ot available

Phase 1

• Conditions: Familial Amyloid Polyneuropathy; MedDRA version: 20.0Level: LLTClassification code 10057949Term: Familial amyloid polyneuropathySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-004561-13-PT
• Lead Sponsor: Ionis Pharmaceuticals, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-05-07
• Study Completion: 2021-01-07
• Last Update Posted: 5 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 135

Inclusion Criteria

1. Completion of ISIS 420915-CS2 with the following as judged by the Investigator and Sponsor:
a. Satisfactory completion of dosing and end of treatment (EOT) efficacy assessments
b. No significant tolerability issues
c. Satisfactory compliance to the ISIS 420915-CS2 protocol requirements
2. Willingness to take vitamin A supplements
3. Satisfy one of the following:
a. Females: Non-pregnant and non-lactating; surgically sterile, postmenopausal, abstinent, or if engaged in sexual relations of childbearing potential, patient is using an acceptable contraceptive method (refer to Section 6.3.1) from the time of signing the informed consent form until at least 3 months after the last dose of ISIS 420915.
b. Males: Surgically sterile, abstinent, or if engaged in sexual relations with a female of childbearing potential, patient is utilizing an acceptable contraceptive method (refer to Section 6.3.1) during and for 3 months after the last dose of ISIS 420915.
4. Must have given written informed consent (signed and dated) and any authorization required by local law and be able to comply with all study requirements
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 65

Exclusion Criteria

Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified