Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruitment ended
Participants planned
150
Countries
7
Sites
11
Myotonic Dystrophy Type 1
To evaluate the efficacy of AOC 1001 on hand function.
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
- 11 Dec 2024 → ongoing
- Decision date (initial)
- 2024-08-21
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Avidity Biosciences Inc.
External identifiers
- EU CT number
- 2024-511378-60-00
- WHO UTN
- U1111-1303-5875
- ClinicalTrials.gov
- NCT06411288
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Pharmacokinetic
To evaluate the efficacy of AOC 1001 on hand function.
Secondary objectives 1
- To evaluate the efficacy of AOC 1001 on measures of mobility, muscle strength, muscle function, and patient-reported outcomes.
Conditions and MedDRA coding
Myotonic Dystrophy Type 1
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 27.0 | LLT | 10090453 | Myotonic dystrophy type 1 | 100000004850 |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency, Food And Drug Administration
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2025-520766-22-00 | A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy 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 3
- 1. Age: 16 to 65. Age: 18-65 (Denmark, Germany)
- 2. Clinical and genetic diagnosis (CTG repeat length ≥ 100) of DM1.
- 3. Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening.
Exclusion criteria 9
- 1. Breastfeeding, pregnancy, or intent to become pregnant during the study.
- 2. Unwilling or unable to comply with contraceptive requirements.
- 3. Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study.
- 4. Diabetes that is not adequately controlled.
- 5. History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
- 6. Body Mass Index > 35 kg/m2 at Screening.
- 7. Recently treated with an investigational drug or biological agent.
- 8. Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.
- 9. Note: Additional protocol defined inclusion and exclusion criteria apply.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Change from baseline to Week 54 in video Hand Opening Time (vHOT).
Secondary endpoints 2
- 1. Change from baseline to Week 54 in: • Hand grip strength. • QMT total composite score. • DM1-ActivC. • 10MWRT
- 2. Change from baseline to Week 54 in: • QMT upper extremity composite score. • QMT lower extremity composite score. • QMT individual muscle group scores. • PGI-S. • EQ-5D-5L. PGI-C at Week 54
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
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
- 28 mg/kg milligram(s)/kilogram
- Max treatment duration
- 54 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- AVIDITY BIOSCIENCES
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2485
PRD11292340 · Product
- Active substance
- Humanised IGG1 Monoclonal Antibody Against TFR1 Conjugated to Double Stranded Sirna Oligonucleotide Against Dmpk via a Non-Cleavable Linker
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 4 mg/kg milligram(s)/kilogram
- Max total dose
- 28 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 54 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- AVIDITY BIOSCIENCES
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2485
Placebo 1
0.9% Saline for IV administration
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
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 8
| Organisation | City, country | Duties |
|---|---|---|
| Atreo Inc. ORG-100045217
|
San Francisco, United States | Interactive response technologies (IRT) |
| Gray Consulting Inc. ORG-100044159
|
Philadelphia, United States | Other |
| Aparito Limited ORG-100026728
|
Wrexham, United Kingdom | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| ProPharma Group GmbH ORG-100008074
|
Berlin, Germany | Code 8 |
| Trinds LLC ORG-100051849
|
Pittsburgh, United States | Other |
| Packaging Coordinators LLC ORG-100011552
|
Philadelphia, United States | Code 14 |
| Medpace Finland Oy ORG-100009147
|
Helsinki, Finland | On site monitoring, Code 10, Code 11, Code 12, Code 2, Interactive response technologies (IRT), Laboratory analysis, Code 5, Data management, E-data capture |
Locations
7 EU/EEA countries · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Denmark | Ongoing, recruitment ended | 6 | 2 |
| France | Ongoing, recruitment ended | 6 | 2 |
| Germany | Ongoing, recruitment ended | 6 | 1 |
| Ireland | Ended | 3 | 1 |
| Italy | Ongoing, recruitment ended | 7 | 2 |
| Netherlands | Ongoing, recruitment ended | 9 | 2 |
| Spain | Ongoing, recruitment ended | 2 | 1 |
| Rest of world
New Zealand, Japan, United Kingdom, Canada, Australia, United States
|
— | 111 | — |
Investigational sites
Aarhus Universitetshospital
Neurological department, J120, Palle Juul-Jensens Boulevard 165, Aarhus N
Copenhagen University Hospital
Neuromuskulaer klinik, Blegdamsvej 9, 2100, Copenhagen Oe
Centre Hospitalier Regional De Marseille
Maladies neuromusculaires, 264 Rue Saint Pierre, 13005, Marseille
Hopitaux Universitaires Pitie Salpetriere
Neuromyologie, 47 To 83 Boulevard De L Hopital, 75013, Paris
Klinikum der Universitaet Muenchen AöR
Department of Neurology, Ziemssenstrasse 1, Ludwigsvorstadt-Isarvorstadt, Munich
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
NeuroScience, Sensory Organs and Chest, Largo Francesco Vito 1, 00168, Rome
Centro Clinico Nemo
Centro Clinico NeMO di Milano, Piazza Dell'ospedale Maggiore 3, 20162, Milan
Stichting Radboud universitair medisch centrum
Neurology, Geert Grooteplein Zuid 10, 6525 GA, Nijmegen
Academisch Ziekenhuis Maastricht
Neurology, P Debyelaan 25, 6229 HX, Maastricht
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 |
|---|---|---|---|---|---|
| Denmark | 2025-03-03 | 2025-04-10 | 2025-07-28 | ||
| France | 2024-12-16 | 2025-01-07 | 2025-07-28 | ||
| Germany | 2025-03-04 | 2025-03-13 | 2025-07-28 | ||
| Italy | 2024-12-11 | 2024-12-12 | 2025-07-28 | ||
| Netherlands | 2024-12-13 | 2025-01-07 | 2025-07-28 | ||
