Clinical trial • Phase III • Musculoskeletal
Humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker for Facioscapulohumeral muscular dystrophy (FSHD)
Phase III trial of Humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavab…
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Facioscapulohumeral muscular dystrophy (FSHD)
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody | Oligonucleotide
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 10-07-2025
- First CTIS Authorization Date
- 04-11-2025
Trial design
Randomised, 0.9% saline for iv administration (placebo)-controlled Phase III trial in Italy, France, Spain and others.
- Randomised
- Yes
- Comparator
- 0.9% Saline for IV administration (placebo)
- Target Sample Size
- 120
- Trial Duration For Participant
- 546
Eligibility
Recruits 120 The protocol indicates vulnerable population selection is used and states: when enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity. (Written informed consent required for all participants.).
- Pregnancy Exclusion
- 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period or
- Vulnerable Population
- The protocol indicates vulnerable population selection is used and states: when enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity. (Written informed consent required for all participants.)
Inclusion criteria
- {"criterion_text":"- 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.\n- 2. Male and females with FSHD confirmed by documented genetic diagnosis as defined below: - FSHD1 - FSHD2\n- 3. 18 to 70 years of age at time of informed consent\n- 4. Able to walk independently at pre-specified walking speed (orthoses or ankle braces are allowed) for at least 10 meters at screening."}
Exclusion criteria
- {"criterion_text":"- 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period or\n- 10. Treatment with an oligonucleotide within 9 months of Screening\n- 11. Blood or plasma donation within 16 weeks of Study Day 1\n- 12. Recent history of or current drug or alcohol abuse in the opinion of the Investigator\n- 13. History of multiple drug allergies or history of allergic reaction to any component of, or excipient in, the Study Drug\n- 14. Presence or history of clinically significant illness, medical condition, or abnormal test result/finding that, in the opinion of the Investigator, could affect a participant’s safety or their ability to comply with study procedures and/or complete the study visit schedule. This may include, but is not limited to, a history of cardiovascular or central nervous system disease, neuromuscular diseases other than FSHD (e.g., myopathy, neuropathy, neuromuscular junction disorders), a cardiopulmonary condition, or a clinically significant mental disorder\n- 2. Males or females not willing to comply with the contraceptive requirements\n- 3. Screening laboratory results or any other clinically significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion.\n- 4. BP > 140/90 mmHg at Screening\n- 5. Anticipated survival less than 2 years\n- 6. Evidence of current or chronic infection with hepatitis C, hepatitis B, or HIV (including chronic infection requiring ongoing treatment to maintain viral suppression)\n- 7. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1 or ongoing symptoms related to recent infection causing impairment to ADL in the opinion of the Investigator\n- 8. Malignancy within 5 years, except for basal or squamous cell carcinoma, melanoma in situ of the skin, carcinoma in situ of the cervix, or other malignancies within 5 years that have been treated with curative intent and which are not expected to recur\n- 9. Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer; or participation or plan to participate in another interventional study with an investigational drug or device or other types of interventional studies (including those studying behavioral modification and/or physical therapy) while on study"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from Baseline to Week 78 in 10MWRT velocity (m/sec)","definition_or_measurement_approach":"Change from baseline to Week 78 measured as 10MWRT (10-Meter Walk/Run Test) velocity in meters/second."}
Secondary endpoints
- {"endpoint_text":"- 1.1 Key Secondary Endpoints • Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) velocity o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN)","definition_or_measurement_approach":"Change from baseline to Week 78 measured by: Timed-Up-and-Go (TUG) velocity; NeuroQoL Upper Extremity Function instrument; Quantitative Muscle Testing (QMT) total composite score (percentage of predicted normal, PPN)."}
- {"endpoint_text":"- 1.2 Other Secondary Endpoints • Change from Baseline over time in: o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity (PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Sleep Disturbance o DUX4-regulated plasma KHDC1L o Serum CK","definition_or_measurement_approach":"Longitudinal change from baseline in PROMIS Physical Function, PROMIS Fatigue, Worst Pain NRS, PGI-S/PGI-C, NeuroQoL Sleep Disturbance, DUX4-regulated plasma KHDC1L, and serum creatine kinase (CK) measured by the respective validated instruments and laboratory assays over study visits."}
Recruitment
- Planned Sample Size
- 120
- Recruitment Window Months
- 30
- Consent Approach
- Written informed consent (signed and dated) is required. The protocol specifies that when enrolling minors a legally designated representative must provide consent and the minor participant will receive information adapted to their age and mental maturity. Subject information and informed consent forms are provided (country-specific ICFs listed in documents).
