Search for a trial

Skeletal phenotyping of patients with Osteogenesis Imperfecta

- to compare the skeletal phenotypes of each OI-type obtained at the standard scan region (i.e. using the fixed offset distance) with a recently published sex- and site-specific normative dataset of a general adult population, obtained with the same…

A natural history study on infantile facioscapulohumeral muscular dystrophy: five-year follow-up

The primary objective of this study is to further discribe the natural course and clinical characteristic of infantile FSHD.The secondary objectives are: - To identify (epi)genetic and environmental disease modifying factors that contribute to…

Function and participation of children with Ponseti treated clubfoot

To investigate the differences in participation, motor abilities and function in more dynamic tasks between clubfoot patients with and without relapse and healthy controls.

A Phase 1, open-label, single-dose, single-center, mass balance study of IPN60130 following a single 60-mg oral dose of IPN60130 containing 9 µCi of [14C]-IPN60130 in healthy male participants

In this study we will investigate how quickly and to what extent the new compound IPN60130 is absorbed, transported, and eliminated from the body (this is called pharmacokinetics). IPN60130 is radioactively labelled with carbon-14 (14C). In this way…

Follow You intervention study: Effectiveness of a multidisciplinary training program on daily physical functioning in children and young adolescents with a hereditary connective tissue disorder: a feasibility study

1) Improving the (physical) daily functioning of children with HCTD2) Testing feasibility of intervention for child / parents and therapists

A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants

This study has been transitioned to CTIS with ID 2024-511469-13-00 check the CTIS register for the current data. Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and…

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with Duchenne Muscular Dystrophy (DMD)

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).