Study identification

PURI

https://redirect.ema.europa.eu/resource/36194

EU PAS number

EUPAS3247

Study ID

36194

Official title and acronym

An international disease registry collecting data on manifestations, interventions and outcomes in patients with tuberous sclerosis complex - TOSCA

DARWIN EU® study

No

Study countries

Australia
Austria
Belgium
China
Denmark
Estonia
France
Germany
Greece
Israel
Italy
Japan
Korea, Republic of
Latvia
Lithuania
Netherlands
Norway
Poland
Portugal
Romania
Russian Federation
Slovakia
Slovenia
South Africa
Spain
Sweden
Taiwan
Thailand
Türkiye
United Kingdom

Study description

Tuberous Sclerosis Complex (TSC) is an autosomal dominant genetic disorder caused by inactivating mutations in the TSC1 or TSC2 genes. Lesions in TSC patients can occur in the brain, kidneys, heart, liver, lungs and skin, and can manifest with renal and or pulmonary complications, autism, mental retardation and epilepsy.There is common agreement that there are still gaps in understanding the course of TSC manifestations and their prognostic role, rare symptoms and co-morbidities, interventions, treatments and their outcomes, and quality of life. The registry will address many of these gaps by collecting data from patients across many countries worldwide. The data collected might influence and improve patient’s treatment standards and flows. An additional purpose of this registry is to inform the research in TSC. Data on the long-term safety of the prescribed treatment with Votubia® (everolimus) in the licensed indications will be collected in a TOSCA safety sub-study.

Study status

Finalised
Research institution and networks

Institutions

Multiple centres: 250 centres are involved in the study

Contact details

Novartis Clinical Disclosure Officer

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Novartis Pharmaceuticals
Study protocol
Initial protocol

CRAD001MIC03_protocol_v03_30Jul2014_Redacted

English (4.93 MB - PDF)View document
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

Non-EU RMP only