Clinical trials: Functional Decline
- Condition Functional Decline
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Primary Immunoglobulin A Nephropathy (IgAN)
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN
The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A…
- Trial name
- NCT06994845
- Testing
- iptacopan
- Where
- 20 sites in United States, Argentina and 6 more
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Asthma
A Study of Efficacy and Safety of Depemokimab Compared With Placebo in Adults and Adolescents With at Risk Type 2 Asthma
The aim of this study is to evaluate the efficacy of depemokimab administered as an adjunctive therapy, in participants with Type 2 asthma at risk of exacerbations compared to the guideline recommended standard of care (SoC).
- Trial name
- MODIFY (NCT07456033)
- Testing
- Depemokimab
- Where
- 17 sites in United States, United Kingdom
- Volunteers
- 456 people
- Sponsor
- GlaxoSmithKline