A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM

Last updated: December 12, 2024
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Amyloidosis

Circulation Disorders

Treatment

ALXN2060

Clinical Study ID

NCT04622046
ALXN2060-TAC-302
  • All Genders

Study Summary

This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with symptomatic ATTR-CM administered on a background of stable heart failure therapy.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTRgenotype.

  2. History of heart failure evidenced by at least 1 prior hospitalization for heartfailure or clinical evidence of heart failure without prior heart failurehospitalization manifested by signs or symptoms of volume overload or elevatedpressures or heart failure symptoms that required or requires ongoing treatment witha diuretic.

  3. New York Heart Association Class I-III symptoms due to ATTR-CM.

  4. On stable doses of cardiovascular medical therapy.

  5. Completed ≥ 150 meters on the 6MWT on 2 tests prior to Day 1.

  6. Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness ≥ 12 millimeters.

  7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic pep (NT-proBNP) level ≥ 300 picograms/milliliter (pg/mL).

Exclusion

Exclusion Criteria:

  1. Acute myocardial infarction, acute coronary syndrome or coronary revascularization,or experienced stroke or transient ischemic attack within 90 days prior toscreening.

  2. Hemodynamic instability at screening.

  3. Likely to undergo heart transplantation within a year of screening.

  4. Current treatment with marketed drug products and other investigational agents forthe treatment of ATTR-CM.

  5. Current treatment with calcium channel blockers with conduction system effects (forexample, verapamil, diltiazem). The use of dihydropyridine calcium channel blockersis allowed.

  6. Confirmed diagnosis of light-chain (AL) amyloidosis.

  7. Biomarkers of myocardial wall stress: NT-ProBNP ≥ 8,500 pg/mL.

  8. Measure of kidney function, estimated glomerular filtration rate by Modification ofDiet in Renal Disease formula < 30 mL/minute/1.73 meters squared.

Study Design

Total Participants: 25
Treatment Group(s): 1
Primary Treatment: ALXN2060
Phase: 3
Study Start date:
November 13, 2020
Estimated Completion Date:
September 23, 2025

Study Description

Participants will receive ALXN2060 for 12 months (Part A). Following the last visit (Month 12) of Part A, participants will continue the study in Part B, which will last for an additional 18 months (30 months from Day 1), during which all participants will continue to receive oral treatment with ALXN2060. Following completion of Month 30 assessments in Part B, participants will be offered the opportunity to continue to receive ALXN2060 in the Extension Period, which will last until ALXN2060 is approved in Japan or for up to 24 additional months, whichever occurs first.

Connect with a study center

  • Clinical Trial Site

    Aichi,
    Japan

    Site Not Available

  • Nagoya University Hospital

    Aichi,
    Japan

    Site Not Available

  • Research Site

    Bunkyo-ku, 113-8431
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukuoka,
    Japan

    Site Not Available

  • Kurume University Hospital

    Fukuoka,
    Japan

    Site Not Available

  • Kyushu University Hospital

    Fukuoka,
    Japan

    Site Not Available

  • Research Site

    Fukuoka-shi, 812-8582
    Japan

    Site Not Available

  • Clinical Trial Site

    Hokkaido,
    Japan

    Site Not Available

  • Sapporo Medical University Hospital

    Hokkaido,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kanagawa,
    Japan

    Site Not Available

  • Kitasato University Hospital

    Kanagawa,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kochi,
    Japan

    Site Not Available

  • Kochi Medical School Hospital

    Kochi,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kumamoto,
    Japan

    Site Not Available

  • Kumamoto University Hospital

    Kumamoto,
    Japan

    Site Not Available

  • Research Site

    Kumamoto-shi, 860-8556
    Japan

    Site Not Available

  • Research Site

    Kurume-shi, 830-0011
    Japan

    Site Not Available

  • Research Site

    Matsumoto-shi, 390-8621
    Japan

    Site Not Available

  • Clinical Trial Site

    Nagano,
    Japan

    Site Not Available

  • Shinshu University Hospital

    Nagano,
    Japan

    Site Not Available

  • Research Site

    Nagoya-shi, 466-8560
    Japan

    Site Not Available

  • Research Site

    Nankoku-shi, 783-8505
    Japan

    Site Not Available

  • Clinical Trial Site

    Okayama,
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama,
    Japan

    Site Not Available

  • Clinical Trial Site

    Osaka,
    Japan

    Site Not Available

  • National Cerebral and Cardiovascular Center

    Osaka,
    Japan

    Site Not Available

  • Research Site

    Sagamihara-shi, 252-0375
    Japan

    Site Not Available

  • Research Site

    Sapporo-shi, 060-8543
    Japan

    Site Not Available

  • Research Site

    Shinjuku-ku, 160-8582
    Japan

    Site Not Available

  • Research Site

    Suita-shi, 564-8565
    Japan

    Site Not Available

  • Clinical Trial Site

    Tokyo,
    Japan

    Site Not Available

  • Juntendo University Hospital

    Tokyo,
    Japan

    Site Not Available

  • Keio University Hospital

    Tokyo,
    Japan

    Site Not Available

  • The University of Tokyo Hospital

    Tokyo,
    Japan

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.