Lead Investigator: Andrew Strahs, Wave Life Sciences
Title of Proposal Research: Analysis of PRO-DMD-01 Functional Data to Inform Endpoints in Wave’s FORWARD-53 Study of WVE-N531 for Duchenne Muscular Dystrophy (DMD) Exon-53 Skipping Therapy
Vivli Data Request: 11038
Funding Source: I am an employee of Wave Life Sciences. Wave Life Sciences is the sponsor of FORWARD-53 and will also fund the proposed analyses.
Potential Conflicts of Interest: I lead the Biometrics team (Biostatistics, Statistical Programming, Data Management) at Wave Life Sciences, based in Cambridge, Massachusetts. I am an employee of Wave and receive salary, bonus and equity awards from Wave. This will be mitigated by Wave outsourcing the conduct of the analysis to James Signorovitch’s team at Analysis Group. (There is a fully executed contract in place.) Also, my employment with Wave will be disclosed in subsequent publications. James Signorovitch is an employee of Analysis Group, a research consultancy which will receive funding from Wave Life Sciences for conducting this research. Funding and employment will be disclosed in subsequent publications. Dr. Tai is an employee of Wave Life Sciences and receives salary, bonus and equity from Wave. This will be mitigated by outsourcing the analysis to James Signorovitch’s team at Analysis Group. Employment will be disclosed in publications of this work. Jessica Marden is an employee of Analysis Group, a research consultancy which will receive funding from Wave Life Sciences for conducting this research. Funding and employment will be disclosed in subsequent publications. Catherine Fernan is an employee of Analysis Group, a research consultancy which will receive funding from Wave Life Sciences for conducting this research. Funding and employment will be disclosed in subsequent publications. Mark is an employee of Wave Life Sciences and receives salary, bonus and equity from the company. Mark’s employment will be disclosed in any publication on which he is an author. This work in this research proposal is being outsourced to Analysis Group.
Summary of the Proposed Research:
Duchenne muscular dystrophy (DMD) is a rare, genetic disorder that causes progressive muscle weakness and loss of function. DMD is caused by mutations in the dystrophin gene, which leads to the absence of a protein essential for muscle health. Approximately 1 in 5,000 males worldwide are born with DMD. It significantly impacts quality of life and life expectancy, with most patients requiring a wheelchair by their early teens and experiencing severe complications later in life.
There is no cure for DMD, but therapies targeting specific mutations in the dystrophin gene, such as exon skipping therapies, show promise in slowing disease progression. Exon skipping therapies are treatments that help the body produce a working version of a protein by skipping over the faulty part of a gene, and aim to restore production of a shorter, yet partially functional, form of the dystrophin protein. WVE-N531 is an exon skipping treatment being developed for patients with DMD who have mutations treatable by exon 53 skipping.
The purpose of this research is to analyze data from the PRO-DMD-01 study. The results from this research will be used as an external control to compare against the corresponding data collected in the FORWARD-53 study sponsored by Wave Life Sciences. FORWARD-53 is a small (N=11), single-arm study of WVE-N531. Wave plans to use the results generated under this research plan for PRO-DMD-01 to assess activity of WVE-N531 in FORWARD-53.
To conduct this research, we will analyze functional outcomes data, such as measures of muscle strength and mobility.
Requested Studies:
A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
Data Contributor: Cure Duchenne
Study ID: NCT01753804
Sponsor ID: PRO-DMD-01