Clinical trial • Phase I • Neurology

ARO-ATXN2 for Spinocerebellar ataxia type 2

Phase I trial of ARO-ATXN2 for Spinocerebellar ataxia type 2. 20 participants.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Spinocerebellar ataxia type 2
Trial Stage
Phase I

Key dates

Initial CTIS Submission Date
03-02-2025
First CTIS Authorization Date
13-05-2025

Trial design

Phase I trial across 13 sites in Spain, Italy, France and others.

Target Sample Size
20

Eligibility

Recruits 20 No vulnerable population selected (isVulnerablePopulationSelected=false). No information on consent or assent handling provided..

Vulnerable Population
No vulnerable population selected (isVulnerablePopulationSelected=false). No information on consent or assent handling provided.

Recruitment

Planned Sample Size
20
Recruitment Window Months
14

Geography

Total Number Of Sites
13
Total Number Of Participants
20

Spain

Earliest CTIS Part Ii Submission Date
08-05-2025
Latest Decision Or Authorization Date
30-10-2025
Processing Time Days
175
Number Of Sites
4
Number Of Participants
4

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Contact Person Name
Jorge Hernandez
Contact Person Email
jorge.hernandez@vallhebron.cat
Site Name
Hospital Clinic De Barcelona
Department Name
Neurologist
Contact Person Name
Jose Esteban Muñoz Garcia
Contact Person Email
jemunoz@clinic.cat
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Neurology
Contact Person Name
Pablo Mir
Contact Person Email
pmir@us.es
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurologist
Contact Person Name
Irene Martinez Torres
Contact Person Email
martinez_ire@gva.es

Italy

Earliest CTIS Part Ii Submission Date
17-04-2025
Latest Decision Or Authorization Date
27-10-2025
Processing Time Days
193
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Dept. of Neuroscience " Rita Levi Montalcini "
Contact Person Name
Andrea Calvo
Contact Person Email
andrea.calvo@unito.it
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Unit of Medical Genetics and Neurogenetics
Contact Person Name
Caterina Mariotti

France

Earliest CTIS Part Ii Submission Date
24-03-2025
Latest Decision Or Authorization Date
27-10-2025
Processing Time Days
217
Number Of Sites
5
Number Of Participants
6

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Medical genetics
Contact Person Name
Durr Alexandra
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Neurology and movement disorder unit
Contact Person Name
Eugenie Mutez
Contact Person Email
eugenie.mutez@chu-lille.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Neurology
Contact Person Name
Cecilia MARELLI
Contact Person Email
c-marelli@chu-montpellier.fr
Site Name
Centre Hospitalier Universitaire D'Angers
Department Name
Neurology
Contact Person Name
Christophe Verny
Contact Person Email
chverny@chu-angers.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Medical genetics
Contact Person Name
Cyril Goizet
Contact Person Email
cyril.goizet@chu-bordeaux.fr

Germany

Earliest CTIS Part Ii Submission Date
05-05-2025
Latest Decision Or Authorization Date
29-10-2025
Processing Time Days
177
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Universitaetsklinikum Ulm AöR
Department Name
Studienzentrum Neurologie
Contact Person Name
David Brenner
Contact Person Email
david.brenner@uni-ulm.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Center of Neurology
Contact Person Name
Matthis Synofzik

Sponsor

Primary sponsor

Full Name
Arrowhead Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Investigational products

Investigational Product Name
ARO-ATXN2
Routes Of Administration
Injection
Route
Injection

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