Bringing a Duchenne trial home – How far can you actually go?

By Dr Graham Wylie · Chair and Chief Executive, Medical Research Network · September 2026

Last week was International Duchenne Muscular Dystrophy Awareness Week. I wanted to write about something MRN has spent a great deal of time on: designing decentralised clinical trials in Duchenne muscular dystrophy (DMD). DMD is one of the busiest areas in rare-disease research, but it is also one of the most challenging environments in which to decentralise a trial effectively. Both of those realities are worth exploring.

A Busy Pipeline

There are currently around 40 products in clinical development for DMD—an extraordinary number for a rare disease. The two largest development classes are gene therapies and exon-skipping antisense oligonucleotides, alongside a spread of small molecules and cell therapies.

As a protocol designer, the detail I care about most isn’t the biological mechanism – it’s the route of administration.

Almost all of these therapeutics are delivered via needle. Gene therapies require a single infusion, while antisense drugs require weekly or bi-weekly infusions throughout the entire study duration. That single operational requirement – a recurring weekly injection – sets the rhythm of the entire trial. Whatever else you do, the patient must be dosed every single week.

Chasing a Small Population

Behind this pipeline lies an intense level of clinical activity. There are currently 86 ongoing interventional DMD trials listed on ClinicalTrials.gov:

• Recruiting: 33 studies
• Active, not recruiting: 25 studies
• Not yet recruiting: 18 studies
• Enrolling by invitation: 10 studies

A third of these trials are actively recruiting right now. They are competing for a highly specific, geographically scattered population of young patients.

This is precisely why your protocol design decisions matter so much before you lock the protocol. If you get the design wrong, you will spend the next two years facing a shortage of trial participants.

How We Approached Protocol Design

To build a realistic home-based trial model, you have to do the preliminary legwork. We developed four core reference frameworks:

1. Typical Study Structure: We analyzed 125 completed trials to map out the shape, length, and visit cadence of a standard Phase 3 DMD trial.
2. Assessment Portability: We evaluated every common DMD assessment to determine if a trained trial nurse can perform it in the patient’s home. We asked: Is it portable? Is the tool validated for home use? Are there country-specific regulatory limitations?
3. Patient Living Patterns: We examined how these boys live between trial visits. Across every major market, patients are cared for close to home and visit specialist centres only once every six months.
4. Geographic Patient Distribution: We mapped out patient population density versus the location of specialist centres across primary trial markets.

Building the Schedule

We applied these assessment verdicts to a model Phase 3 schedule featuring screening, baseline assessments, an intensive early phase, quarterly safety checks out to 52 weeks, and weekly injections.

Every visit’s setting is determined by its required assessments:

• Site Visits: Required if the visit involves complex imaging, specialized respiratory tests, or muscle biopsies.
• Home Visits: Utilized whenever site-anchored diagnostic equipment is not required.

Key Finding: Duchenne trials remain heavily anchored to site centres. Of twelve scheduled major assessment visits, nine must remain at the site due to specialised diagnostics and the necessity for direct healthcare professional contact during experimental drug monitoring. However, three key assessment visits – plus the vast majority of regular dosing visits – can transition to the home.

Why an 18% Reduction in Site Visits Drives Recruitment

For severe pediatric conditions, the physical and emotional toll of traveling to a centre of excellence is extreme. The distance is often vast, requiring entire families to travel.

When you transition regular parenteral drug administration to the home, over 80% of total actual trial touchpoints become home visits.

Average DMD Recruitment Rate (Published Data): 0.270 patients/site/month


With MRN Home Trial Model Integration: 0.408 patients/site/month (+51%)

Integrating home trial support increases recruitment velocity by over 50%, shortening recruitment timelines by approximately one-third.

For a typical trial of 150 patients across six countries, this translates to running 20 sites instead of 31. That means 11 fewer sites to identify, contract, train, and monitor – a massive operational gain in a patient population with limited specialist centres.

Global Patient Pools & Recruitment Strategy

Planning recruitment requires understanding national care networks, local referral pathways, and general practitioner involvement. While global incidence remains stable at approximately 1 in 3,500–5,000 live male births, prevalence and patient pool volumes differ significantly by country:

Note: Baseline morbidity and mortality differences must also be factored into trial design to ensure statistical models aren’t skewed when combining data across regions.

Activating the Infrastructure

Recruiting DMD patients requires leaning on established clinical networks, national registries, and advocacy organisations:

• Specialist Networks: FILNEMUS (France), DGM / EURO-NMD (Germany), NorthStar (UK), NEMO (Italy), CSUR (Spain), MDA Care Center Network (USA), NMD4C (Canada), and Muscular Dystrophy Clinical Trial Network (Japan).
• Global Registries & Advocacy: TREAT-NMD, The Duchenne Registry, Remudy, PPMD, AFM-Téléthon, Duchenne UK, Muscular Dystrophy UK, and CureDuchenne.

Strategic Advice: Avoid focusing exclusively on top-tier trial sites with the most research experience. These centres face intense competition for trial-eligible patients. Instead, target accredited network sites with fewer active competing trials. By pairing these secondary sites with a robust home trial model, you can activate patients over long distances without needing the local clinic to operate as a full-service trial site.

Key Takeaways

1. Dosing dictates design: Weekly parenteral drug administration sets the foundation for your operational framework.
2. Partial decentralisation is key: Specialised safety imaging, respiratory testing, and biopsies mean you cannot fully decentralise a DMD trial – and you don’t need to.
3. Small shifts yield big results: Moving weekly dosing and select visits to the home disproportionately boosts patient enrolment and retention.
4. Target low-competition sites: Focus site activation strategy on secondary network clinics with un-competed patient pools.
5. Optimise early: Decentralisation strategies must be embedded before locking the clinical protocol.

Optimize Your Duchenne Protocol

If you are currently designing a Phase 3 Duchenne trial, reach out to our team before locking your protocol. We will review your schedule of assessments visit-by-visit, map out exact home-versus-site allocations, and project the specific impacts on your site counts and recruitment timelines. Simply follow this link, fill out your contact information and we’ll get back to you immediately to schedule a time to discuss how we can support your trial.

Appendix 1: Phase 3 Schedule of Assessments Model

Legend: S = Screening | BL = Baseline | W = Week | X = Assessment Performed
Note: Interim weekly visits (Weeks 5, 7, 9–11, 13–23, 25–35, 37–47) are conducted at home for dosing and safety checks.

*Biopsy optional at interim points based on protocol.

Appendix 2: Duchenne Clinical & Support Infrastructure

• Clinical Networks: FILNEMUS (France), DGM / SPZ System (Germany), EURO-NMD (Europe), NorthStar & Adult North Star Network (UK), NEMO (Italy), CSUR (Spain), MDA Care Center Network & PPMD Certified Centers (USA), Canadian Pediatric Neuromuscular Group (Canada), Muscular Dystrophy Clinical Trial Network (Japan).
• Registries & Patient Advocacy: TREAT-NMD, The Duchenne Registry (USA), Canadian Neuromuscular Disease Registry, Remudy (Japan), PPMD, AFM-Téléthon, Duchenne UK, Muscular Dystrophy UK, CureDuchenne, World Duchenne Organisation.

Appendix 3: Sources

2018 DMD Care Considerations, Lancet Neurology (TREAT-NMD / MDA).
ClinicalTrials.gov — interventional DMD studies (retrieved 10 September 2026).
FDA prescribing information (Elevidys, Duvyzat, Agamree, Exondys 51, Vyondys 53, Amondys 45, Viltepso).
Sponsor press releases and conference presentations (MDA 2026, AAN 2026).
FILNEMUS (France); DGM / EURO-NMD and SPZ system (Germany); NorthStar and Adult North Star Network (UK).
Italian reference-centre readiness study (Acta Myologica); Spanish clinical-practice survey.
MDA Care Center network and PPMD Certified Duchenne Care Centers (USA).
Canadian paediatric neuromuscular physicians survey; Brazil unmet-needs study; Japanese Registry of Muscular Dystrophy (Remudy).
DMD epidemiology: global and European prevalence meta-analyses, German health-claims analysis, UK / Italian / Spanish national studies, and a nine-country prevalence model.
Specialist-centre counts: FILNEMUS, DGM, NorthStar, PPMD / MDA, Canadian Pediatric Neuromuscular Group and the Japanese Muscular Dystrophy Clinical Trial Network.
The Phase 3 schedule of events is an illustrative MRN template, not a specific sponsor protocol. Cadences and figures quoted are typical patterns; individual centres, protocols and patients vary with disease stage and complications. Retrieved 10–11 September 2026.