Transthyretin Amyloid CardiopathyPhase 2NCT04843020
ION-682884 in Patients With TTR Amyloid Cardiomyopathy
A Phase 2 study testing ION 682884 in people with Transthyretin Amyloid Cardiopathy, run by Brigham and Women's Hospital at 1 site in United States.
Trial summary
Transthyretin is a protein produced in the liver that transports thyroid hormone and vitamin A. A single substitution of an amino acid in the structure of TTR can result in a relatively unstable protein, the breakdown products of which (predominantly monomers) aggregate abnormally and produce proteinaceous deposits in… Read more about this study
- Who is it for?
- Ages 65 to 85 years
- Conditions the study is for
- Transthyretin Amyloid Cardiopathy
- What is being tested?
- ION 682884
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2021to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 65 to 85 years
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- 1. Only patient who have completed 24 months of therapy in the open label clinical trial of inotersen, "A 24-month open-label study of the tolerability and efficacy of an antisense oligonucleotide (inotersen) in patients with transthyretin (TTR) amyloid cardiomyopathy" (Protocol #:2018-P001436) will be enrolled. All patients in that study had either wild-type transthyretin amyloidosis (ATTRwt) or mutant transthyretin amyloidosis (ATTRm) cardiac amyloidosis, defined by standard criteria. Additional criteria for the current study are as follows:
- Patients should, in the opinion of the Investigator, be in a stable state in terms of New York Heart Association (NYHA) class. Class I-III patients will be recruited.
- Age 65-85 years
- Male, or non-pregnant, non-lactating females. If a male partners with a premenopausal woman, he must be willing to use the following methods of contraception: condoms, oral/hormonal contraception, Intrauterine Device, diaphragm, or abstinence (all patients are either male of post-menopausal) (NB: There will be no premenopausal women in the proposed study)
- Written informed consent to be obtained prior to study treatment
- Willingness to return to the treating center for follow-up
- Willingness and ability to self-administer, or to have spouse administer subcutaneous injections of study drug every 4 weeks.
- Willingness to take oral Vitamin A supplementation throughout the study and for 3 months thereafter.
Who cannot join
- 1. Acute coronary syndrome, unstable angina, stroke, transient ischemic attack,, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 emergency room for worsening of HF with discharge date within 4 weeks prior to or during Screening 3. Uncontrolled hypertension (systolic blood pressure [BP] > 160 or diastolic BP > 100 mmHg) 4. Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment (e.g., no pacemaker, although indicated) 5. Severe uncorrected cardiac valvular disease 6. Cardiomyopathy not primarily caused by amyloidosis, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease 7. Screening laboratory results as follows, or any other clinically significant abnormalities in Screening laboratory values that would render a patient unsuitable for inclusion
- Alanine aminotransferase/aspartate aminotransferase (ALT/AST) > 2.0 × upper limit of normal (ULN)
- Total bilirubin ≥ 1.5 × ULN (patients with total bilirubin ≥ 1.5 × ULN may be allowed on study if indirect bilirubin only is elevated, ALT/AST is not greater than the ULN and known to have Gilbert's disease)
- Platelets < 125 × 109/L
- Urine protein creatinine ratio (UPCR) ≥ 750 mg/g. In the event of UPCR above this threshold ineligibility may be confirmed by a repeat random spot UPCR ≥ 750 mg/g
- Positive test for blood (including trace) on urinalysis that is subsequently confirmed with urine microscopy showing > 5 red blood cells per high power field and is related to glomerulopathies. In women, this exclusion criterion must be assessed outside of menstrual period. If in the opinion of the Investigator the hematuria is not considered related to glomerulopathies the patient may be considered eligible, pending proper follow-up and a discussion with the Medical Monitor. Patients with history of bladder cancer must have been treated with curative intent and have not presented recurrence within the prior 5 years
Show all 11Show fewer
- Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 at Screening. If the eGFR is thought to be underestimated, the CKD-EPI creatinine-cystatin C equation can be used for confirmation.
