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Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

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ClinicalTrials.gov Identifier: NCT06131983
Recruitment Status : Recruiting
First Posted : November 15, 2023
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive a single dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 2 or 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Condition or disease Intervention/treatment Phase
Facio-Scapulo-Humeral Dystrophy Drug: ARO-DUX4 for Injection Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 52 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Actual Study Start Date : February 26, 2024
Estimated Primary Completion Date : April 2025
Estimated Study Completion Date : June 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ARO-DUX4
ARO-DUX4 for Injection
 Drug: ARO-DUX4 for Injection
single or multiple doses of ARO-DUX4 by intravenous (IV) infusion
Placebo Comparator: Placebo
(0.9%NaCl)
 Drug: Placebo
calculated volume to match active treatment by IV infusion


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS) [ Time Frame: Single dose phase: Up to Day 90; multiple dose phase: Up to Day 360 ]

Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of ARO-DUX4: Maximum Observed Plasma Concentration (Cmax) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  2. PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  3. PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  4. PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time from Zero to Infinity ( [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
    AUCinf)

  5. PK of ARO-DUX4: Terminal Elimination Half-Life (t1/2) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  6. PK of ARO-DUX4: Systemic Clearance (CL) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  7. PK of ARO-DUX4: Volume of Distribution (Vss) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  8. PK of ARO-DUX4: Recovery of Unchanged Drug in Urine Over 0-24 Hours (Amount Excreted: Ae) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  9. PK of ARO-DUX4: Fraction of Drug Excreted in Urine as Percent of Intravenous (IV) Dose (Fe) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]
  10. PK of ARO-DUX4: Renal Clearance (CLr) [ Time Frame: Single dose phase: through 48 hours post-dose, Multiple dose phase: through 24 hours post first and second dose ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record
  • Clinical severity score between 3 and 8 (scale, 0 to 10)
  • Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
  • A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study
  • Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12weeks following the end of study or last dose of study medication, whichever is later.

Exclusion Criteria:

  • Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening
  • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
  • Uncontrolled hypertension
  • Severe cardiovascular disease
  • History of thrombolic eve4nts
  • Platelet count less that the lower limit of normal at Screening
  • History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.
  • Any contraindication to muscle biopsy or MRI

Note: additional inclusion/exclusion criteria may apply per protocol

Contacts and Locations
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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): NCT06131983


Contacts
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Contact: Medical Monitor 626-304-3400 ARO_DUX4@arrowheadpharma.com

Locations
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New Zealand
Research Site Recruiting
Auckland, New Zealand, 1010
Sponsors and Collaborators
Arrowhead Pharmaceuticals
More Information
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Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT06131983    
Other Study ID Numbers: ARODUX4-1001
First Posted: November 15, 2023    Key Record Dates
Last Update Posted: March 15, 2024
Last Verified: March 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
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
Muscular Disorders, Atrophic
Muscular Diseases
 Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn