Health TrialsbyHealthPanel

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

ConditionsFacioscapulohumeral Muscular DystrophyFSHDFSHD - Facioscapulohumeral Muscular DystrophyFSHD1FSHD2Fascioscapulohumeral Muscular DystrophyFascioscapulohumeral Muscular Dystrophy Type 1Fascioscapulohumeral Muscular Dystrophy Type 2Facioscapulohumeral Muscular Dystrophy 1Facioscapulohumeral DystrophyFacio-Scapulo-Humeral DystrophyFacioscapulohumeral Muscular Dystrophy 2Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)FSH Muscular DystrophyLandouzy Dejerine DystrophyLandouzy-Dejerine Muscular DystrophyLandouzy-Dejerine Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age16-70
SponsorAvidity Biosciences, Inc.

About this trial

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Clinical and genetic diagnosis of FSHD1 or FSHD2

Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening

Adequate muscle strength based on QMT composite score

Disqualifiers

Breastfeeding, pregnancy, or intent to become pregnant during the study

Unwilling or unable to comply with contraceptive requirements

Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study

Blood Pressure > 140/90 mmHg at Screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • AOC-1020

    Drug

    Del-brax will be administered by intravenous (IV) infusion.

  • Placebo

    Drug

    Placebo will be administered by intravenous (IV) infusion.

Treatment groups

200 Participants
are divided into 2 treatment groups
Group A: Del-braxExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)

Assessment of muscle strength using standardized quantitative measurements

Time frame
Baseline through Week 78

Secondary outcomes

1

Change from Baseline to Week 78 in 10MWRT

Assessment of the time required to walk or run 10 meters as quickly and safely as possible

Time frame
Baseline through Week 78
2

Change from Baseline to Week 78 in Timed Up-and-Go (TUG)

Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down

Time frame
Baseline through Week 78
3

Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire

Assessment of patient-reported physical function using the PROMIS Physical Function Short Form 20a questionnaire

Time frame
Baseline through Week 78
4

Change from Baseline to Week 78 in PROMIS Fatigue

Assessment of patient-reported fatigue using the PROMIS Fatigue questionnaire

Time frame
Baseline through Week 78

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Avidity Biosciences, Inc.

Lead sponsor

Source ClinicalTrials.gov
Apply now