acceptable 2025-01-27
|
2025-06-13 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-10-23 | Sweden | Acceptable 2025-01-27
|
2025-10-23 |