Interventional trials in ENR-RND – Ataxia and HSP – ERN-RND | European Reference Network on Rare Neurological Diseases

Disease	Treatment	Center	Contact	Key information
Friedreich Ataxia	Vatiquinone	Paris Brain Institute - Pitié Sapetriere University Hospital Paris France	Contact
SCA3	Trehalose	Hospital Clinic Barcelona Spain	Contact	Patient recruitment temporarily halted
Hereditary Spastic Paraplegia		University Hospital Innsbruck Austria	Contact	not yet registered
SCA1 and SCA3	Antisense oligonucleotide, intrathecally injected; phase1/2A	Radboud university medical center Nijmegen Netherlands	Contact	Recruitment of Dutch patients only
Friedreich Ataxia	Omaveloxolon	Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy
Friedreich Ataxia	Phase 3	Bambino Gesù — Children's Hospital Rome Italy
Friedreich Ataxia	Vatiquinone	Bambino Gesù — Research Hospital Rome Italy	Rene Gallegos- Associate Director Clinical Operations MOVE-FA Briana Johnson - Manager Clinical Operations PTC743-003e
FRDA	N/A	TUH Dublin Ireland	Contact
SCA and CANVAS	rTMS	University Hospital Bonn Germany	Contact
MSA	i.v. TAK-341	DZNE Bonn Germany	Contact
SCA1, SCA3, Huntington	ASO	DZNE Bonn Germany	Contact
SCA3	Intrathecal ASO	DZNE Bonn Germany	Contact	trial stopped
Friedreich Ataxia	Omaveloxolon	Center for rare movement disorders Innsbruck Austria	Contact