Trial record 1 of 2 for:
cardio-ttransform
CARDIO-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 Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04136171 |
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Recruitment Status :
Recruiting
First Posted : October 23, 2019
Last Update Posted : March 27, 2023
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Sponsor:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
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Brief Summary:
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Drug: Eplontersen Drug: Placebo | Phase 3 |
This is a multicenter, double-blind study in approximately 1400 participants, who will be randomized to receive subcutaneous (SC) injections of either eplontersen or placebo once every 4 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 1400 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) |
| Actual Study Start Date : | March 13, 2020 |
| Estimated Primary Completion Date : | June 2025 |
| Estimated Study Completion Date : | November 2025 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Transthyretin amyloidosis
| Arm | Intervention/treatment |
|---|---|
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Experimental: Eplontersen
Eplontersen by subcutaneous injection once every 4 weeks
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Drug: Eplontersen
Eplontersen by subcutaneous injection
Other Names:
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Placebo Comparator: Placebo
Eplontersen-matching placebo by subcutaneous injection once every 4 weeks
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Drug: Placebo
Eplontersen-matching placebo by subcutaneous injection |
Primary Outcome Measures :
- Composite Outcome of Cardiovascular (CV) Mortality and Recurrent CV Clinical Events up to Week 140 [ Time Frame: Baseline up to Week 140 ]
Secondary Outcome Measures :
- Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 121 [ Time Frame: Baseline to Week 121 ]The 6MWT is a submaximal exercise test that entails measurement of distance walked over a span of 6 minutes. The 6MWT provides information regarding functional capacity, response to therapy and prognosis across a range of chronic cardiopulmonary conditions.
- Change From Baseline in the Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores at Week 121 [ Time Frame: Baseline to Week 121 ]
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a 23-item self-administered questionnaire developed to independently measure the participant's perception of their health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life (QOL) within a 2-week recall period.
The KCCQ tool quantifies the following six (6) distinct domains and two (2) summary scores. KCCQ responses are provided along a rating scale continuum with equal spacing from worst to best, with a higher score equaling a better score.
- CV Clinical Events up to Week 140 [ Time Frame: Baseline up to Week 140 ]
- CV Mortality up to Week 140 [ Time Frame: Baseline up to Week 140 ]
- All-Cause Mortality up to Week 140 [ Time Frame: Baseline up to Week 140 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
- Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
- Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99m Tc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
- End-diastolic interventricular septum thickness of > 12 millimeters (mm) on Screening echocardiogram
- New York Heart Association (NYHA) class I-III
Exclusion Criteria:
- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
- Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
- Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For participants with chronic kidney disease (CKD) and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits
- Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
- Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or ribonucleic acid (RNA) therapeutic (including small interfering ribonucleic acid [siRNA]; does not apply to COVID-19 mitochondrial [mRNA] vaccinations)
- Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or non-dihydropyridine calcium-channel blocker (e.g., verapamil, diltiazem). Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04136171
Contacts
| Contact: Ionis Pharmaceuticals | (844) 413-0219 | ionisNCT04136171study@clinicaltrialmedia.com |
Locations
Show 144 study locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Additional Information:
| Responsible Party: | Ionis Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT04136171 |
| Other Study ID Numbers: |
ION-682884-CS2 2019-002835-27 ( EudraCT Number ) |
| First Posted: | October 23, 2019 Key Record Dates |
| Last Update Posted: | March 27, 2023 |
| Last Verified: | March 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | Yes |
Keywords provided by Ionis Pharmaceuticals, Inc.:
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TTR Amyloidosis Cardiomyopathy |
Additional relevant MeSH terms:
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Cardiomyopathies Amyloidosis Heart Diseases |
Cardiovascular Diseases Proteostasis Deficiencies Metabolic Diseases |