A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) - (RESTORE)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/41854
• EU PAS number:
  EUPAS41853
• Study ID:
  41854
• Official title and acronym:
  A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) - (RESTORE)
• DARWIN EU® study:
  No
• Study countries:
  Argentina

  Chile

  Greece

  Ireland

  Japan

  Korea, Democratic People's Republic of

  Poland

  Portugal

  Romania

  Russian Federation

  Taiwan

  United States
• Study description:
  This prospective observational registry will assess long-term outcomes of patients with a diagnosis of SMA, including long term safety and effectiveness of OAV-101.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Novartis Pharmaceuticals

Networks

iSMAC, French SMA Registry, SMArtCARE, CuidAME, Cure SMA

Study timelines

Date when funding contract was signed

Planned: 01/10/2017

Actual: 12/01/2018

Study start date

Planned: 25/09/2018

Actual: 25/09/2018

Data analysis start date

Planned: 25/09/2018

Actual: 31/12/2019

Date of interim report, if expected

Planned: 12/12/2019

Actual: 02/07/2020

Date of final study report

Planned: 28/10/2038

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Novartis

Study protocol

Initial protocol

PRO-1300 RESTORE AVXS-101-RG-001_Redacted

Updated protocol

PMA1234_RESTORE Protocol Amendment 3.0_V4.0 final_Redacted.pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)
• Regulatory procedure number:
  AVXS-101-RG-001

Other study registration identification numbers and links

AVXS-101-RG-001, NCT04174157, COAV101A12001

Link to ClinicalTrials.gov