Clinical trial • Phase III • Musculoskeletal
HUMANISED IGG1 MONOCLONAL ANTIBODY AGAINST TFR1 CONJUGATED TO DOUBLE STRANDED SIRNA OLIGONUCLEOTIDE AGAINST DMPK VIA A NON-CLEAVABLE LINKER for Myotonic dystrophy type 1
Phase III trial of HUMANISED IGG1 MONOCLONAL ANTIBODY AGAINST TFR1 CONJUGATED TO DOUBLE STRANDED SIRNA OLIGONUCLEOTIDE AGAINST DMPK VIA A NON-CLEAVABLE LI…
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Myotonic dystrophy type 1
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody|Oligonucleotide|ADC
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 06-06-2025
- First CTIS Authorization Date
- 29-09-2025
Trial design
open-label Phase III trial across 8 sites in Denmark, France, Germany and others.
- Open Label
- Yes
- Target Sample Size
- 171
Eligibility
Recruits 171 paediatric patients.
- Pregnancy Exclusion
- 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period.
- Vulnerable Population
- Minors are included: "When enrolling participants who are minors, it is necessary to also obtain consent from a legally authorized representative and the participant will receive information in a way adapted to their age and mental maturity." Assent and parental consent documents are provided (assent, parental ICF files present); subject information and ICFs are available in multiple languages as per national Part II documents.
Inclusion criteria
- {"criterion_text":"- 1. Ability to provide 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 authorized representative and the participant will receive information in a way adapted to their age and mental maturity.\n- 2. Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator."}
Exclusion criteria
- {"criterion_text":"- 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period.\n- 2. Unwilling to comply with contraceptive requirements.\n- 3. Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- 1. Incidence of TEAEs","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 171
- Recruitment Window Months
- 57
- Consent Approach
- Written informed consent is required. For minors, consent must also be obtained from a legally authorized representative and participants receive information adapted to their age and mental maturity. Assent and parental ICF documents are provided. Subject information and informed consent forms are available in multiple languages (English, French, Spanish, Dutch, Italian and country-specific versions as per Part II documents).
Geography
- Total Number Of Sites
- 8
- Total Number Of Participants
- 46
Denmark
- Earliest CTIS Part Ii Submission Date
- 28-08-2025
- Latest Decision Or Authorization Date
- 29-09-2025
- Processing Time Days
- 32
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Aarhus Universitethospital
- Department Name
- Neurological department
- Contact Person Name
- Henning Andersen
- Contact Person Email
- hennande@rm.dk
- Site Name
- Copenhagen University Hospital
- Department Name
- Neuromuskulaer klinik
- Contact Person Name
- Nicolai Preisler
- Contact Person Email
- nicolai.rasmus.preisler@regionh.dk
France
- Earliest CTIS Part Ii Submission Date
- 15-08-2025
- Latest Decision Or Authorization Date
- 02-10-2025
- Processing Time Days
- 48
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Hopitaux Universitaires Pitie Salpetriere
- Department Name
- Neuromyologie
- Contact Person Name
- Guillaume Bassez
- Contact Person Email
- guillaume.bassez@aphp.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 28-07-2025
- Latest Decision Or Authorization Date
- 06-10-2025
- Processing Time Days
- 70
- Number Of Sites
- 1
- Number Of Participants
- 8
Sites
- Site Name
- LMU Klinikum Muenchen AöR
- Department Name
- Department of Neurology
- Contact Person Name
- Stephan Wenninger
- Contact Person Email
- Stephan.Wenninger@med.uni-muenchen.de
Spain
- Earliest CTIS Part Ii Submission Date
- 20-08-2025
- Latest Decision Or Authorization Date
- 30-09-2025
- Processing Time Days
- 41
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Hospital Universitario Donostia
- Department Name
- Neurology
- Contact Person Name
- Roberto Fernandez Torron
- Contact Person Email
- roberto.fernandeztorron@osakidetza.eus
Netherlands
- Earliest CTIS Part Ii Submission Date
- 01-09-2025
- Latest Decision Or Authorization Date
- 01-10-2025
- Processing Time Days
- 30
- Number Of Sites
- 2
- Number Of Participants
- 13
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Neurology
- Contact Person Name
- Karlien Mul
- Contact Person Email
- karlien.mul@radboudumc.nl
- Site Name
- Academisch Ziekenhuis Maastricht
- Department Name
- Neurology
- Contact Person Name
- Catharina Faber
- Contact Person Email
- c.faber@mumc.nl
Italy
- Earliest CTIS Part Ii Submission Date
- 11-08-2025
- Latest Decision Or Authorization Date
- 02-10-2025
- Processing Time Days
- 52
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Centro Clinico Nemo
- Department Name
- Centro Clinico NeMO di Milano
- Contact Person Name
- Valeria Sansone
- Contact Person Email
- valeria.sansone@centrocliniconemo.it
Sponsor
Primary sponsor
- Full Name
- Avidity Biosciences Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Third parties
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code:7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Trinds LLC","duties_or_roles":"code:15: Clinical Evaluator Oversight & Motor Function Test Training/Activities","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"codes:1,11,12,2,4,5,6,7","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Atreo Inc.","duties_or_roles":"code:3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"code:15: Patient Travel and Reimbursement Support","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"code:15: Video Hand Opening Time Assesment","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"ProPharma Group GmbH","duties_or_roles":"code:8","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- AOC 1001
- Active Substance
- HUMANISED IGG1 MONOCLONAL ANTIBODY AGAINST TFR1 CONJUGATED TO DOUBLE STRANDED SIRNA OLIGONUCLEOTIDE AGAINST DMPK VIA A NON-CLEAVABLE LINKER
- Modality
- Monoclonal antibody|Oligonucleotide|ADC
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Orphan Designation
- Yes
- Maximum Dose
- 4 mg/kg (max daily); max total dose amount 112 (dose units mg/kg as provided)
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