Facioscapulohumeral Muscular Dystrophy (FSHD)Phase 2NCT07872306
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 study testing forazapadin in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Satellos Bioscience, Inc..
Trial summary
This is a two-part, Phase 2, randomized, placebo-controlled, double-blind study, followed by a randomized active treatment period, to evaluate the safety and efficacy of orally administered forazapadin (SAT-3247) in adult participants with FSHD Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- What is being tested?
- forazapadin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 51 participants
- Where
- No sites listed yet
- Study dates
- 15 Dec 2026to 30 Jun 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- definitive diagnosis of FSHD1 or FSHD2, confirmed by clinical findings and prior genetic testing results
- presence of weakness in upper limbs at Screening, defined as a Brooke score ≥ 2
- presence of weakness in a lower limb at Screening, defined as a MRC score of ≤4 on ankle dorsiflexion
- ambulatory at Screening
- if taking herbal remedies and supplements which can impact muscle strength and function, must maintain a stable regimen for the duration of the trial
- if taking prescription medicines, over-the-counter medicines and/or herbal supplements for supportive care, doses should be stable for ≥ 1 month prior to the Screening Visit and remain stable for the duration of the trial
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- if on a physical therapy/strength training regimen, the regimen must be stable for ≥ 2 months prior to the Screening Visit and remain stable for the duration of the trial
- ability to provide written informed consent
- all participants, if sexually active, agree to follow the contraception requirements and sperm donation limitations
Who cannot join
- ambulatory participants expected to experience loss of ambulation within ≤ 12 months
- medical condition, other than FSHD, that would impact ambulation or ability to perform the functional assessments
- presence of acute or chronic illness, or history of chronic illness (other than FSHD)
- prior or ongoing medical condition, medical history, physical examination findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant
- evidence of renal impairment at Screening, including renal impairment demonstrated by cystatin C-based estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m², a history of clinically significant renal disease, or current acute kidney injury or acute kidney injury
- evidence of significant hepatic dysfunction, defined as glutamate dehydrogenase (GLDH) >2× upper limit of normal (ULN) at the Screening Visit, or known hepatic impairment classified as Child-Pugh Class B (moderate) or Child-Pugh Class C (severe)
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- history of seizures or seizure-like events
- surgery (e.g., stomach bypass) or medical condition that might affect absorption of medicines
- acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea, heartburn) or acute infection (such as influenza) or a significant infection or known inflammatory process at Screening
- presence or history of severe adverse reaction to any excipients in the study drug tablets
- treatment with another investigational drug or biological agent within 30 days or 5 half-lives of the investigational drug or biological agent
- received SAT-3247 in another study
- positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibody or Human Immunodeficiency Virus (HIV)
- current or expected use during the study of any medications that are known to be strong or moderate inhibitors of Cytochrome P450 isoform 3A4 (CYP3A4)
- treatment with a glucagon-like peptide-1 (GLP-1) receptor agonist or dual glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 receptor agonist within 3 months prior to Screening
- consumption of grapefruit, grapefruit juice, and grapefruit-containing products
- employee, contractors, or consultants of the Sponsor, the contract research organization (CRO), and/or study site or their relatives
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study Followed by an Active Treatment Period to Evaluate the Safety, Tolerability, and Efficacy of SAT-3247 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
This is a two-part, Phase 2, randomized, placebo-controlled, double-blind study, followed by a randomized active treatment period, to evaluate the safety and efficacy of orally administered forazapadin (SAT-3247) in adult participants with FSHD
- Being tested
- forazapadin, Placebo Comparator
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
forazapadinDrug
Forazapadin (SAT 3247) is a potent, orally available, muscle penetrant, small molecule inhibitor of AAK1
Placebo ComparatorDrug
Matching placebo oral tablets
| Study group | Role | What they receive |
|---|---|---|
| forazapadin (SAT-3247) 60 mg | Active comparator | Not described Receives: Drug: forazapadin; Drug: Placebo Comparator |
| forazapadin (SAT-3247) 120 mg | Active comparator | Not described Receives: Drug: forazapadin; Drug: Placebo Comparator |