Geography
- Total Number Of Sites
- 20
- Total Number Of Participants
- 80
Italy
- Earliest CTIS Part Ii Submission Date
- 06-10-2025
- Latest Decision Or Authorization Date
- 07-04-2026
- Processing Time Days
- 183
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Neurology
- Principal Investigator Name
- Mauro Monforte
- Principal Investigator Email
- mauro.monforte@policlinicogemelli.it
- Contact Person Name
- Mauro Monforte
- Contact Person Email
- mauro.monforte@policlinicogemelli.it
- Site Name
- Azienda Ospedaliero-Universitaria Sant Andre
- Department Name
- UOC Neurologia
- Principal Investigator Name
- Matteo Garibaldi
- Principal Investigator Email
- matteo.garibaldi@uniroma1.it
- Contact Person Name
- Matteo Garibaldi
- Contact Person Email
- matteo.garibaldi@uniroma1.it
- Site Name
- Centro Clinico Nemo
- Department Name
- Centro Clinico NeMO di Milano
- Principal Investigator Name
- Valeria Sansone
- Principal Investigator Email
- valeria.sansone@centrocliniconemo.it
- Contact Person Name
- Valeria Sansone
- Contact Person Email
- valeria.sansone@centrocliniconemo.it
- Site Name
- Azienda Ospedaliero Universitaria Pisana
- Department Name
- Experimental and Clinical Medicine, University of Pisa
- Principal Investigator Name
- Giulia Ricci
- Principal Investigator Email
- giuli.ricci@med.unipi.it
- Contact Person Name
- Giulia Ricci
- Contact Person Email
- giuli.ricci@med.unipi.it
France
- Earliest CTIS Part Ii Submission Date
- 17-09-2025
- Latest Decision Or Authorization Date
- 26-03-2026
- Processing Time Days
- 190
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- Neurology Peripheral Nervous System and Muscle
- Principal Investigator Name
- Sabrina SACCONI
- Principal Investigator Email
- sacconi.s@chu-nice.fr
- Contact Person Name
- Sabrina SACCONI
- Contact Person Email
- sacconi.s@chu-nice.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Neuromyology
- Principal Investigator Name
- Guillaume BASSEZ
- Principal Investigator Email
- guillaume.bassez@aphp.fr
- Contact Person Name
- Guillaume BASSEZ
- Contact Person Email
- guillaume.bassez@aphp.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Centre de Reference des Maladies Neuromusculaires rares et de la SLA
- Principal Investigator Name
- Shahram ATTARIAN
- Principal Investigator Email
- shahram.attarian@ap-hm.fr
- Contact Person Name
- Shahram ATTARIAN
- Contact Person Email
- shahram.attarian@ap-hm.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Service Explorations Neurologiques et centre de reference SLA
- Principal Investigator Name
- Florence Esselin
- Principal Investigator Email
- f-esselin@chu-montpellier.fr
- Contact Person Name
- Florence Esselin
- Contact Person Email
- f-esselin@chu-montpellier.fr
Spain
- Earliest CTIS Part Ii Submission Date
- 07-10-2025
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 168
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Neurology
- Principal Investigator Name
- Raul Juntas Morales
- Principal Investigator Email
- raul.juntas@vallhebron.cat
- Contact Person Name
- Raul Juntas Morales
- Contact Person Email
- raul.juntas@vallhebron.cat
- Site Name
- Hospital Universitario Donostia
- Department Name
- Neurology
- Principal Investigator Name
- Adolfo Jose Lopez de Munain Arregui
- Principal Investigator Email
- adolfojose.lopezdemunainarregui@osakidetza.eus
- Contact Person Name
- Adolfo Jose Lopez de Munain Arregui
- Contact Person Email
- adolfojose.lopezdemunainarregui@osakidetza.eus
- Site Name
- Bellvitge University Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Velina Nedkova Hristova
- Principal Investigator Email
- vnedkova@bellvitgehospital.cat
- Contact Person Name
- Velina Nedkova Hristova
- Contact Person Email
- vnedkova@bellvitgehospital.cat
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurology
- Principal Investigator Name
- Nuria Muelas Gómez
- Principal Investigator Email
- muelas_nur@gva.es
- Contact Person Name
- Nuria Muelas Gómez
- Contact Person Email
- muelas_nur@gva.es
Netherlands
- Earliest CTIS Part Ii Submission Date
- 07-10-2025
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 168
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Neurology
