Pelizaeus-Merzbacher disease

2 trials 2 recruiting MedDRA PT
2 Registered trials
2 Recruiting
2 Unique sponsors
2 Unique IMPs
1 Commercial trials
Drug classes (ATC)
— ATC code not specified 1
V Various (ATC V) 1
Mechanism of action (INN-stem analysis)
Unknown / small molecule 2 substances
Phase II: 1 Phase I: 1

Filter these 2 trials by country, sponsor, date range and more.

Open in Search → Gap analysis →
Show: 2 trials

Phase II

1 trial
  • Amsterdam UMC Stichting Academic
    DEFERIPRONE
    2024-511968-81-00 1 country 2024 n=10
    Recruiting

Phase I

1 trial
  • Ionis Pharmaceuticals Inc. Commercial
    2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING PLP1 PRE-MRNA
    2022-502432-39-00 3 countries 2024 n=33
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Ionis Pharmaceuticals Inc. Commercial 1 Phase I 1
Amsterdam UMC Stichting Academic 1 Phase II 1
New trials by year
2024
2
← All indication landscapes