- Abnormal thyroid function tests with clinical significance per Investigator judgement
- Hemoglobin A1c (HbA1c) > 9.5% 8. 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 patients with CKD and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25.
- 9. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1 10. Known history of or positive test for human immunodeficiency virus (HIV) (as evidenced by positive tests for HIV antibody and HIV RNA), hepatitis C (as evidenced by positive tests for HCV antibody and HCV RNA) or hepatitis B (as evidenced by a positive test for hepatitis B surface antigen) 11. History of bleeding, diathesis or coagulopathy (e.g., liver cirrhosis, hematologic malignancy, antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease) 12. If receiving oral anticoagulants (except vitamin K antagonists), the dose must have been stable for 4 weeks prior to the first dose of Study Drug and regular monitoring must be performed, per clinical practice during the study. If the patient is receiving vitamin K antagonists (e.g., warfarin) INR should be in therapeutic range, as established by the Investigator, for 4 weeks prior to the first dose 13. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin, melanoma in situ, prostate carcinoma grade group 1, breast ductal carcinoma in situ, or carcinoma in situ of the cervix successfully treated. Patients with a history of other malignancies who have been treated with curative intent and without recurrence within 5 years may also be eligible per Investigator judgment 14. Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization 15. Karnofsky performance status of ≤ 50% 16. Contraindication for immunosuppressive therapy, per Investigator's discretion 17. Known Light chain/Primary Amyloidosis (AL) 18. Known leptomeningeal amyloidos
- -
Trial locations and contacts
1 site in 1 country.
United States1 site
Brigham and Women's Hospital
Boston, Massachusetts, 02115
About this study
Official title: An Open-Label Study of ION-682884 in Patients With TTR Amyloidosis Who Have Completed a 24-Open Label Study of Inotersen for TTR Amyloidosis Cardiomyopathy
Transthyretin is a protein produced in the liver that transports thyroid hormone and vitamin A. A single substitution of an amino acid in the structure of TTR can result in a relatively unstable protein, the breakdown products of which (predominantly monomers) aggregate abnormally and produce proteinaceous deposits in nerves and the heart. These deposits are known as amyloid and produce progressive nerve and heart damage. Amyloidosis due to a mutant TTR is usually an autosomal dominant and hence is a familial condition. Wild-type TTR is also capable of producing amyloid deposits which predominantly involves the heart (rather than the nervous system) resulting in a progressive decrease in cardiac function with increasing signs of heart failure. This study aims to determine whether subcutaneous injection of an antisense oligonucleotide drug, known as ION-682884, that has been specifically designed to reduce production of the protein transthyretin by the liver, can slow or stop the progression of TTR amyloid cardiomyopathy as compared to historical controls, using advanced echocardiography and cardiac MRI. This study drug will only be administered to patients who have completed a 24-month study of a similar drug, inotersen (clinicaltrials.gov identifier NCT037028289).The study also aims to determine the tolerability and safety of this drug when administered over a 36+-month period to patients with TTR amyloid cardiomyopathy. The study duration is open-ended and will continue either until this agent is approved by the FDA, or production is discontinued based on results of ongoing double-blinded studies.
Read the detailed description
The study is limited to those patients who have completed 24 months in our single-center open-label trial of inotersen for amyloid cardiomyopathy (NCT037028289). Patients must have an estimated glomerular filtration rate of greater than 30 and a platelet count greater than 130,000. They will have either wildtype or mutant transthyretin cardiomyopathy. It is anticipated that up to 17 patients will be eligible for the study, age 65-85 years.
