Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Ongoing, recruiting
Participants planned
36
Countries
4
Sites
13
spinocerebellar ataxia type 2
Key facts
- Sponsor
- Arrowhead Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 4 Dec 2025 → ongoing
- Decision date (initial)
- 2025-05-14
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-514763-25-01
- WHO UTN
- U1111-1308-5875
- ClinicalTrials.gov
- NCT06672445
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
spinocerebellar ataxia type 2
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.0 | LLT | 10057660 | Spinocerebellar ataxia | 10010331 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Arrowhead Pharmaceuticals Inc.
- Sponsor organisation
- Arrowhead Pharmaceuticals Inc.
- Address
- 177 East Colorado Boulevard Suite 700
- City
- Pasadena
- Postcode
- 91105-1976
- Country
- United States
Scientific contact point
- Organisation
- Arrowhead Pharmaceuticals Inc.
- Contact name
- Grigoriy Shekhtman, Medical Director
Public contact point
- Organisation
- Arrowhead Pharmaceuticals Inc.
- Contact name
- Medical Monitor
Locations
4 EU/EEA countries · 13 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 6 | 5 |
| Germany | Authorised, recruiting | 4 | 2 |
| Italy | Authorised, recruiting | 2 | 2 |
| Spain | Ongoing, recruiting | 4 | 4 |
| Rest of world
New Zealand, Canada, United Kingdom, Taiwan, Australia
|
— | 20 | — |
Investigational sites
Centre Hospitalier Universitaire D'Angers
Neurology, 4 Rue Larrey, 49100, Angers
Assistance Publique Hopitaux De Paris
Medical genetics, 47 Boulevard De L Hopital, 75651, Paris Cedex 13
Centre Hospitalier Universitaire De Bordeaux
Medical genetics, Place Amelie Raba Leon, 33000, Bordeaux
Centre Hospitalier Universitaire De Lille
Neurology and movement disorder unit, Boulevard Du Professeur Jules Leclercq, 59000, Lille
Centre Hospitalier Universitaire De Montpellier
Neurology, 80 Avenue Augustin Fliche, 34295, Montpellier Cedex 5
Universitaetsklinikum Ulm AöR
Studienzentrum Neurologie, Oberer Eselsberg 45, Eselsberg, Ulm
Universitaetsklinikum Tuebingen AöR
Center of Neurology, Hoppe-Seyler-Strasse 3, Nordstadt, Tuebingen
IRCCS Foundation Istituto Neurologico Carlo Besta
Unit of Medical Genetics and Neurogenetics, Via Giovanni Celoria 11, 20133, Milan
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Dept. of Neuroscience " Rita Levi Montalcini ", Corso Bramante 88, 10126, Turin
Hospital Universitari Vall D Hebron
Neurology, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
University Hospital Virgen Del Rocio S.L.
Neurology, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Clinic De Barcelona
Neurologist, Calle Villarroel 170, 08036, Barcelona
Hospital Universitario Y Politecnico La Fe
Neurologist, Avenida Fernando Abril Martorell 106, 46026, Valencia
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 |
|---|---|---|---|---|---|
| France | 2026-01-16 | 2026-01-20 | |||
| Germany | 2026-01-12 | ||||
| Italy | 2026-01-19 | ||||
| Spain | 2025-12-04 | 2026-01-09 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-02-03 | Spain | Acceptable with conditions 2025-05-13
|
2025-05-13 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-07-23 | Spain | Acceptable 2025-10-27
|
2025-10-27 |