Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Conditions
Keywords
ALXN2220, Cliramitug, NI006, Transthyretin Amyloid Cardiomyopathy, ATTR-CM, Open-Label Extension Study
Brief summary
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study ALXN2220-ATTRCM-301.
Interventions
DRUGALXN2220
To be administered as an IV infusion
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
Study 302 is a single arm, OLE study. All participants will receive open-label ALXN2220 upon entry into the OLE study. No IMP masking or blinding procedures will be implemented or maintained during the OLE study.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Inclusion Criteria * Type of Participant: Participants who have completed the Blinded Treatment Period (including the PC visit) of Study 301. * Sex and Contraceptive/Barrier Requirements: Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement). * Informed Consent: Participant, or legally authorized representative where relevant and allowed by law, is willing and able to comply with all study requirements and to provide signed informed consent. *
Exclusion criteria
* Participants who permanently discontinued study intervention in Study 301 * Participants who withdrew full consent during Study 301. * Medical Conditions: Suspected or known intolerance/allergy to proteins or any components of ALXN2220. Has had a heart transplant or permanent CMAD implantation. Has any new or worsening of existing medical or psychological condition or risk factor that in the opinion of the Investigator or Sponsor might jeopardize participant's safety or compliance with the protocol, or pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study. * Prior/Concurrent/Future Clinical Study Experience: Current or future participation in another investigational clinical study or intake of investigational drug within 30 calendar days or 5 half-lives of the study intervention, whichever is longer, before signing the ICF, except for study intervention used in Study 301.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants with TEAEs | Up to 24 months | To evaluate the long-term safety and tolerability of ALXN2220 in participants with ATTR-CM |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Total occurrence of ACM and CV clinical events | Up to 24 months | To evaluate the long-term efficacy of ALXN2220 on ACM and CV clinical events |
| Total occurrence of CV clinical events | Up to 24 months | To evaluate the long-term efficacy of ALXN2220 on CV clinical events |
| Time to ACM | Up to 24 months | To evaluate the long-term efficacy of ALXN2220 on ACM |
| Time to first CV clinical event or ACM | Up to 24 months | To evaluate the long-term efficacy of ALXN2220 on first CV clinical event or ACM |
| Time to CVM | Up to 24 months | To evaluate the long-term efficacy of ALXN2220 on CVM |
| Change from baseline in KCCQ-OS by visit | Up to 24 months | To assess the long-term efficacy of ALXN2220 on symptoms, functionality, and health-related QoL. |
| Change from baseline in 6MWT by visit | Up to 24 months | To assess the long-term efficacy of ALXN2220 on functional capacity |
| ADA incidence, response categories, and titer | Up to 24 months | To assess long-term immunogenicity to ALXN2220 |
| NAb incidence and response categories | Up to 24 months | To assess long-term immunogenicity to ALXN2220 |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Czechia, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Japan, Netherlands, Norway, Poland, South Korea, Spain, Sweden, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc., Sponsor
Outcome results
None listed