Clinical trial • Phase I/II • Neurology

Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA for Amyotrophic lateral sclerosis (ALS)

Phase I/II trial of Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA for Amyotrophic lateral sclerosis (ALS).

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Amyotrophic lateral sclerosis (ALS)
Trial Stage
Phase I/II
Drug Modality
Gene therapy
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
16-10-2024
First CTIS Authorization Date
28-01-2025

Trial design

None/Not specified-controlled, adaptive Phase I/II trial across 1 site in Sweden.

Comparator
None/Not specified
Adaptive
True, Single ascending dose (dose-escalation) design; no additional adaptive rules, interim analyses, or stopping rules are specified in the available data.
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
15

Eligibility

Recruits 15 No vulnerable population selected. Participants must be willing and able to provide informed consent (listed as an inclusion criterion). Subject information and informed consent forms are listed among the trial documents (Swedish language versions available). No assent or minor consent procedures are specified..

Vulnerable Population
No vulnerable population selected. Participants must be willing and able to provide informed consent (listed as an inclusion criterion). Subject information and informed consent forms are listed among the trial documents (Swedish language versions available). No assent or minor consent procedures are specified.

Inclusion criteria

  • {"criterion_text":"- Previously received investigational AMT-162 on the EPISOD1 (AMT-162-001) study at least 6 months prior to consent."}
  • {"criterion_text":"- Willing and able to provide informed consent"}

Exclusion criteria

  • {"criterion_text":"- 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 endpoints

  • {"endpoint_text":"- Incidence of treatment-emergent adverse events (TEAEs)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Mean change from baseline (BL) in clinical safety laboratory tests","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Mean change from BL in vital sign parameters","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Mean change from BL in electrocardiogram (ECG) parameters","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- AMT-162 vector shedding","definition_or_measurement_approach":""}
  • {"endpoint_text":"- B-cell and T-cell immunological response (neutralizing antibodies to AAVrh10 viral capsid, anti-AAV total IgG/IgM antibodies, and T cell response)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change from BL in slow vital capacity (SVC) percent of predicted value","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Hand-held dynamometry (HHD) scores","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Neurofilament light chain (NfL) protein levels in serum up to 5 years post-treatment","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
15
Recruitment Window Months
71
Consent Approach
Participants must be willing and able to provide informed consent (listed as an inclusion criterion). Subject information and informed consent forms are listed among trial documents (including Swedish-language ICFs). No assent procedures or minor-specific consent documents are specified; trial is for adult participants.

Geography

Total Number Of Sites
1
Total Number Of Participants
5

Sweden

Earliest CTIS Part Ii Submission Date
08-01-2025
Latest Decision Or Authorization Date
16-04-2025
Processing Time Days
98
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Umea University
Department Name
Neurology
Principal Investigator Name
Peter Munch Andersen
Principal Investigator Email
peter.andersen@umu.se
Contact Person Name
Peter Munch Andersen
Contact Person Email
peter.andersen@umu.se
Number Of Participants
5

Sponsor

Primary sponsor

Full Name
uniQure biopharma B.V.
Organisation Type
Pharmaceutical company
Country Of Registered Address
Netherlands

Investigational products

Investigational Product Name
AMT-162
Active Substance
Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA
Modality
Gene therapy
Routes Of Administration
Intrathecal
Route
Intrathecal
Orphan Designation
Yes
Frequency
Single dose

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