- Principal Investigator Name
- Nicol Voermans
- Principal Investigator Email
- nicol.voermans@radboudumc.nl
- Contact Person Name
- Nicol Voermans
- Contact Person Email
- nicol.voermans@radboudumc.nl
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- Neurology
- Principal Investigator Name
- Umesh Arvind Badrising
- Principal Investigator Email
- u.a.badrising@lumc.nl
- Contact Person Name
- Umesh Arvind Badrising
- Contact Person Email
- u.a.badrising@lumc.nl
Denmark
- Earliest CTIS Part Ii Submission Date
- 30-09-2025
- Latest Decision Or Authorization Date
- 24-03-2026
- Processing Time Days
- 175
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- Copenhagen University Hospital
- Department Name
- Neuromuscular Center
- Principal Investigator Name
- John Vissing
- Principal Investigator Email
- john.vissing@regionh.dk
- Contact Person Name
- John Vissing
- Contact Person Email
- john.vissing@regionh.dk
- Site Name
- Aarhus Universitethospital
- Department Name
- Neurological department
- Principal Investigator Name
- Henning Andersen
- Principal Investigator Email
- hennande@rm.dk
- Contact Person Name
- Henning Andersen
- Contact Person Email
- hennande@rm.dk
Germany
- Earliest CTIS Part Ii Submission Date
- 06-10-2025
- Latest Decision Or Authorization Date
- 25-03-2026
- Processing Time Days
- 170
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Universitaetsklinikum Bonn AöR
- Department Name
- Department of Neuromuscular Diseases
- Principal Investigator Name
- Jens Reimann
- Principal Investigator Email
- jens.reimann@ukbonn.de
- Contact Person Name
- Jens Reimann
- Contact Person Email
- jens.reimann@ukbonn.de
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- Department of Neurology
- Principal Investigator Name
- Jana Zschüntzsch
- Principal Investigator Email
- j.zschuentzsch@med.uni-goettingen.de
- Contact Person Name
- Jana Zschüntzsch
- Contact Person Email
- j.zschuentzsch@med.uni-goettingen.de
- Site Name
- Universitaetsklinikum Ulm AöR
- Department Name
- Department of Neurology
- Principal Investigator Name
- Angela Rosenbohm
- Principal Investigator Email
- angela.rosenbohm@uni-ulm.de
- Contact Person Name
- Angela Rosenbohm
- Contact Person Email
- angela.rosenbohm@uni-ulm.de
- Site Name
- LMU Klinikum Muenchen AöR
- Department Name
- Department of Neurology
- Principal Investigator Name
- Benedikt Schoser
- Principal Investigator Email
- benedikt.schoser@med.uni-muenchen.de
- Contact Person Name
- Benedikt Schoser
- Contact Person Email
- benedikt.schoser@med.uni-muenchen.de
Sponsor
Primary sponsor
- Full Name
- Avidity Biosciences Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Medpace Finland Oy
- Responsibilities
- Medical Monitoring, IDMC, QA, Site Contract and Budget Negotiation/Execution, Study Vendor Management
- Name
- PPD Development LP
- Name
- ProPharma Group GmbH
- Name
- Gray Consulting Inc.
- Responsibilities
- Patient Travel and Reimbursement Support
Third parties
- {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Clinical Evaluator Oversight and Training/Activities for Measures of Function and Strength Assessments","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"University Of Iowa Hospitals And Clinics","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient Travel and Reimbursement Support","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"Medical Monitoring, IDMC, QA, Site Contract and Budget Negotiation/Execution, Study Vendor Management","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Packaging Coordinators LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Atreo Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"ProPharma Group GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- AOC 1020
- Active Substance
- Humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker
- Modality
- Monoclonal antibody | Oligonucleotide
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Orphan Designation
- Yes
- Maximum Dose
- Max daily dose 2 mg/kg; max total dose 26 mg
- Investigational Product Name
- 0.9% Saline for IV administration
- Modality
- Other
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
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