Research design:
After completion of the 24 months in the inotersen study, patients will undergo their final visit and assessment. This will include an echocardiogram, lab work, 6-minute walk test, cardiopulmonary exercise testing and, in those without a pacemaker, a cardiac MRI. If the lab work from the end of study confirms eligibility for the new study, patients will be offered the option of continuing with the TTR-lowering drug, ION-682884. After informed consent the patient will be supplied with prefilled syringes containing ION-682884 and the first injection administered. They will return for follow up visits at six weeks, 12 weeks, three months, and six months thereafter. At each six-monthly visit they will undergo the same testing as they had in the inotersen study, namely laboratory measurements, 6-minute walk test, cardiopulmonary exercise testing, echocardiogram and, unless contraindicated, cardiac MRI. The patients will remain in the study for an open-ended period of time until study ending criteria occur (reduction in the glomerular filtration rate to less than 30 or platelet count with a significant fall) or until the drug is FDA-approved or the manufacturer or principal investigator terminates the study.
Because ION-682884 is an antisense oligonucleotide similar to inotersen and because inotersen has been associated with thrombocytopenia and worsening renal function, patients will have weekly lab draws done in their own home for safety monitoring. These bloods will be used to measure platelet function every week, and renal function every other week. This is currently mandated by the Food and Drug administration in the double-blinded study of ION-682884 but the frequency of blood draws may be decreased overtime depending on the interim safety analysis results from that study and FDA evaluation of that data.
- Conditions
- Transthyretin Amyloid Cardiopathy
- Being tested
- ION 682884
- Keywords
- amyloidosis, transthyretin, antisense oligonucleotide, ION682884, cardiomyopathy
- Medical subject headings
- Amyloidosis, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ION 682884Drug
Subcutaneous injection every month
| Study group | Role | What they receive |
|---|---|---|
| Drug subcutaneous injection | Experimental | Monthly injection of ION 682884, administered subcutaneously at a dose of 45 mg. Receives: Drug: ION 682884 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Echocardiographic change
change in echocardiographically-derived left ventricular longitudinal strain (measured as percentage change from end diastole to end systole)
Measured: 48 months
Change in cardiac MRI
Change in extracellular volume (measure as a percentage of total of LV mass) between baseline MRI and 48 months
Measured: 48 months
Change in cardiac MRI
Change in left ventricular mass, measured by cardiac MRI (gm/m2) , between baseline and 48 months
Measured: 48 months
change in 6 minute walk
change in distance walked (measured in meters) between baseline 6 minute walk and walk after 48 months of therapy
Measured: 48 months
change in cardiopulmonary testing
change in maximal oxygen consumption (VO2 max measured in ml/kg.min) between baseline cardiopulmonary testing and cardiopulmonary testing at 48 months of therapy
Measured: 48 months
change in the cardiac biomarker NTproBNP
change between baseline and after 48 months of therapy of serum NTproBNP (measured in pg/ml)
Measured: 48 months
change in the cardiac biomarker high-sensitivity troponin
change between baseline and after 48 months of therapy of serum high-sensitivity troponin T measured in ng/L)
Measured: 48 months
Secondary outcomes (4)
Response of transthyretin levels to therapy
Absolute decrease in TTR levels (measured in mg/dl) after initiation of therapy, compared to response, in previous study, to weekly inotersen injections.
Measured: 3 months
Time frame of response of TTR levels to therapy
Time frame to nadir of TTR levels (weeks) after initiation of therapy, compared to response, in previous study, to weekly inotersen injections.
Measured: 3 months
Side-effect profile: renal function
Effect of therapy on renal function, measured as estimated glomerular filtration rate (mL/min/1.73m2), between baseline measurements and 48 months, measured every 2 weeks
Measured: 48 months
Side effect profile: platelets
Effect of therapy on platelet count (normal 150,000 - 450,000/uL), between baseline measurements and 48 months, measured every week
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2021
- Study start1 Jun 2021 (estimated)
- Primary completion30 May 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Why it stopped: no subjects available
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin Amyloid CardiopathyStudies of the same condition, open and under way.
- More trials run by Brigham and Women's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- open label study of investigational antisense oligonucleotide targeting TTR
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rodney H. Falk, MD, Brigham and Women's Hospital
- Investigators
- Rodney H Falk, Study director, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04843020
- Sponsor's protocol ID
- 2021P000784
- Status last verified
- Feb 2026
- First submitted
- 5 Apr 2021
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study of ION-682884 in Patients With TTR Amyloidosis Who Have Completed a 24-Open Label Study of Inotersen for TTR Amyloidosis Cardiomyopathy
- ClinicalTrials.gov ID
- NCT04843020
- Acronym / sponsor ID
- 2021P000784
- Conditions
- Transthyretin Amyloid Cardiopathy
- Keywords
- amyloidosis, transthyretin, antisense oligonucleotide, ION682884, cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no subjects available
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Study start
- 1 Jun 2021 (estimated)
- Primary completion
- 30 May 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 13 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Transthyretin is a protein produced in the liver that transports thyroid hormone and vitamin A. A single substitution of an amino acid in the structure of TTR can result in a relatively unstable protein, the breakdown products of which (predominantly monomers) aggregate abnormally and produce proteinaceous deposits in nerves and the heart. These deposits are known as amyloid and produce progressive nerve and heart damage. Amyloidosis due to a mutant TTR is usually an autosomal dominant and hence is a familial condition. Wild-type TTR is also capable of producing amyloid deposits which predominantly involves the heart (rather than the nervous system) resulting in a progressive decrease in cardiac function with increasing signs of heart failure. This study aims to determine whether subcutaneous injection of an antisense oligonucleotide drug, known as ION-682884, that has been specifically designed to reduce production of the protein transthyretin by the liver, can slow or stop the progression of TTR amyloid cardiomyopathy as compared to historical controls, using advanced echocardiography and cardiac MRI. This study drug will only be administered to patients who have completed a 24-month study of a similar drug, inotersen (clinicaltrials.gov identifier NCT037028289).The study also aims to determine the tolerability and safety of this drug when administered over a 36+-month period to patients with TTR amyloid cardiomyopathy. The study duration is open-ended and will continue either until this agent is approved by the FDA, or production is discontinued based on results of ongoing double-blinded studies.
Detailed description
The study is limited to those patients who have completed 24 months in our single-center open-label trial of inotersen for amyloid cardiomyopathy (NCT037028289). Patients must have an estimated glomerular filtration rate of greater than 30 and a platelet count greater than 130,000. They will have either wildtype or mutant transthyretin cardiomyopathy. It is anticipated that up to 17 patients will be eligible for the study, age 65-85 years.
Research design:
After completion of the 24 months in the inotersen study, patients will undergo their final visit and assessment. This will include an echocardiogram, lab work, 6-minute walk test, cardiopulmonary exercise testing and, in those without a pacemaker, a cardiac MRI. If the lab work from the end of study confirms eligibility for the new study, patients will be offered the option of continuing with the TTR-lowering drug, ION-682884. After informed consent the patient will be supplied with prefilled syringes containing ION-682884 and the first injection administered. They will return for follow up visits at six weeks, 12 weeks, three months, and six months thereafter. At each six-monthly visit they will undergo the same testing as they had in the inotersen study, namely laboratory measurements, 6-minute walk test, cardiopulmonary exercise testing, echocardiogram and, unless contraindicated, cardiac MRI. The patients will remain in the study for an open-ended period of time until study ending criteria occur (reduction in the glomerular filtration rate to less than 30 or platelet count with a significant fall) or until the drug is FDA-approved or the manufacturer or principal investigator terminates the study.
Because ION-682884 is an antisense oligonucleotide similar to inotersen and because inotersen has been associated with thrombocytopenia and worsening renal function, patients will have weekly lab draws done in their own home for safety monitoring. These bloods will be used to measure platelet function every week, and renal function every other week. This is currently mandated by the Food and Drug administration in the double-blinded study of ION-682884 but the frequency of blood draws may be decreased overtime depending on the interim safety analysis results from that study and FDA evaluation of that data.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- open label study of investigational antisense oligonucleotide targeting TTR
| Arm | Type | Receives |
|---|---|---|
| Drug subcutaneous injection | Experimental | Drug: ION 682884Monthly injection of ION 682884, administered subcutaneously at a dose of 45 mg. |
Who may qualify
- Ages
- 65 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (8 listed)
- 1. Only patient who have completed 24 months of therapy in the open label clinical trial of inotersen, "A 24-month open-label study of the tolerability and efficacy of an antisense oligonucleotide (inotersen) in patients with transthyretin (TTR) amyloid cardiomyopathy" (Protocol #:2018-P001436) will be enrolled. All patients in that study had either wild-type transthyretin amyloidosis (ATTRwt) or mutant transthyretin amyloidosis (ATTRm) cardiac amyloidosis, defined by standard criteria. Additional criteria for the current study are as follows:
- Patients should, in the opinion of the Investigator, be in a stable state in terms of New York Heart Association (NYHA) class. Class I-III patients will be recruited.
