Tuberous Sclerosis Complex

Rare Diseases
3
Pipeline Programs
3
Companies
5
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Jazz Pharmaceuticals
Jazz PharmaceuticalsCA - Palo Alto
2 programs
2
GWP42003-PPhase 31 trial
GWP42003-PPhase 31 trial
Active Trials
NCT02544763Completed224Est. Feb 2019
NCT02544750Completed199Est. Jun 2021
Alliance Pharmaceuticals
2 programs
1
RAD001Phase 21 trial
Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)N/A1 trial
Active Trials
NCT01780441Completed166Est. Dec 2020
NCT01289912Completed52Est. Dec 2014
Genomics
GenomicsUK - Oxford
1 program
Studies in Patients With Tuberous Sclerosis ComplexN/A1 trial
Active Trials
NCT03276195Completed32Est. Nov 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Jazz PharmaceuticalsGWP42003-P
Jazz PharmaceuticalsGWP42003-P
Alliance PharmaceuticalsRAD001
GenomicsStudies in Patients With Tuberous Sclerosis Complex
Alliance PharmaceuticalsEarly Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Clinical Trials (5)

Total enrollment: 673 patients across 5 trials

NCT02544750Jazz PharmaceuticalsGWP42003-P

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Start: Aug 2016Est. completion: Jun 2021199 patients
Phase 3Completed
NCT02544763Jazz PharmaceuticalsGWP42003-P

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Start: Apr 2016Est. completion: Feb 2019224 patients
Phase 3Completed
NCT01289912Alliance PharmaceuticalsRAD001

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

Start: Jan 2011Est. completion: Dec 201452 patients
Phase 2Completed
NCT03276195GenomicsStudies in Patients With Tuberous Sclerosis Complex

Studies in Patients With Tuberous Sclerosis Complex

Start: May 2016Est. completion: Nov 202332 patients
N/ACompleted
NCT01780441Alliance PharmaceuticalsEarly Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Start: Jan 2013Est. completion: Dec 2020166 patients
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space

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