NCT07798609

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of SFL-0821 in adult patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
90

participants targeted

Target at P75+ for phase_1

Timeline
18mo left

Started Sep 2026

Geographic Reach
2 countries

6 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress6%
Sep 2026Apr 2028

First Submitted

Initial submission to the registry

August 17, 2026

Completed
15 days until next milestone

First Posted

Study publicly available on registry

September 1, 2026

Completed
Same day until next milestone

Study Start

First participant enrolled

September 1, 2026

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2028

Expected
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2028

Last Updated

September 29, 2026

Status Verified

September 1, 2026

Enrollment Period

1.5 years

First QC Date

August 17, 2026

Last Update Submit

September 26, 2026

Conditions

Facioscapulohumeral Muscular Dystrophy (FSHD)

Outcome Measures

Primary Outcomes (1)

  • Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.

    Part A: Up to Week 24; Part B: Up to Week 48

Secondary Outcomes (10)

  • Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)

    Part A and B: through 24 hours post dose (all cohorts)

  • Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])

    Part A and B: through 24 hours post dose (all cohorts)

  • Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)

    Part A and B: through 24 hours post dose (all cohorts)

  • DUX4-regulated gene expression in skeletal muscle

    Part A: Up to Week 24; Part B: Up to Week 48

  • Incidence of anti-drug antibodies (ADAs), and if positive, of neutralizing ADAs

    Part A: Up to Week 24; Part B: Up to Week 48

  • +5 more secondary outcomes

Study Arms (2)

SFL-0821

EXPERIMENTAL

SFL-0821 for Injection

Drug: SFL-0821 for injection

Placebo

PLACEBO COMPARATOR

Placebo for Injection

Drug: Placebo

Interventions

SFL-0821 for injectionDRUG

single or multiple doses of SFL-0821 by intravenous (IV) infusion

SFL-0821
PlaceboDRUG

calculated volume to match active treatment by IV infusion

Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Genetically confirmed diagnosis of FSHD 1 or 2
  • FSHD Clinical Severity Score ≥1.5 to ≤4 at screening (5-point Ricci scale)
  • Ambulatory and able to walk 10 meters between ≥5 and ≤14 seconds (only assistive devices allowed are single cane or splints for ankle-foot orthoses)
  • Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader

You may not qualify if:

  • History of any illness or any clinical condition that, in the opinion of the Investigator, might confound the results of the study or pose an additional risk in administering investigational product to the participant.
  • History of biopsy of the same muscle within 30 days of the Screening biopsy or planning to undergo any non-study muscle biopsies over the duration of the study
  • Treatment with an oligonucleotide, gene therapy, or other experimental therapies for FSHD.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

NOT YET RECRUITING

Liverpool Hospital

Liverpool, New South Wales, 2170, Australia

NOT YET RECRUITING

Wesley Research Institute

Brisbane, Queensland, 4066, Australia

NOT YET RECRUITING

Mater Hospital Brisbane

South Brisbane, Queensland, 4101, Australia

RECRUITING

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

NOT YET RECRUITING

Pacific Clinic Research Network

Auckland, Auckland, 0622, New Zealand

NOT YET RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral

Interventions

Injections

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration RoutesDrug TherapyTherapeutics

Central Study Contacts

Soufflé Therapeutics, Inc.

CONTACT

1 617-458-7581SFL0821clinicaltrials@souffletx.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 17, 2026

First Posted

September 1, 2026

Study Start

September 1, 2026

Primary Completion (Estimated)

March 1, 2028

Study Completion (Estimated)

April 1, 2028

Last Updated

September 29, 2026

Record last verified: 2026-09

Data Sharing

IPD Sharing
Will not share

Locations

NZ(1)AU(5)