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
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 17-05-2024
- First CTIS Authorization Date
- 08-08-2024
Trial design
Randomised, placebo: 0.9% saline for iv administration (placebo comparator). no dose/schedule specified in the available dataset.-controlled Phase III trial in France, Germany, Denmark and others.
- Randomised
- Yes
- Comparator
- Placebo: 0.9% Saline for IV administration (placebo comparator). No dose/schedule specified in the available dataset.
- Target Sample Size
- 111
- Trial Duration For Participant
- 378
Eligibility
Recruits 111 paediatric patients.
- Pregnancy Exclusion
- 1. Breastfeeding, pregnancy, or intent to become pregnant during the study.
- Vulnerable Population
- Vulnerable population selected. Minors are included in some countries (age 16–17 allowed per inclusion criteria; Denmark and Germany restrict to 18–65). Assent and parental/guardian consent documents are provided (documents listed include L1_SIS and ICF_Assent, L1_SIS and ICF_Parental, L1_SIS and ICF_Parent_Guardian), indicating assent for minors and parental/guardian consent handling as per the provided subject information and consent forms.
Inclusion criteria
- {"criterion_text":"- 1. Age: 16 to 65. Age: 18-65 (Denmark, Germany)\n- 2. Clinical and genetic diagnosis (CTG repeat length ≥ 100) of DM1.\n- 3. Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening."}
Exclusion criteria
- {"criterion_text":"- 1. Breastfeeding, pregnancy, or intent to become pregnant during the study.\n- 2. Unwilling or unable to comply with contraceptive requirements.\n- 3. Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study.\n- 4. Diabetes that is not adequately controlled.\n- 5. History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.\n- 6. Body Mass Index > 35 kg/m2 at Screening.\n- 7. Recently treated with an investigational drug or biological agent.\n- 8. Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.\n- 9. Note: Additional protocol defined inclusion and exclusion criteria apply."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline to Week 54 in video Hand Opening Time (vHOT).","definition_or_measurement_approach":"Measured as change from baseline to Week 54 in video Hand Opening Time (vHOT)."}
Secondary endpoints
- {"endpoint_text":"- 1. Change from baseline to Week 54 in: • Hand grip strength. • QMT total composite score. • DM1-ActivC. • 10MWRT","definition_or_measurement_approach":"Measured as change from baseline to Week 54 in the listed measures (hand grip strength, QMT total composite score, DM1-ActivC, 10MWRT)."}
- {"endpoint_text":"- 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","definition_or_measurement_approach":"Measured as change from baseline to Week 54 in the listed QMT composite and individual scores, PGI-S, EQ-5D-5L and PGI-C at Week 54."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 111
- Recruitment Window Months
- 24
- Consent Approach
- Informed consent documented via L1_SIS and ICF_Adults forms for adult participants. Assent is provided using L1_SIS and ICF_Assent forms for minors; parental/guardian consent handled via L1_SIS and ICF_Parental / L1_SIS and ICF_Parent_Guardian forms. Country-specific age inclusion differences noted (general age 16–65; Denmark and Germany 18–65), so local age-specific consent/assent procedures apply as per the provided subject information and consent documents.
Methods
- Country-specific recruitment arrangements and materials (K1 recruitment arrangements documents per country).
- Printed and participant-facing materials: brochure, participant handbook, participant journey (K2 recruitment material files).
- Site-based recruitment at participating neurology/neuromuscular clinics and hospitals listed in Part II site lists.
- Patient travel and reimbursement support (service provided by Gray Consulting Inc. / Clincierge) to facilitate participation.
- Use of digital tools and ePRO (ePRO screenshots, ePRO reminders, TrialPACE/ePRO materials) to support participant procedures and communications.
- Video-based assessment support (video Hand Opening Time) provided by Aparito (documented duty: Video hand opening time assessment).
Geography
- Total Number Of Sites
- 11
- Total Number Of Participants
- 39
France
- Earliest CTIS Part Ii Submission Date
- 01-08-2024
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 616
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Hopitaux Universitaires Pitie Salpetriere
- Department Name
- Neuromyologie
- Contact Person Name
- Guillaume Bassez
- Contact Person Email
- guillaume.bassez@aphp.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Maladies neuromusculaires
- Contact Person Name
- Shahram Attarian
- Contact Person Email
- Shahram.ATTARIAN@ap-hm.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 09-08-2024
- Latest Decision Or Authorization Date
- 09-04-2026
- Processing Time Days
- 608
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Klinikum der Universitaet Muenchen AöR
- Department Name
- Department of Neurology
- Contact Person Name
- Stephan Wenninger
- Contact Person Email
- Stephan.Wenninger@med.uni-muenchen.de
Denmark
- Earliest CTIS Part Ii Submission Date
- 06-08-2024
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 610
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Copenhagen University Hospital
- Department Name
- Neuromuskulaer klinik
- Contact Person Name
- Nicolai Preisler
- Contact Person Email
- nicolai.rasmus.preisler@regionh.dk
- Site Name
- Aarhus Universitetshospital
- Department Name
- Neurological department
- Contact Person Name
- Henning Andersen
- Contact Person Email
- hennande@rm.dk
Spain
- Earliest CTIS Part Ii Submission Date
- 10-07-2024
- Latest Decision Or Authorization Date
- 13-04-2026
- Processing Time Days
- 642
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitario Donostia
- Department Name
- Neurology
- Contact Person Name
- Adolfo Jose Lopez de Munain Arregui
- Contact Person Email
- adolfojose.lopezdemunainarregui@osakidetza.eus
Ireland
- Earliest CTIS Part Ii Submission Date
- 18-07-2024
- Latest Decision Or Authorization Date
- 23-04-2025
- Processing Time Days
- 279
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Beaumont Hospital
- Department Name
- Neurology
- Contact Person Name
- Stela Lefter
- Contact Person Email
- stelalefter2@beaumont.ie
Netherlands
- Earliest CTIS Part Ii Submission Date
- 12-08-2024
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 604
- Number Of Sites
- 2
- Number Of Participants
- 9
Sites
- Site Name
- Stichting Radboud universitair medisch centrum
- 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
- 03-06-2024
- Latest Decision Or Authorization Date
- 14-04-2026
- Processing Time Days
- 680
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- NeuroScience, Sensory Organs and Chest
- Contact Person Name
- Gabriella Silvestri
- Contact Person Email
- gabriella.silvestri@policlinicogemelli.it
- 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
Contract research organisations
- Name
- Medpace Finland Oy
- Responsibilities
- Sponsor duties codes: 1,10,11,12,2,3,4,5,6,7
- Name
- ProPharma Group GmbH
- Responsibilities
- Sponsor duties code: 8
- Name
- Medidata Solutions Inc.
- Responsibilities
- Sponsor duties code: 7
Third parties
- {"country":"United States","full_name":"Packaging Coordinators LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"ProPharma Group GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Aparito Limited","duties_or_roles":"Video hand opening time assessment","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient travel and reimbursement support","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Trinds LLC","duties_or_roles":"Clinical evaluator oversight & Motor function test training/Activities","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Atreo Inc.","duties_or_roles":"","organisation_type":"Non-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
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Orphan Designation
- Yes
- Maximum Dose
- Max daily: 4 mg/kg; Max total: 28 mg/kg
- Investigational Product Name
- 0.9% Saline for IV administration
- Modality
- Other
- Routes Of Administration
- Intravenous
- Route
- Intravenous
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