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- AMT-162 / uniQure
EPISOD1: A Phase 1/2, multicenter study to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162 in patients with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (Poster Hall at the Palais de Congres Montreal; 517b/c) - Nov 7, 2024 - Abstract #ALSMND2024ALS_MND_575;
P1/2
The clinical study design (NCT06100276) presented here is the first-in-human clinical trial using the investigational gene therapy AMT-162 focused on safety, tolerability, and exploratory efficacy in ALS patients with SOD1 mutations.
- || AMT-162 / uniQure
Enrollment open, Gene therapy: Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - Aug 4, 2024
P1/2, N=20, Recruiting, Sponsor: UniQure Biopharma B.V.
The clinical study design (NCT06100276) presented here is the first-in-human clinical trial using the investigational gene therapy AMT-162 focused on safety, tolerability, and exploratory efficacy in ALS patients with SOD1 mutations. Not yet recruiting --> Recruiting
- ||| AMT-162 / uniQure
Enrollment change, Gene therapy: Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - Jul 7, 2024
P1/2, N=20, Not yet recruiting, Sponsor: UniQure Biopharma B.V.
Not yet recruiting --> Recruiting N=12 --> 20
- ||| AMT-162 / uniQure
Enrollment change, Trial completion date, Trial initiation date, Trial primary completion date, Gene therapy: Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - May 15, 2024
P1/2, N=12, Not yet recruiting, Sponsor: UniQure Biopharma B.V.
N=12 --> 20 N=42 --> 12 | Trial completion date: Feb 2032 --> Mar 2031 | Initiation date: Feb 2024 --> May 2024 | Trial primary completion date: Jun 2026 --> Sep 2026
- || APB-101 / Apic Bio
Review, Journal: Alpha-methyltryptamine and 5-(2-methylaminopropyl)-benzofuran (5-MAPB) fatal co-intoxication: case report and review of literature. (Pubmed Central) - Apr 23, 2024
AMT and 5-MAPB were quantified using ultra-high performance liquid chromatography coupled to tandem mass spectrometry (UHPLC/MS-MS), revealing concentrations of AMT 4690 ng/mL and 5-MAPB 101 ng/mL in postmortem peripheral blood. We additionally reviewed the literature to help interpret the likely roles of these molecules in the occurrence of death.
- | AMT-162 / uniQure
Trial initiation date, Gene therapy: Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - Nov 19, 2023
P1/2, N=42, Not yet recruiting, Sponsor: UniQure Biopharma B.V.
We additionally reviewed the literature to help interpret the likely roles of these molecules in the occurrence of death. Initiation date: Nov 2023 --> Feb 2024
- || AMT-162 / uniQure
New P1/2 trial, Gene therapy: Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - Oct 25, 2023
P1/2, N=42, Not yet recruiting, Sponsor: UniQure Biopharma B.V.
- APB-102 / Apic Bio
Development of a ddPCR Based Relative Potency Assay for APB-102 (Poster Board Number: M-309; Hall D) - Apr 20, 2022 - Abstract #ASGCT2022ASGCT_1247;
An accelerated stability study showed that this potency assay is stability indicating and is more sensitive than a traditional TCID50 infectivity assay. The assay has been revised and streamlined to simplify the method after transfer to Quality Control for use as a release assay.