Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 8 trials

Late-Stage Pipeline

13%

1 trials in Phase 3/4

Results Transparency

0%

2 of 0 completed trials have results

Key Signals

4 recruiting2 with results

Enrollment reporting

Analytics

Phase 2
5(62.5%)
Phase 1
1(12.5%)
Phase 2/3
1(12.5%)
Phase 3
1(12.5%)
8Total
Phase 2(5)
Phase 1(1)
Phase 2/3(1)
Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (8)

Showing 8 of 8 trials
NCT04987463Phase 2/3Recruiting

Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

Role: lead

NCT05534672Phase 3Recruiting

Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex

Role: lead

NCT07865130Phase 1Not Yet Recruiting

Study of CDK8 Inhibitor RVU120 in Combination With Everolimus in Children With Recurrent or Progressive Group 3 or 4 Medulloblastoma

Role: lead

NCT04930627Phase 2Unknown

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

Role: lead

SYF1fba4875855740e1af1c070071d7de78Phase 2Recruiting

Study of the effectiveness of drugs selected depending on the molecular profile of diffuse intrinsic pontine glioma (DIPG).

Role: lead

SYF76948e9de8554a309339af37d8b84820Phase 2Recruiting

This is a multicenter, double-blind, randomized study to compare the safety, effectiveness and immunogenicity (level and body resistance to drugs) of two biological drugs administered intravenously - vedolizumab (a drug not registered in pediatric patients) and infliximab (used as standard therapy) in pediatric patients with ulcerative colitis aged 6-17 years.

Role: lead

SYFd1f1a8639f334b0f870678c90294ebaaPhase 2Unknown

Open-label study to assess safety and efficacy of rapamycin in drugresistant epilepsy associated with mTOR pathway pathologies

Role: lead

SYFe50816e98ecf4c509a95dca2ca29b99ePhase 2Unknown

Clinical study to assess safety and efficacy of rapamycin in paediatric high grade glioma

Role: lead

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