| Spain | 2025-03-28 | 2025-04-10 | 2025-07-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 98 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-511378-60-00_Avidity_REDACTED | 8.2 |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_DE_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_DK_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_ES_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_FR_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_IE_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_IT_Avidity_Licensing agreement statement_blank | N/A |
| Protocol (for publication) | D4_Patient facing documents_ePRO Screenshots_NL_Avidity_Licensing agreement statement_blank | N/A |
| Recruitment arrangements (for publication) | K1_Additional Document_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DE_Avidity_redacted | 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_IE_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_redacted | 1.0 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_Blank | None |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | NL V1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Brochure_Avidity_redacted | NL V1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Handbook_Avidity_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Handbook_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Handbook_Avidity_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Handbook_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Handbook_Avidity_redacted | EU V1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Handbook_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Handbook_Avidity_Blank | None |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Handbook_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_Blank | None |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | N/A |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | NA |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_blank | na |
| Recruitment arrangements (for publication) | K2_Recruitment material_Participant Journey_Avidity_redacted | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Parent_Guardian_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adults_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_Avidity_redacted | 2.0 |
| 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 | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_Avidity_redacted | 2.0 |
| 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_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 Adult Parent_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main_Avidity_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-Screening_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-Screening_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-screening_Avidity_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-Screening_Avidity_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-Screening_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pre-Screening_Avidity_redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnancy_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Participant_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner_Avidity_redacted | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_PregnantPartner_Avidity_Redacted | 2.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ C-SSRS baseline_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ C-SSRS since last visit_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ ePRO Practice Entry Email Reminders_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ ePRO Quick Reference Guide_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ TrialPACE system Terms and Conditions_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincierge Travel Policy IRL_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincierge Travel Policy Stipends IRL_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincierge_Pay Portal Guide_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Emergency Contact Card_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_GP Letter_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Participant Welcome Letter IRL_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Participant Welcome Letter Stipends IRL_Avidity | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Retention Sheet_Avidity_blank | NA |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Your rights as a trial participant_Avidity | None |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_Sodium Chloride_Avidity | NA |
| Synopsis of the protocol (for publication) | D1_ProtocolLaySynopsis_EN_2024-511378-60_Avidity | 8.2 |
| Synopsis of the protocol (for publication) | D1_ProtocolLaySynopsis_ES_2024-511378-60_Avidity | 8.2 |
| Synopsis of the protocol (for publication) | D1_ProtocolLaySynopsis_NL_2024-511378-60_Avidity | 8.2 |
| Synopsis of the protocol (for publication) | D1_ProtocolTECHsynopsis_EN_2024-511378-60_Avidity_redacted | 8.2 |
| Synopsis of the protocol (for publication) | D1_ProtocolTECHsynopsis_FR_2024-511378-60_Avidity_redacted | 8.2 |
| Synopsis of the protocol (for publication) | D1_ProtocolTECHsynopsis_IT_2024-511378-60_Avidity_redacted | 8.2 |
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-05-17 | Netherlands | No conclusion 2024-08-05
|
2024-08-08 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-09-30 | Netherlands | Acceptable 2024-11-25
|
2024-11-25 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-01-10 | Netherlands | Acceptable 2025-04-22
|
2025-04-22 |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-04-29 | Netherlands | Acceptable 2025-08-11
|
2025-08-11 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-11-18 | Netherlands | Acceptable 2025-08-11
|
2025-11-18 |
| 6 | SUBSTANTIAL MODIFICATION | SM-4 | 2026-01-08 | Netherlands | Acceptable 2026-04-08
|
2026-04-08 |
| 7 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-04-14 | Acceptable 2026-04-08
|
2026-04-14 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-04-15 | Acceptable | 2026-06-01 |