Trial record 1 of 1 for:
NCT05479981
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)
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| ClinicalTrials.gov Identifier: NCT05479981 |
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Recruitment Status :
Enrolling by invitation
First Posted : July 29, 2022
Last Update Posted : October 17, 2022
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Sponsor:
Avidity Biosciences, Inc.
Information provided by (Responsible Party):
Avidity Biosciences, Inc.
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Brief Summary:
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| DM1 Muscular Dystrophies Myotonic Dystrophy Myotonic Dystrophy 1 Myotonic Disorders Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases | Drug: Placebo Drug: AOC 1001 | Phase 2 |
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.
Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.
The total duration of active treatment in AOC 1001-CS2 is approximately 24 months. Once participants have completed active treatment, they will be followed through a 9-month safety follow-up period. The sponsor may extend active treatment beyond 24 months at a future timepoint.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 44 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Masking Description: | Participants will receive one blinded treatment at Day 43. Treatment assignment on this day will be based on treatment received in AOC 1001-CS1 so that participants who did not receive active drug on Day 43 in AOC 1001-CS1 will received active drug on Day 43 in AOC 1001-CS2. |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients |
| Actual Study Start Date : | August 4, 2022 |
| Estimated Primary Completion Date : | April 2025 |
| Estimated Study Completion Date : | April 2025 |
Resource links provided by the National Library of Medicine
| Arm | Intervention/treatment |
|---|---|
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Experimental: AOC 1001
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2.
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Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion. |
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Experimental: AOC 1001 (with Placebo at Day 43)
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2.
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Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.
Other Name: Saline Drug: AOC 1001 AOC 1001 will be administered by intravenous (IV) infusion. |
Primary Outcome Measures :
- Number and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Through study completion, up to Day 729 ]
Secondary Outcome Measures :
- Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 729 ]Maximum and trough plasma concentration
- AOC 1001 levels in muscle tissue [ Time Frame: Through Day 183 ]
- Change and percentage change from baseline in DMPK mRNA knockdown [ Time Frame: Through Day 183 ]
- Change and percentage change from baseline in Spliceopathy [ Time Frame: Through Day 183 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 66 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Key Exclusion Criteria:
- Pregnancy, intent to become pregnant, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Any new conditions or worsening of existing conditions 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
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): NCT05479981
Locations
| United States, California | |
| University of California Los Angeles | |
| Los Angeles, California, United States, 90095 | |
| Stanford University | |
| Palo Alto, California, United States, 94304 | |
| United States, Colorado | |
| University of Colorado | |
| Denver, Colorado, United States, 80045 | |
| United States, Florida | |
| University of Florida | |
| Gainesville, Florida, United States, 32608 | |
| United States, Kansas | |
| Kansas University Medical Center | |
| Kansas City, Kansas, United States, 66205 | |
| United States, New York | |
| University of Rochester Medical Center | |
| Rochester, New York, United States, 14642 | |
| United States, Ohio | |
| Ohio State University | |
| Columbus, Ohio, United States, 43221 | |
| United States, Virginia | |
| Virginia Commonwealth University | |
| Richmond, Virginia, United States, 23298 | |
Sponsors and Collaborators
Avidity Biosciences, Inc.
Investigators
| Study Director: | Li Tai, MD | Avidity Biosciences, Inc. |
| Responsible Party: | Avidity Biosciences, Inc. |
| ClinicalTrials.gov Identifier: | NCT05479981 |
| Other Study ID Numbers: |
AOC 1001-CS2 |
| First Posted: | July 29, 2022 Key Record Dates |
| Last Update Posted: | October 17, 2022 |
| Last Verified: | October 2022 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Avidity Biosciences, Inc.:
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DM1 Myotonic Dystrophy 1 Myotonic Dystrophy Type 1 (DM1) Myotonic Dystrophy DM Dystrophy Myotonic Myotonic Disorders |
Steinert Disease MARINA-OLE MARINA Avidity Biosciences Avidity AOC 1001 Myotonic Muscular Dystrophy |
Additional relevant MeSH terms:
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Muscular Dystrophies Musculoskeletal Diseases Myotonic Dystrophy Muscular Diseases Myotonic Disorders Muscular Disorders, Atrophic Nervous System Diseases |
Neurodegenerative Diseases Neuromuscular Diseases Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Disease Pathologic Processes |