A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AOC-1020, Placebo.
- Who it may be relevant to
- Registry conditions: Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1. Basic parameters: 16 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada, Denmark, France, Germany +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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)
Overview
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)
Detailed description
The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).
Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.
After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety.
An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Interventions
- Drug AOC-1020
Del-brax will be administered by intravenous (IV) infusion. - Drug Placebo
Placebo will be administered by intravenous (IV) infusion.
Primary outcome measures
- Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN) [Time frame: Baseline through Week 78]
Secondary outcome measures (10)
- Change from Baseline to Week 78 in 10MWRT [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in Timed Up-and-Go (TUG) [Time frame: Baseline through Week 78]
- 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 [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in PROMIS Fatigue [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS) [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C) [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L [Time frame: Baseline through Week 78]
- Change from Baseline to Week 78 in Serum CK [Time frame: Baseline through Week 78]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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
- Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
- Treatment with an oligonucleotide within 9 months of Screening
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 14 centers
- University of California Irvine — Orange
- Stanford University — Palo Alto
- University of Colorado — Denver
- University of Florida — Gainesville
- University of Iowa — Iowa City
- Kansas University Medical Center — Kansas City
- Kennedy Krieger Institute — Baltimore
- University of Massachusetts — Worcester
- … and 6 more centers
Canada · 4 centers
- University of Calgary - Cumming School of Medicine — Calgary
- University of Alberta Hospital — Edmonton
- The Ottawa Hospital — Ottawa
- Genge Partners Inc — Montreal
France · 4 centers
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timon — Marseille
- CHU de Montpellier - Hopital Gui de Chauliac — Montpellier
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires — Nice
- AP-HP Hopital Pitie-Salpetriere — Paris
Germany · 4 centers
- Universitaetsklinikum Bonn — Bonn
- Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Go — Göttingen
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München
- Universitaetsklinikum Ulm — Ulm
Italy · 4 centers
- Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan
- Azienda Ospedaliero Universitaria Pisana — Pisa
- Azienda Ospedaliero-Universitaria Sant'Andrea — Roma
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuo — Roma
Japan · 4 centers
- National Hospital Organization Osaka Toneyama Medical Center — Osaka
- The University of Osaka Hospital — Osaka
- National Hospital Organization Sendai Nishitaga Hospital — Sendai
- National Center of Neurology and Psychiatry — Tokyo
Spain · 4 centers
- Hospital Universitari de Bellvitge — Barcelona
- Hospital Universitario Vall d'Hebron — Barcelona
- Hospital Universitario Donostia — San Sebastián
- Hospital Universitari i Politecnic La Fe — Valencia
United Kingdom · 4 centers
- National Hospital for Neurology & Neurosurgery — London
- St. George's University Hospitals NHS Foundation Trust — London
- Royal Victoria Infirmary — Newcastle upon Tyne
- Royal Hallamshire Hospital — Sheffield
Denmark · 2 centers
- Aarhus University Hospital — Aarhus
- Rigshospitalet — Copenhagen
Netherlands · 2 centers
- Academisch Ziekenhuis Leiden — Leiden
- Stichting Radboud Universitair Medisch Centrum — Nijmegen
Identifiers
NCT: NCT07038200 · AOC 1020-CS3