Detail

Basic Information Clinical Results Relationship Graph

Gene symbol	TTR	Synonyms	ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN	Type of gene	protein-coding
Chromosome	18	Map location	18q12.1	dbXrefs
Gene perturbation-related omics dataset	PerturbAtlas
Description	transthyretin

GTO ID	GTC0458
Trial ID	NCT03728634
Disease	Amyloidosis
Altered gene	TTR
Therapeutic/Target gene	Target gene
Therapy	ASO
Treatment	AKCEA-TTR-LRx\|Eplontersen\|ION-682884\|IONIS-TTR-LRx\|ION-682884\|Wainua
Co-treatment	Vitamin A
Location approved	US
Phase	Phase1\|Phase2
Recruitment status	Completed
Title	This Was a Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study Conducted at a Single Center for the Healthy Volunteer Cohorts in up to 56 Participants. It Consisted of 1 Single-dose Cohort and 3 Multiple-dose Cohorts (n = 12 Per Cohort, 10 Active:2 Placebo). The Open-label, hATTR Patient Cohort Portion of the Study Was Conducted at Multiple Centers.
Year	2018
Country	Canada
Company sponsor	Ionis Pharmaceuticals, Inc.
Other ID(s)	ION-682884-CS1

Vector information

Vector	No vector was used

Cohort1: Placebo_multiple Dose

Administration route	subcutaneous injection\|oral
Dosage	Placebo, Q4W (total of 4 doses), 13-week
Pts	6
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:3 50.0%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:0.0\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

Cohort2: ION-682884_dose level 1

Administration route	subcutaneous injection\|oral
Dosage	ION-682884, 45 mg, Q4W (total of 4 doses), 13-week
Pts	10
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:1 10.0%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:0.0\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

Cohort3: ION-682884_dose level 2

Administration route	subcutaneous injection\|oral
Dosage	ION-682884, 60 mg, Q4W (total of 4 doses), 13-week
Pts	10
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:3 30.0%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:30.0\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

Cohort4: ION-682884_dose level 3

Administration route	subcutaneous injection\|oral
Dosage	ION-682884, 90 mg, Q4W (total of 4 doses), 13-week
Pts	10
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:7 70.0%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:0.0\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

Cohort5: Placebo_single dose

Administration route	subcutaneous injection\|oral
Dosage	placebo, single dose, Day 1
Pts	2
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:1 50.0%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:0.0\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

Cohort6: ION-682884_dose level 4

Administration route	subcutaneous injection\|oral
Dosage	ION-682884, 120 mg, single dose, Day 1
Pts	9
Age	Adult, Older_Adult
Outcome	Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE):Measure Type: Count of Participants:4 44.4%\|Percentage of Participants Using Concomitant Medications:Measure Type: Number:22.2\|Number of Participants With Clinically Significant Laboratory Values:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Physical Examination Findings:Measure Type: Count of Participants:0 0.0%\|Number of Participants With Clinically Significant Electrocardiogram (ECG) Values:Measure Type: Count of Participants:0 0.0%
Adverse reactions	No serious clinical adverse events
References	PMID: 33283485

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