SAT-3247 for Duchenne Muscular Dystrophy

(BASECAMP Trial)

Not currently recruiting at 17 trial locations

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: Satellos Bioscience, Inc.

Must be taking: Glucocorticoids

Disqualifiers: Loss of ambulation, Hepatic dysfunction, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of SAT-3247, a new oral medication for treating Duchenne Muscular Dystrophy (DMD), a condition that weakens muscles over time. Researchers compare two different doses of SAT-3247 against a placebo (a pill with no active medication) over 12 weeks to determine which dose is most effective and safe for patients. Boys aged 7 to 10 years with a confirmed DMD diagnosis who can still walk and have been on a stable treatment regimen might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important early findings.

Do I need to stop my current medications to join the trial?

You can continue taking your current medications if they are stable doses of prescription medicines, including glucocorticoids, ACE inhibitors, β-blockers, and diuretics, as well as over-the-counter medicines and herbal supplements. However, you should not start or stop any medications without consulting the trial team.

Is there any evidence suggesting that SAT-3247 is likely to be safe for humans?

Research has shown that SAT-3247 is generally safe based on earlier trials. In one study involving adults with Duchenne Muscular Dystrophy (DMD), participants who took SAT-3247 experienced only mild side effects over 28 days. These side effects were not serious and did not lead to discontinuation of the treatment. The study also noted an improvement in grip strength, which is a positive outcome.

SAT-3247 was initially tested in healthy adults and adults with DMD before being studied in children. This initial testing ensures SAT-3247's safety. However, as the current trial is in the early stages of testing in children, ongoing studies will continue to provide more information about its safety and effectiveness in this younger group. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Researchers are excited about SAT-3247 for Duchenne Muscular Dystrophy because it offers a new approach compared to standard treatments like corticosteroids. Most current treatments focus on managing symptoms, but SAT-3247 works by potentially targeting the underlying cause of muscle degeneration. This treatment is delivered as an oral tablet and comes in two dosages: 60 mg and 120 mg, with the higher dose being studied internationally. This innovative approach could provide new hope for patients by possibly slowing down disease progression more effectively.

What evidence suggests that SAT-3247 might be an effective treatment for Duchenne Muscular Dystrophy?

Research shows that SAT-3247 has promising results for treating Duchenne Muscular Dystrophy (DMD). In earlier studies, patients who took SAT-3247 for 28 days experienced a significant increase in grip strength. Specifically, the strength of their dominant hand improved by 118.6%, and their nondominant hand improved by 97.9%. These improvements surpass typical progression in DMD. This trial will evaluate SAT-3247 at different dosages, including 60 mg and 120 mg, to further assess its potential to improve muscle function in people with DMD. ^{1 2 4 6}

Who Is on the Research Team?

Satellos Chief Medical Officer

Principal Investigator

Satellos Bioscience, Inc.

Are You a Good Fit for This Trial?

This trial is for boys aged 7 to less than 10 years with Duchenne Muscular Dystrophy (DMD) who can walk. They must be able to follow the study procedures and have a caregiver who agrees to help them comply.

Inclusion Criteria

I took Elevidys over 18 months ago, and my muscle function has been stable or worsened for at least 3 months.

Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit

I took an exon skipper over 6 months ago and my muscle function has been stable or declining for at least 3 months.

Exclusion Criteria

Consumption of grapefruit juice or grapefruit containing products

Participants for whom MRI or open muscle biopsy are contraindicated

Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Treatment

Participants receive SAT-3247 or placebo daily for 12 weeks to assess dose, safety, tolerability, and efficacy

12 weeks

Baseline visit, follow-up phone call at Week 1, visits at Week 4, Week 8, and Week 12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SAT-3247

Trial Overview

The trial tests two doses of SAT-3247 against a placebo in young boys with DMD. It's randomized, meaning patients are put into groups by chance, double-blind so neither doctors nor patients know who gets what, and controlled by using a placebo.

How Is the Trial Designed?

Group I: SAT-3247 60 mg Active Control 1 Intervention

SAT-3247 60 mg oral tablets administered daily for 12 weeks

Group II: SAT-3247 120 mg Active Control 1 Intervention

SAT-3247 120 mg oral tablets administered daily for 12 weeks; note the 120 mg dose will not be studied in the US and Canada

Group III: placebo Placebo Group 1 Intervention

placebo oral tablets administered daily for 12 weeks

Who Is Running the Clinical Trial?

Satellos Bioscience, Inc.

Lead Sponsor

Trials

2

Recruited

90+

Citations

1.

ir.satellos.com

ir.satellos.com/news/news-details/2025/Satellos-Announces-New-Data-Further-Demonstrating-Safety-Tolerability-and-Functional-Impact-of-SAT-3247-in-First-in-Human-Trial-of-Adults-with-Duchenne-Muscular-Dystrophy/default.aspx

News Details

Individuals treated with SAT-3247 over a 28-day period demonstrated an increase in grip strength far greater than seen in the Duchenne natural ...

2.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/satellos-data-sat3247-duchenne-trial/

Satellos reports safety data of SAT-3247 in Duchenne ...

Subjects receiving SAT-3247 over the 28-day period exhibited a notable improvement in grip strength that surpassed the natural progression ...

3.

defeatduchenne.ca

defeatduchenne.ca/satellos-new-data-on-safety-and-effectiveness-of-sat-3247/

Satellos: New Data on Safety and Effectiveness of SAT-3247

Satellos: New Data on Safety and Effectiveness of SAT-3247 · 118.6% increase in dominant hand gripping strength · 97.9% increase in nondominant ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06867107

NCT06867107 | A Long-term Follow-up Study for ...

This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD that previously participated in SAT-3247-CL-101.

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06565208

First in Human SAD/MAD Safety and PK Study With Adult ...

This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy volunteers (HVs) and adult participants with DMD.

6.

firstwordpharma.com

firstwordpharma.com/story/6736861

Satellos Receives Clearance by U.S. FDA and Global ...

Placebo-controlled Phase 2 study will evaluate SAT-3247 treatment over three months among ambulatory children with Duchenne