CompletedPhase 3

NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

United States168 participantsStarted 2019-12-11

Plain-language summary

The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). For more information, please visit http://www.neuro-ttransform.com/.

Who can participate

Age range18 Years – 82 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aged 18 to 82 years at the time of informed consent
✓. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
✓. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participantss non-pregnant female partner must be using a highly effective contraceptive method
✓. Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:

Exclusion criteria

✕. Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a participants unsuitable for inclusion, including but not limited to abnormal safety labs
✕. Karnofsky performance status ≤ 50
✕. Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
✕. Prior liver transplant or anticipated liver transplant within 1-yr of Screening
✕. New York Heart Association (NYHA) functional classification of ≥ 3
✕. Acute coronary syndrome within 6 months of screening or major surgery within 3 months of Screening
✕. Other types of amyloidosis

What they're measuring

Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66

Timeframe: Baseline, Week 66

Change From Baseline in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66

Timeframe: Baseline, Week 66

Percent Change From Baseline in Serum TTR Concentration at Week 65

Timeframe: Baseline, Week 65

Percent Change From Baseline in Serum TTR Concentration at Week 35

Timeframe: Baseline, Week 35

Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 35

Timeframe: Baseline, Week 35

Trial details

NCT IDNCT04136184

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-04-11

Results submitted2024-09-10

View on ClinicalTrials.gov More Hereditary Transthyretin-Mediated Amyloid Polyneuropathy trials

Plain-language summary

Who can participate

Age range18 Years – 82 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aged 18 to 82 years at the time of informed consent
✓. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
✓. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participantss non-pregnant female partner must be using a highly effective contraceptive method
✓. Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:

Exclusion criteria

✕. Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a participants unsuitable for inclusion, including but not limited to abnormal safety labs
✕. Karnofsky performance status ≤ 50
✕. Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
✕. Prior liver transplant or anticipated liver transplant within 1-yr of Screening
✕. New York Heart Association (NYHA) functional classification of ≥ 3
✕. Acute coronary syndrome within 6 months of screening or major surgery within 3 months of Screening
✕. Other types of amyloidosis

What they're measuring

Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66

Timeframe: Baseline, Week 66

Change From Baseline in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66

Timeframe: Baseline, Week 66

Percent Change From Baseline in Serum TTR Concentration at Week 65

Timeframe: Baseline, Week 65

Percent Change From Baseline in Serum TTR Concentration at Week 35

Timeframe: Baseline, Week 35

Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 35

Timeframe: Baseline, Week 35