CPVT ARM210 Trial

Phase 2 Trial of ARM210 for the Treatment of CPVT

ARMGO recently announced the enrollment of the first patient in a Phase 2 proof of concept clinical trial using its Rycal ARM210 (also known as S48168) for the treatment of CPVT.

The trial, performed at the Amsterdam University Medical Center (Amsterdam UMC), Netherlands and the Mayo Clinic, Rochester MN, USA will investigate the safety and efficacy of ARM210 in CPVT.

ARM210 is a potential disease modifying therapy for CPVT as it repairs leaky RyR2 channels. By binding and stabilizing the leaky channel, ARM210 can restore normal function, as demonstrated in animal models and in high resolution structures of mutant RyR2.

Eligibility Criteria

Participants must meet all the following conditions to be eligible for enrollment into the study:

  1. Participant must be 18 to 65 years of age inclusive, at the time of signing the informed consent.
  2. Participants who are willing and able to comply with scheduled visits, study drug administration plan, study restrictions, and study procedures.
  3. Participants have a confirmed genetic diagnosis of CPVT1 and supporting clinical phenotype, including residual ventricular ectopy (a complexity score ≥ 2; requiring at minimum the presence of PVCs in bigeminy on exercise stress test) on a stable (at least 1 month) standard-of-care, CPVT1-directed treatment regimen as decided by their CPVT treating physician.
  4. Have a body mass index (BMI) ≤ 36 kg/m2 (inclusive) at screening.
  5. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
  6. Daily use of medicines and dietary supplements need to be approved by the PI and Sponsor, or a drug/supplement-dependent wash-out prior to inclusion.
  7. Male participants agree to not donate sperm from the first day of dosing of study drug until 5 half-lives plus 90 days (approximately 94 days) after the last dose of study drug.
  8. Female participants are eligible to participate if she is not pregnant or breastfeeding, and uses one of the following highly effective birth control methods (from the first dose until 5 half-lives plus 90 days (approximately 94 days):
    • Prescribed hormonal oral contraceptives, vaginal ring, or transdermal patch.
    • Intrauterine device (IUD).
    • Intrauterine hormone-releasing system (IUS).
    • Depot/implantable hormone (e.g., Depo-Provera®, Implanon).
    • Bilateral tubal occlusion/ligation.
    • Sexual abstinence.
    • Refraining from heterosexual intercourse during the entire period of risk associated with the study requirements.
    • If the participant decides to become sexually active during the study, then one of the highly effective birth control methods must be used.

OR Is a woman of non-childbearing potential; defined by at least 1 of the following criteria:

    • Postmenopausal defined as 12 months of spontaneous amenorrhea without a medical cause and follicle stimulating hormone (FSH) serum level > 40 mIU/mL without the use of hormonal supplementation. Appropriated documentation of FSH levels is required.
    • Surgically sterile by hysterectomy and/or bilateral oophorectomy with appropriate documentation of surgical procedure.
      Has a congenital condition resulting in no uterus.
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