- Age 65-85 years
- Male, or non-pregnant, non-lactating females. If a male partners with a premenopausal woman, he must be willing to use the following methods of contraception: condoms, oral/hormonal contraception, Intrauterine Device, diaphragm, or abstinence (all patients are either male of post-menopausal) (NB: There will be no premenopausal women in the proposed study)
- Written informed consent to be obtained prior to study treatment
Key exclusion criteria (11 listed)
- 1. Acute coronary syndrome, unstable angina, stroke, transient ischemic attack,, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 emergency room for worsening of HF with discharge date within 4 weeks prior to or during Screening 3. Uncontrolled hypertension (systolic blood pressure [BP] > 160 or diastolic BP > 100 mmHg) 4. Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment (e.g., no pacemaker, although indicated) 5. Severe uncorrected cardiac valvular disease 6. Cardiomyopathy not primarily caused by amyloidosis, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease 7. Screening laboratory results as follows, or any other clinically significant abnormalities in Screening laboratory values that would render a patient unsuitable for inclusion
- Alanine aminotransferase/aspartate aminotransferase (ALT/AST) > 2.0 × upper limit of normal (ULN)
- Total bilirubin ≥ 1.5 × ULN (patients with total bilirubin ≥ 1.5 × ULN may be allowed on study if indirect bilirubin only is elevated, ALT/AST is not greater than the ULN and known to have Gilbert's disease)
- Platelets < 125 × 109/L
- Urine protein creatinine ratio (UPCR) ≥ 750 mg/g. In the event of UPCR above this threshold ineligibility may be confirmed by a repeat random spot UPCR ≥ 750 mg/g
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ION 682884Subcutaneous injection every month | Drug | Drug subcutaneous injection |
Primary outcome
Echocardiographic change
Time frame: 48 months
change in echocardiographically-derived left ventricular longitudinal strain (measured as percentage change from end diastole to end systole)
Change in cardiac MRI
Time frame: 48 months
Change in extracellular volume (measure as a percentage of total of LV mass) between baseline MRI and 48 months
Change in cardiac MRI
Time frame: 48 months
Change in left ventricular mass, measured by cardiac MRI (gm/m2) , between baseline and 48 months
change in 6 minute walk
Time frame: 48 months
change in distance walked (measured in meters) between baseline 6 minute walk and walk after 48 months of therapy
change in cardiopulmonary testing
Time frame: 48 months
change in maximal oxygen consumption (VO2 max measured in ml/kg.min) between baseline cardiopulmonary testing and cardiopulmonary testing at 48 months of therapy
change in the cardiac biomarker NTproBNP
Time frame: 48 months
change between baseline and after 48 months of therapy of serum NTproBNP (measured in pg/ml)
change in the cardiac biomarker high-sensitivity troponin
Time frame: 48 months
change between baseline and after 48 months of therapy of serum high-sensitivity troponin T measured in ng/L)
4 secondary outcomes
Response of transthyretin levels to therapy
Time frame: 3 months
Time frame of response of TTR levels to therapy
Time frame: 3 months
Side-effect profile: renal function
Time frame: 48 months
Side effect profile: platelets
Time frame: 48 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rodney H Falk study director
Brigham and Women's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ION 682884 in people with Transthyretin Amyloid Cardiopathy, run by Brigham and Women's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Echocardiographic change (time frame: 48 months). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.