Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
217
Countries
6
Sites
8
Myotonic Dystrophy Type 1
To evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients
Key facts
- Sponsor
- Avidity Biosciences Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- 21 Jan 2026 → ongoing
- Decision date (initial)
- 2025-09-29
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Avidity Biosciences Inc.
External identifiers
- EU CT number
- 2025-520766-22-00
- WHO UTN
- U1111-1317-9634
- ClinicalTrials.gov
- NCT07008469
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Others
To evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients
Conditions and MedDRA coding
Myotonic Dystrophy Type 1
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 28.0 | LLT | 10090453 | Myotonic dystrophy type 1 | 100000004850 |
Regulatory references
- Scientific advice from competent authorities
- Food And Drug Administration, European Medicines Agency
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-511378-60-00 | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 | Avidity Biosciences Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- 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.
- 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 3
- 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period.
- 2. Unwilling to comply with contraceptive requirements.
- 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 and secondary outcome measures (English text)
Primary endpoints 1
- 1. Incidence of TEAEs
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11866797 · Product
- Active substance
- Humanised IGG1 Monoclonal Antibody Against TFR1 Conjugated to Double Stranded Sirna Oligonucleotide Against Dmpk via a Non-Cleavable Linker
- Substance synonyms
- AV01mAb-MD01, AOC 1001
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 4 mg/kg milligram(s)/kilogram
- Max total dose
- 112 mg/kg milligram(s)/kilogram
- Max treatment duration
- 48 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- AVIDITY BIOSCIENCES
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2485
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Avidity Biosciences Inc.
- Sponsor organisation
- Avidity Biosciences Inc.
- Address
- 3020 Callan Road
- City
- San Diego
- Postcode
- 92121-1109
- Country
- United States
Scientific contact point
- Organisation
- Avidity Biosciences Inc.
- Contact name
- Avidity Biosciences Inc.
Public contact point
- Organisation
- Avidity Biosciences Inc.
- Contact name
- Avidity Biosciences Inc.
Third parties 7
| Organisation | City, country | Duties |
|---|---|---|
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| Trinds LLC ORG-100051849
|
Pittsburgh, United States | Other |
| Medpace Finland Oy ORG-100009147
|
Helsinki, Finland | On site monitoring, Code 11, Code 12, Code 2, Laboratory analysis, Code 5, Data management, E-data capture |
| Atreo Inc. ORG-100045217
|
San Francisco, United States | Interactive response technologies (IRT) |
| Gray Consulting Inc. ORG-100044159
|
Philadelphia, United States | Other |
| Chillibean Limited ORG-100042592
|
London, United Kingdom | Other |
| ProPharma Group GmbH ORG-100008074
|
Berlin, Germany | Code 8 |
Locations
6 EU/EEA countries · 8 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Denmark | Authorised, recruitment pending | 7 | 2 |
| France | Ongoing, recruiting | 5 | 1 |
| Germany | Ongoing, recruiting | 8 | 1 |
| Italy | Ongoing, recruiting | 10 | 1 |
| Netherlands | Ongoing, recruiting | 13 | 2 |
| Spain | Authorised, recruitment pending | 3 | 1 |
| Rest of world
Canada, United States, United Kingdom, Japan
|
— | 171 | — |
Investigational sites
Aarhus Universitethospital
Neurological department, Palle Juul Jensens Boulevard 165, 8200, Aarhus N
Copenhagen University Hospital
Neuromuskulaer klinik, Blegdamsvej 9, 2100, Copenhagen Oe
Hopitaux Universitaires Pitie Salpetriere
Neuromyologie, 47 To 83 Boulevard De L Hopital, 75013, Paris
LMU Klinikum Muenchen AöR
Department of Neurology, Ziemssenstrasse 1, Ludwigsvorstadt-Isarvorstadt, Munich
Centro Clinico Nemo
Centro Clinico NeMO di Milano, Piazza Dell'ospedale Maggiore 3, 20162, Milan
Academisch Ziekenhuis Maastricht
Neurology, P Debyelaan 25, 6229 HX, Maastricht
Radboud universitair medisch centrum Stichting
Neurology, Geert Grooteplein Zuid 10, 6525 GA, Nijmegen
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2026-02-11 | 2026-03-18 | |||
| Germany | 2026-04-15 | 2026-04-16 | |||
| Italy | 2026-01-21 | 2026-02-23 | |||
| Netherlands | 2026-01-30 | 2026-02-09 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 43 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2025-520766-22-00_Avidity_redacted | 2.3(EU) |
| Protocol (for publication) | D4_Patient facing documents _Avidity_Licensing agreement statement | NA |
| Recruitment arrangements (for publication) | K1_Additional Document_Avidity | NA |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DE_Avidity | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DK_Avidity_redacted | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_ES_Avidity_redacted | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_FR_Avidity_redacted | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_IT_Avidity_redacted | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_NL_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Clincierge Data Protection | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Clincierge Data Protection Notice_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Clincierge Data Protection Notice_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Clincierge Data Protection Notice_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Data Protection_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main adults_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PregnantParticipant_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PregnantPartner_Avidity_redacted | 1.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_Water for Injection_Avidity | NA |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_DU_2025-520766-22-00_Avidity | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_EN_2025-520766-22-00_Avidity | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_ES_2025-520766-22-00_Avidity | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol lay synopsis_FR_2025-520766-22-00_Avidity | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_EN_2025-520766-22-00_Avidity_redacted | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_FR_2025-520766-22-00_Avidity_redacted | 2.3(EU) |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_IT_2025-520766-22-00_Avidity_redacted | 2.3(EU) |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-06-06 | Spain | Acceptable 2025-09-29
|
2025-09-29 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-11-18 | Spain | Acceptable 2025-09-29
|
2025-11-18 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-01-08 | Spain | Acceptable 2026-03-10
|
2026-03-10 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-04-09 | Acceptable 2026-03-10
|
2026-04-09 |