A Study to Assess the Safety of RG6496 in Huntington’s Disease Gene Expansion Carriers with the Selected Genetic Variant (POINT-HD)

2025-520631-17-00 Protocol BP45378 Human pharmacology (Phase I) - First administration to humans Authorised, recruitment pending

Status Authorised, recruitment pending · 6 EU/EEA countries · 16 sites · Protocol BP45378

Overview

Sponsor-declared trial summary

Phase Human pharmacology (Phase I) - First administration to humans
Status Authorised, recruitment pending
Participants planned 79
Countries 6
Sites 16

Huntington’s Disease Gene Expansion Carriers

Key facts

Sponsor
F. Hoffmann-La Roche AG
Participant type
Patients
Age range
18-64 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Decision date (initial)
2026-05-11
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Conditions and MedDRA coding

Huntington’s Disease Gene Expansion Carriers

VersionLevelCodeTermSystem organ class
28.0 PT 10070668 Huntington´s disease 100000004850

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

F. Hoffmann-La Roche AG

Sponsor organisation
F. Hoffmann-La Roche AG
Address
Grenzacherstrasse 124
City
Basel
Postcode
4058
Country
Switzerland

Scientific contact point

Organisation
F. Hoffmann-La Roche AG
Contact name
Trial Information System - TISL

Public contact point

Organisation
F. Hoffmann-La Roche AG
Contact name
Trial Information System - TISL

Locations

6 EU/EEA countries · 16 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Authorised, recruitment pending 3 1
Germany Authorised, recruitment pending 12 4
Italy Authorised, recruitment pending 4 2
Poland Authorised, recruitment pending 6 3
Portugal Authorised, recruitment pending 6 2
Spain Authorised, recruitment pending 12 4
Rest of world
Argentina, United Kingdom, Australia, Canada, New Zealand
36

Investigational sites

Denmark

1 site · Authorised, recruitment pending
Rigshospitalet
Clinical Research, Blegdamsvej 9, 2100, Copenhagen Oe

Germany

4 sites · Authorised, recruitment pending
Universitaetsklinikum Erlangen AöR
Molekular-Neurologische Abteilung, Schwabachanlage 6, Innenstadt, Erlangen
Universitaetsklinikum Ulm AöR
Klinik für Neurologie, Oberer Eselsberg 45, Eselsberg, Ulm
Charite Universitaetsmedizin Berlin KöR
Klinik für Psychiatrie und Psychotherapie, Chariteplatz 1, Mitte, Berlin
Katholisches Klinikum Bochum gGmbH
Forschungszentrum für Neurodegeneration, Gudrunstrasse 56, Grumme, Bochum

Italy

2 sites · Authorised, recruitment pending
Azienda Ospedaliero Universitaria Careggi
Clinical Trial Unit 1, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
IRCCS Foundation Istituto Neurologico Carlo Besta
Unità di Fase I, Via Giovanni Celoria 11, 20133, Milan

Poland

3 sites · Authorised, recruitment pending
Copernicus Podmiot Leczniczy Sp. z o.o.
Szpital Św. Wojciecha, Kliniczny Oddział Neurologiczny/ Udarowy GUmed, Al. Jana Pawla II 50, 80-462, Gdansk
Mtz Clinical Research Powered By Pratia
MTZ Clinical Research Powered by Pratia, Ul. Gładka 22, 02-172, Warsaw
Wojskowy Instytut Medycyny Lotniczej
Wojskowy Instytut Medycyny Lotniczej, Ul. Zygmunta Krasinskiego 54/56, 01-755, Warsaw

Portugal

2 sites · Authorised, recruitment pending
CCAB Centro Clinico Academico Braga Associacao
Neurology, Lugar De Sete Fontes S Victor, 4710-243, Braga
Unidade Local De Saude De Santa Maria E.P.E.
Neurology, Avenida Professor Egas Moniz, 1649-035, Lisbon

Spain

4 sites · Authorised, recruitment pending
Hospital Universitario De Badajoz
Neurology, Avenida Elvas S/n, 06006, Badajoz
Hospital De La Santa Creu I Sant Pau
Neurology, Carrer De San Quinti 89, 08041, Barcelona
Hospital Universitario De Burgos
Neurology, Avenida De Las Islas Baleares 3, 09006, Burgos
Hospital Universitario Ramon Y Cajal
Neurology, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2026-01-20 Denmark Acceptable
2026-05-11
2026-05-11