Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ION-682884 · 1 trial · 2 indications
An adverse event (AE) is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not the AE was considered related to the medicinal (investigational) product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of study drug and subsequently worsened or was not present prior to receiving the first dose of study drug but subsequently appeared.
A concomitant therapy was any non-protocol-specified drug or substance (including over-the-counter \[OTC\] medications, herbal medications, and vitamin supplements) administered between signing of informed consent and the last study visit.
Laboratory parameters included measurement of blood chemistry, hematology, coagulation, complement, or urinalysis parameters for the single-dose and multiple-dose cohorts. Number of participants with clinically significant values in laboratory based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
Physical examination included measurement of height and weight for body mass index (BMI) determination. Number of participants with clinically significant findings in physical examination based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
ECG measurements included assessment of ventricular rate (VR), PR interval, QR interval, QT interval, QT corrected using Fridericia's formula (QTcF), and QT corrected using Bazett's formula (QTcB). Number of participants with clinically significant values in electrocardiogram based on Investigator's assessment are reported. Any value outside the normal range was to be flagged for the attention of the investigator was to assess whether or not a flagged value is of clinical significance.
| Arm | Type | Description |
|---|---|---|
| Multiple Dose Cohort: Placebo | PLACEBO_COMPARATOR | Participants received ION-682884 matching placebo, subcutaneously (SC) once every 4 weeks \[Q4W\] (total of 4 doses) along with daily oral supplemental doses of the recommended daily allowance (RDA) of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort A: ION-682884 45 mg | EXPERIMENTAL | Participants received ION-682884, 45 milligrams (mg), SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort E: ION-682884 60 mg | EXPERIMENTAL | Participants received ION-682884, 60 mg, SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Multiple Dose Cohort B: ION-682884 90 mg | EXPERIMENTAL | Participants received ION-682884, 90 mg, SC, Q4W (total of 4 doses) along with daily oral supplemental doses of the RDA of vitamin A during the 13-week Treatment Period. |
| Single Dose Cohort: Placebo | PLACEBO_COMPARATOR | Participants received single dose of ION-682884 matching placebo, SC along with daily oral supplemental doses of the RDA of vitamin A on Day 1. |
| Single Dose Cohort C: ION-682884 120 mg | EXPERIMENTAL | Participants received single dose of ION-682884, 120 mg, SC along with daily oral supplemental doses of the RDA of vitamin A on Day 1. |
| Name | Type | Description |
|---|---|---|
| ION-682884 | DRUG | ION-682884 administered SC |
| Placebo | DRUG | Placebo comparator calculated volume to match ION-682884 administered SC |
| Vitamin A | DIETARY_SUPPLEMENT | Oral supplement |
Inclusion Criteria for Healthy Volunteers (Cohorts A, B, C, and E) 1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal 2. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the su...
ION-682884 is an investigational small molecule being developed by Ionis Pharmaceuticals for rare disease. It is currently in Phase 1 clinical development. The drug has been studied in healthy volunteers to evaluate its safety, tolerability, and pharmacokinetic and pharmacodynamic profiles.
ION-682884 is being studied for use in healthy volunteers as part of early clinical development. The completed Phase 1 trial also included patients with hereditary transthyretin-mediated amyloidosis (hATTR), though the drug's specific intended indication has not been established beyond this investigational context.
ION-682884 is developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker IONS. Ionis is conducting the clinical development of this investigational drug candidate.
ION-682884 is in Phase 1 clinical development. The drug is investigational and has not been approved by regulatory authorities. One Phase 1 trial has been completed, and no active trials are currently listed for this candidate.
ION-682884 has been studied in one completed Phase 1 trial, NCT03728634. This randomized, double-blind, placebo-controlled study evaluated single and multiple doses of the drug administered subcutaneously to healthy volunteers and patients with hATTR amyloidosis. The trial enrolled 47 participants in Canada.
Yes, ION-682884 is the same as eplontersen. The Phase 1 trial NCT03728634 evaluated eplontersen, which is the alternative name for ION-682884. The study assessed the safety, tolerability, and pharmacokinetic and pharmacodynamic profiles of this drug candidate.