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Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)

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ClinicalTrials.gov Identifier: NCT05747924
Recruitment Status : Recruiting
First Posted : February 28, 2023
Last Update Posted : February 2, 2024
Sponsor:
Information provided by (Responsible Party):
Avidity Biosciences, Inc.

Brief Summary:
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Condition or disease Intervention/treatment Phase
FSHD FSHD1 FSHD2 FMD FMD2 Fascioscapulohumeral Muscular Dystrophy Fascioscapulohumeral Muscular Dystrophy Type 1 Fascioscapulohumeral Muscular Dystrophy Type 2 Dystrophies, Facioscapulohumeral Muscular Dystrophy, Facioscapulohumeral Muscular Facioscapulohumeral Muscular Dystrophy 1 Facioscapulohumeral Muscular Dystrophy 2 Facio-Scapulo-Humeral Dystrophy Atrophy, Facioscapulohumeral Atrophies, Facioscapulohumeral Facioscapulohumeral Atrophy Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral FSH Muscular Dystrophy Landouzy Dejerine Dystrophy Landouzy-Dejerine Muscular Dystrophy Dystrophies, Landouzy-Dejerine Dystrophy, Landouzy-Dejerine Landouzy-Dejerine Syndrome Muscular Dystrophy, Landouzy Dejerine Progressive Muscular Dystrophy FSH Drug: AOC 1020 Drug: Placebo Phase 1 Phase 2

Detailed Description:

AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in adult participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).

Part A is a dose titration design which includes a single and multiple dose schedule with 1 cohort. Part B is a single-ascending and multiple-ascending dose design with 2 cohorts. Part C is a parallel, multi-dose cohort design with 1 cohort. For each of Part A, B, and C the patient duration is 12 months as the active treatment period is approximately 9 months followed by a 3-month follow-up period. Once participants have completed active treatment and the follow-up period, they may have the option to participate in a planned open-label extension. If patients do not enroll in the open-label extension, they will be followed through a 6-month safety follow-up period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Actual Study Start Date : April 4, 2023
Estimated Primary Completion Date : September 2025
Estimated Study Completion Date : September 2025


Arm Intervention/treatment
Experimental: AOC 1020 Regimen 1
Part A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion

Experimental: AOC 1020 Regimen 2
Part B1 & C: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion

Experimental: AOC 1020 Regimen 3
Part B2 & C: AOC 1020 Dose Regimen 3; Five doses administered intravenously over 9 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion

Placebo Comparator: Placebo (Saline)
Part A, B & C: Placebo; Five doses administered intravenously over 9 months
Drug: Placebo
Placebo will be administered via intravenous (IV) infusion
Other Name: Saline




Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events [ Time Frame: Through study completion, up to Day 365 ]

Secondary Outcome Measures :
  1. Plasma pharmacokinetic (PK) parameters of AOC 1020 [ Time Frame: Through study completion; up to Day 365 ]
    Observed maximum concentration

  2. Plasma pharmacokinetic (PK) parameters of AOC 1020 [ Time Frame: Through study completion; up to Day 365 ]
    Observed half-life

  3. Plasma pharmacokinetic (PK) parameters of AOC 1020 [ Time Frame: Through study completion; up to Day 365 ]
    Observed area under the curve

  4. Muscle drug concentration [ Time Frame: Day 120 ]
    Concentration of siRNA component in skeletal muscle

  5. Urine drug concentration [ Time Frame: 0 - 24 hours after first and third dose ]
    Fraction of drug excreted in urine



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
  • 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 (testing provided by Sponsor)
  • Muscle weakness in both upper and lower body, as determined by Investigator

Exclusion Criteria:

  • Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
  • Unwilling or unable to continue to comply with contraceptive requirements
  • Body mass index (BMI) >35.0 kg/m2 at Screening
  • 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
  • Any contraindication to MRI
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05747924


Contacts
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Contact: Amy Halseth, PhD 858-771-7038 medinfo@aviditybio.com

Locations
Show Show 17 study locations
Sponsors and Collaborators
Avidity Biosciences, Inc.
Additional Information:
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Responsible Party: Avidity Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT05747924    
Other Study ID Numbers: AOC 1020-CS1
2022-002704-20 ( EudraCT Number )
2022-502096-32-00 ( Other Identifier: EU CT Number )
First Posted: February 28, 2023    Key Record Dates
Last Update Posted: February 2, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Avidity Biosciences, Inc.:
FORTITUDE
Avidity
Avidity Biosciences
AOC 1020
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
Atrophy
Pathological Conditions, Anatomical
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn