Huntington's disease

9 trials 3 recruiting MedDRA PT
9 Registered trials
3 Recruiting
9 Unique sponsors
8 Unique IMPs
7 Commercial trials
Drug classes (ATC)
— ATC code not specified 9
Mechanism of action (INN-stem analysis)
Unknown / small molecule 7 substances
Antisense oligonucleotide 1 substance
Phase III: 1 Phase II: 4 Phase I/II: 1 Phase I: 1 Phase I (First in Human): 2

Filter these 9 trials by country, sponsor, date range and more.

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Show: 9 trials

Phase III

1 trial
  • Assistance Publique Hopitaux De Paris Academic
    2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE
    2024-516022-63-00 1 country 2024 n=100
    Recruiting

Phase II

4 trials
  • F. Hoffmann-La Roche AG Commercial
    TOMINERSEN
    2023-503928-10-00 8 countries 2023 n=162
    Ended
  • Medibiofarma S.L. Commercial
    MBF-015
    2023-505241-10-00 1 country 2024 n=10
    Ended
  • Novartis Pharma AG Commercial
    VOTOPLAM
    2023-504628-24-00 6 countries 2023 n=172 Orphan
    Ended
  • PTC Therapeutics Inc. Commercial
    PTC518
    2023-509835-26-00 6 countries 2022 n=121
    Ended

Phase I/II

1 trial
  • Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla Academic
    BIOTIN
    2023-508637-14-00 1 country 2024 n=24
    Recruiting

Phase I

1 trial
  • Alnylam Pharmaceuticals Inc. Commercial
    IMP not specified
    2024-515732-68-00 1 country 2025 n=20
    Recruiting

Phase I (First in Human)

2 trials
  • AskBio France Commercial
    ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE RH10 CONTAINING THE HUMAN CHOLESTEROL 24-HYDROXYLASE GENE
    2024-510895-18-00 1 country 2024 n=18
    Ended
  • uniQure biopharma B.V. Commercial
    IFEZUNTIRGENE INILPARVOVEC
    2024-511766-37-00 1 country 2021 n=10 Orphan
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Alnylam Pharmaceuticals Inc. Commercial 1 Phase I 1
F. Hoffmann-La Roche AG Commercial 1 Phase II 0
Medibiofarma S.L. Commercial 1 Phase II 0
Novartis Pharma AG Commercial 1 Phase II 0
PTC Therapeutics Inc. Commercial 1 Phase II 0
AskBio France Commercial 1 Phase I (First in Human) 0
uniQure biopharma B.V. Commercial 1 Phase I (First in Human) 0
Assistance Publique Hopitaux De Paris Academic 1 Phase III 1
Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla Academic 1 Phase I/II 1
New trials by year
2021
1
2022
1
2023
2
2024
4
2025
1
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