Registry enquiries

  • Rebecca Leary
    Rebecca Leary
    Becca Leary is a member of the TREAT-NMD Secritariat and responsible for the Global SMA...

The TREAT-NMD Global Registries are recognised as a leading source for trial planning and recruitment in these diseases at an international level. In addition, these registries can help researchers to answer questions about global disease prevelance. The TREAT-NMD Global Registries also support other activities to improve patient care, such as the assessment of care standards in different countries.

There have been over 20 enquires to the TREAT-NMD Global Registries so far. These have included bothe DMD and SMA enquiries Consisting of feasibility enquiries for clinical programmes looking at exon skipping and nonsense mutation as well as recruitment for clinical trials looking at exon skipping and a multinational study focusing on health economics. All of this is with the aim of ensuring that promising new treatments can be brought to patients as quickly as possible.

Pharmaceutical companies interested in locating patients for a clinical trial are able to request information from the TREAT-NMD Global Registries in a secure fashion that means that patients' details are never disclosed directly to the company, but companies can speedily find the information they need.

All national registries adhere to a core data set, this was chosen with clinical trial readiness in mind.

DMD dataset

SMA dataset

The types of enquiries that are usually received by the TREAT-NMD Global Registries are:

  • Feasibility enquiries – general data on patient numbers within particular countries (or worldwide), that meet certain criteria, such as age range, ambulation status, etc. Usually requested in the planning phases of a clinical trial.
  • Assistance with recruitment into a clinical trial – patients can be contacted through their national registry if they meet the clinical trial inclusion criteria and are then asked to contact their local trial site. Patients not seen at a particular site, and who wouldn't otherwise be known to staff at that site, can also be informed about the trial.

The process for requesting data from the TREAT-NMD Global Registries is outlined below:
Enquiry flow chart

07 Sep 2018