A European observational registry collecting efficacy and safety data in newly diagnosed pediatric Philadelphia-positive (Ph+) Acute Lymphoblastic Leukemia (ALL) patients treated with chemotherapy + imatinib ± hematopoietic stem cell treatment (±HSCT)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/39519
• EU PAS number:
  EUPAS6665
• Study ID:
  39519
• Official title and acronym:
  A European observational registry collecting efficacy and safety data in newly diagnosed pediatric Philadelphia-positive (Ph+) Acute Lymphoblastic Leukemia (ALL) patients treated with chemotherapy + imatinib ± hematopoietic stem cell treatment (±HSCT)
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Greece

  Hungary

  Italy

  Poland

  Portugal

  Romania

  Russian Federation

  Slovakia

  Slovenia

  Spain

  Ukraine
• Study description:
  This study was an observational, multi-center disease registry to collect efficacy and safety data in Ph+ ALL pediatric patients treated with chemotherapy + imatinib, with or without hematopoietic stem cell treatment (±HSCT), primarily in European countries. The study concept was endorsed by the EMA as part of the post-marketing commitment (EMEA/H/C/000406/II/80) and the design was aligned with ensuing discussions, as well as routine medical practice in the treatment of newly diagnosed Ph+ ALL in pediatric patients across Europe. This was an EMA imposed non-interventional post-authorization safety study (NI-PASS), conducted per GVP module VIII. For each patient enrolled in the registry, a minimum of 5 years observational follow-up data was collected or such available data until early discontinuation, as measured from the date of diagnosis.
• Study status:
  Finalised

Research institutions and networks

Institutions

Novartis Pharmaceuticals

Multiple centres: 33 centres are involved in the study

Study timelines

Date when funding contract was signed

Planned: 20/12/2013

Actual: 20/12/2013

Study start date

Planned: 30/06/2014

Actual: 14/07/2014

Data analysis start date

Actual: 01/09/2022

Date of final study report

Planned: 20/02/2023

Actual: 02/02/2023

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Novartis Pharma AG

Study protocol

Initial protocol

STI571I2201-v02--protocol_Redacted

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)

Other study registration identification numbers and links

CSTI571I2201