NATIVE (Nash Trial to validate IVA337 Efficacy) Phase IIb trial
FASST (For A Systemic Sclerosis Treatment) Phase IIb trial
Systemic sclerosis (SSc) is a complex, multiorgan, rare disease, affecting the immune system, the microvascular system and the connective tissue. This disease involves mostly the skin but also lung, heart, gastrointestinal tract and kidneys. Organ progressive failures make SSc a severe and lethal disease with a high death toll.
The FASST study is designed to compare for 48 weeks two doses of Lanifibranor with a placebo control level over 48 weeks. The study is currently recruiting in 8 European countries and more than 50 centers have been selected.
The primary end point will be the mean change of the MRSS (Modified Rodnan Skin score) from baseline to 48 weeks.
Further information on the FASST clinical trial is available on https://clinicaltrials.gov/ with the identifier NCT02503644. Please also watch the video of Professor Yannick Allanore performing the MRSS score by visiting www.fassttrial.com
IMPROVES (IMPROVE MPS TREATMENT) PROOF OF CONCEPT PHASE IIa TRIAL
Mucopolysaccharidoses (MPS) are a group of rare genetic disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs) or mucopolysaccharides.
Odiparcil (IVA336) is a small molecule with the potential to become the first oral substrate reduction therapy for MPS I, II and VI.
A 26 week, double blinded, randomized, placebo controlled phase IIa clinical study to investigate the safety, pharmacokinetics and efficacy of oral administration of odiparcil in adult patients with MPS type VI receiving enzyme replacement therapy (ERT) is currently ongoing. The study also has an open label arm with patients currently not on ERT. The iMProveS trial open and recruiting patients.