Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - First administration to humans
Status
Ended
Participants planned
72
Countries
2
Sites
3
Facioscapulohumeral muscular dystrophy (FSHD)
To evaluate the safety and tolerability of ascending doses of AOC 1020 in participants with FSHD
Key facts
- Sponsor
- Avidity Biosciences Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- completed 20 Mar 2026
- Decision date (initial)
- 2023-07-26
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Avidity Biosciences Inc.
External identifiers
- EU CT number
- 2022-502096-32-00
- ClinicalTrials.gov
- NCT05747924
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic, Pharmacodynamic, Efficacy, Safety
To evaluate the safety and tolerability of ascending doses of AOC 1020 in participants with FSHD
Secondary objectives 1
- To evaluate the pharmacokinetic (PK) profile of intravenous (IV) doses of AOC 1020 in participants with FSHD
Conditions and MedDRA coding
Facioscapulohumeral muscular dystrophy (FSHD)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10064087 | Facioscapulohumeral muscular dystrophy | 100000004850 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Treatment/ Placebo 3-part, multi-center, Phase 1/2, randomized, double- blind, placebo-controlled study.
Part A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Part B comprises 2 placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohorts (Cohorts B1 and B2). Part C comprises a 3-arm, parallel, randomized, placebo-controlled, multiple-dose cohort expansion (Cohort C1).
Study duration:
Approximately 14 months (8-week Screening/Baseline, 9-month treatment, 3-month Post-treatment Follow-up)
Participants who do not immediately roll-over into the OLE study or decline participation in the OLE study will be followed for another 6 months in an Extended Follow-up Period.
|
Randomised Controlled | Double | [{"id":24837,"code":2,"name":"Investigator"},{"id":24838,"code":3,"name":"Monitor"},{"id":24839,"code":4,"name":"Analyst"},{"id":24840,"code":1,"name":"Subject"}] | Part A- Dose Titration and Nested SAD (Single Ascending Dose)/MAD (Multiple Ascending Dose): 1 cohort: Cohort A1 1 mg/kg or placebo first dose up to 2 mg/kg or placebo Part B- Nested SAD (Single Ascending Dose)/MAD (Multiple Ascending Dose): 2 cohorts Cohort B1: Up to 4 mg/kg or placebo Cohort B2: Up to 8 mg/kg or placebo Part C- Parallel, Multiple Dose Expansion: 1 cohort Up to 4 mg/kg, up to 8 mg/kg or placebo |
Regulatory references
- Scientific advice from competent authorities
- Medicines Evaluation Board
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing
- Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
- At least 1 muscle region suitable for biopsy
- Muscle weakness in both upper and lower body, as determined by Investigator
Exclusion criteria 10
- Any contraindication to MRI
- Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
- Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
- Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening
- Unwilling or unable to comply with the contraceptive requirements
- Body mass index (BMI) >35.0 kg/m2 at Screening. Body weight of > 120 kg at Screening ineligible for participation in cohorts with planned siRNA dose levels > 4 mg/kg.
- History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
- History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
- Anticipated survival less than 2 years
- Blood or plasma donation within 16 weeks of Study Day 1
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Incidence of treatment-emergent adverse events
Secondary endpoints 2
- Estimation of plasma PK parameters, including maximum plasma concentration, half-life, and area under the curve of AOC 1020
- Concentration of siRNA component in skeletal muscle (Day 120)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10206320 · Product
- Active substance
- Humanised IGG1 Monoclonal Antibody Against TFR1 Conjugated to Double Stranded Sirna Oligonucleotide Against DUX4 Mrna via a Non-Cleavable Linker
- Substance synonyms
- AV01mAb-FSHD01, AOC 1020
- Pharmaceutical form
- POWDER FOR INFUSION
- Route of administration
- INTRAVENOUS
- Authorisation status
- Not Authorised
- ATC code
- NOTASSIGN — -
- MA holder
- AVIDITY BIOSCIENCES
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/23/2756
Placebo 1
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
- 10578 Science Center Drive Suite 125
- City
- San Diego
- Postcode
- 92121-1145
- Country
- United States
Scientific contact point
- Organisation
- Avidity Biosciences Inc.
- Contact name
- Teresa Brandt
Public contact point
- Organisation
- Avidity Biosciences Inc.
- Contact name
- Teresa Brandt
Third parties 14
| Organisation | City, country | Duties |
|---|---|---|
| Propharma Group The Netherlands B.V. ORG-100013065
|
Leiden, Netherlands | Other |
| 4 Global Lean Ra ORG-100027377
|
Pont-A-Celles, Belgium | Interactive response technologies (IRT) |
| University Of Iowa Hospitals And Clinics ORG-100032405
|
Iowa City, United States | Other, Laboratory analysis |
| Eclinical Solutions LLC ORG-100044778
|
Mansfield, United States | Data management, E-data capture |
| Gray Consulting Inc. ORG-100044159
|
Philadelphia, United States | Other |
| Biologics Development Services LLC ORG-100044619
|
Tampa, United States | Other, Laboratory analysis |
| Longboat Clinical Limited ORG-100045828
|
Limerick, Ireland | Other |
| Bioniks ORG-100046879
|
Alameda, United States | Other |
| Axolabs GmbH ORG-100043876
|
Kulmbach, Germany | Other, Laboratory analysis |
| Worldwide Clinical Trials ORG-100030991
|
Grad Zagreb, Croatia | Code 11, Code 12, Other, Other, Code 2, Code 5, Data management, Code 8 |
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | Other |
| Biotel Research LLC ORG-100039864
|
Rochester, United States | Other |
| Elligo Health Research Inc. ORG-100044201
|
Austin, United States | Other |
| Acm Global Central Laboratory Limited ORG-100042459
|
York, United Kingdom | Laboratory analysis |
Locations
2 EU/EEA countries · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Ended | 6 | 2 |
| Netherlands | Ended | 6 | 1 |
| Rest of world
United States, Canada, United Kingdom
|
— | 60 | — |
Investigational sites
Azienda Ospedaliero-Universitaria Sant Andre
Neurology Unit, Via Di Grottarossa 1035-1039, 00189, Rome
Centro Clinico Nemo
Centro Clinico NeMO -MIlano, Piazza Dell'ospedale Maggiore 3, 20162, Milan
Centre for Human Drug Research
Neurology, Zernikedreef 8, 2333 CL, Leiden
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-03-31 | Netherlands | Acceptable with conditions 2023-07-24
|
2023-07-25 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2023-08-10 | Netherlands | Acceptable with conditions 2023-07-24
|
2023-08-10 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-08-29 | Netherlands | Acceptable 2023-12-04
|
2023-12-04 |