| placebo | Placebo comparator | Not described Receives: Drug: Placebo Comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety evaluation of forazapadin in FSHD
Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)
Measured: 48 weeks
Efficacy of forazapadin as treatment in FSDH
Change in muscle force measurements as determined by dynamometry
Measured: 24 and 48 weeks
Secondary outcomes (3)
Effects of forazapadin on muscle MRI
Whole-body MRI will be utilized as a non-invasive, quantitative imaging modality to assess skeletal muscle composition across participants. MRI will be used to measure muscle fat fraction (MFF) and characterize the extent of muscle fat infiltration (MFI)
Measured: 24 and 48 weeks
Pharmacokinetic (PK) profile
Determine the pharmacokinetic (PK) profile of SAT-3247 in participants with FSHD calculating maximum concentration (Cmax)
Measured: 24 weeks
Pharmacokinetic (PK) profile
Determine the pharmacokinetic (PK) profile of SAT-3247 in participants with FSHD calculating time to maximum concentration (Tmax)
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2026
- Study start15 Dec 2026 (estimated)
- Primary completion30 Dec 2028 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facioscapulohumeral Muscular Dystrophy (FSHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 51 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Satellos Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07872306
- Sponsor's protocol ID
- SAT-3247-CL-211
- Status last verified
- Sep 2026
- First submitted
- 22 Sep 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study Followed by an Active Treatment Period to Evaluate the Safety, Tolerability, and Efficacy of SAT-3247 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
- ClinicalTrials.gov ID
- NCT07872306
- Acronym / sponsor ID
- SAT-3247-CL-211
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 51 participants (estimated)
- Lead sponsor
- Satellos Bioscience, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Dec 2026 (estimated)
- Primary completion
- 30 Dec 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 13 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a two-part, Phase 2, randomized, placebo-controlled, double-blind study, followed by a randomized active treatment period, to evaluate the safety and efficacy of orally administered forazapadin (SAT-3247) in adult participants with FSHD
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| forazapadin (SAT-3247) 60 mg | Active comparator | Drug: forazapadin, Drug: Placebo Comparator |
| forazapadin (SAT-3247) 120 mg | Active comparator | Drug: forazapadin, Drug: Placebo Comparator |
| placebo | Placebo comparator | Drug: Placebo Comparator |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- definitive diagnosis of FSHD1 or FSHD2, confirmed by clinical findings and prior genetic testing results
- presence of weakness in upper limbs at Screening, defined as a Brooke score ≥ 2
- presence of weakness in a lower limb at Screening, defined as a MRC score of ≤4 on ankle dorsiflexion
- ambulatory at Screening
- if taking herbal remedies and supplements which can impact muscle strength and function, must maintain a stable regimen for the duration of the trial
Key exclusion criteria (17 listed)
- ambulatory participants expected to experience loss of ambulation within ≤ 12 months
- medical condition, other than FSHD, that would impact ambulation or ability to perform the functional assessments
- presence of acute or chronic illness, or history of chronic illness (other than FSHD)
- prior or ongoing medical condition, medical history, physical examination findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant
- evidence of renal impairment at Screening, including renal impairment demonstrated by cystatin C-based estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m², a history of clinically significant renal disease, or current acute kidney injury or acute kidney injury
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| forazapadinForazapadin (SAT 3247) is a potent, orally available, muscle penetrant, small molecule inhibitor of AAK1 | Drug | forazapadin (SAT-3247) 120 mg, forazapadin (SAT-3247) 60 mg |
| Placebo ComparatorMatching placebo oral tablets | Drug | forazapadin (SAT-3247) 120 mg, forazapadin (SAT-3247) 60 mg, placebo |
Primary outcome
Safety evaluation of forazapadin in FSHD
Time frame: 48 weeks
Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)
Efficacy of forazapadin as treatment in FSDH
Time frame: 24 and 48 weeks
Change in muscle force measurements as determined by dynamometry
3 secondary outcomes
Effects of forazapadin on muscle MRI
Time frame: 24 and 48 weeks
Pharmacokinetic (PK) profile
Time frame: 24 weeks
Pharmacokinetic (PK) profile
Time frame: 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing forazapadin in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Satellos Bioscience, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Satellos Medical Information (+1 647-660-1780, medicalinfo@satellos.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety evaluation of forazapadin in FSHD (time frame: 48 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.