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Found 62 clinical trials
Study to Assess the Efficacy and Safety of 2 Dosage Regimens of Oral IPN60130 for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). (FALKON)

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility …

  • 0 views
  • 23 Apr, 2022
  • 7 locations
JAK 1/2 Inhibitor, Baricitinib, in the Treatment of Adult IIM

This study aims to investigate the clinical efficacy of baricitinib in patients with adult idiopathic inflammatory myositis (IIM). Half of the patients enrolled onto the study will receive 24

weakness
muscle enzyme
rash
hydroxychloroquine
methotrexate
  • 0 views
  • 15 Dec, 2021
  • 1 location
A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies. (PIVOINE)

The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP. The aim of the study is also to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study …

  • 0 views
  • 15 May, 2022
  • 3 locations
To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (Progress)

This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

  • 0 views
  • 15 May, 2022
  • 1 location
Saracatinib Trial TO Prevent FOP (STOPFOP)

This is a phase 2 study, designed as a European multicentre 6-month double blind random-ized controlled trial (RCT) of AZD0530 versus matched placebo, followed by a 12 month trial comparing open-label extended AZD0530 treatment with historical control data. Study population: Male and female adult patients aged 18 years and older …

  • 0 views
  • 10 May, 2022
  • 1 location
Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)

This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the feasibility and initial efficacy of Gamunex-C IVIG mono-therapy in HMGCR necrotizing myopathy.

  • 0 views
  • 24 Mar, 2022
  • 1 location
Intraorbital Injection Versus Oral Steroid in Anterior Idiopathic Orbital Inflammation

A multicenter randomised double-arm clinical trial, to compare safety and efficacy of oral versus intralesional injection of steroid in a group of patients suffering from idiopathic orbital inflammation is designed. Outcome measures include number of recurrences, duration of remission, and side effects.

  • 6 views
  • 08 Jul, 2021
  • 1 location
Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)

GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), …

  • 0 views
  • 14 May, 2022
  • 2 locations
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Adults With Dermatomyositis (DM) (RECLAIIM)

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of IgPro20 (subcutaneous Ig) treatment in adult subjects with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of IgPro20 subcutaneous (SC) doses in comparison to placebo in adult subjects with DM, as measured by responder …

prednisolone
rash
dermatomyositis
rheumatism
corticosteroids
  • 35 views
  • 17 Feb, 2022
  • 59 locations
Abatacept for the Treatment of Myositis-associated Interstitial Lung Disease (ATtackMy-ILD)

A randomized, controlled pilot trial to evaluate the efficacy and safety of subcutaneous Abatacept in treating interstitial lung disease associated with the anti-synthetase syndrome.

immunosuppression
anakinra
igiv
myositis
tocilizumab
  • 309 views
  • 13 May, 2022
  • 5 locations