Treatment for Heart Diseases

Phase-Based Progress Estimates
3
Effectiveness
3
Safety
Weill Cornell Medicine, New York, NY
Heart Diseases+2 More
Eligibility
65+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether N-of-1 trials are feasible for deprescribing beta-blockers in patients with cardiac amyloidosis.

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Eligible Conditions

  • Heart Diseases
  • Cardiac Amyloidosis

Treatment Effectiveness

Effectiveness Progress

3 of 3
This is further along than 93% of similar trials

Other trials for Heart Diseases

Study Objectives

This trial is evaluating whether Treatment will improve 2 primary outcomes and 5 secondary outcomes in patients with Heart Diseases. Measurement will happen over the course of End of intervention Period 1 (Weeks 2, 3, 4, 5, or 6) and Period 2 (Weeks 8, 9, 10, 11, or 12).

Month 12
Change in patient-reported health status when on beta-blocker versus when off beta-blocker, as measured by Kansas City Cardiomyopathy Questionnaire (KCCQ-12)
Week 20
Change in patient-reported health when on beta-blocker versus when off beta-blocker, as measured by the EuroQol-5D Visual Analogue System (EQ-5D VAS)
Week 20
Change in lower extremity function when on beta-blocker and off beta-blocker, as measured by a Short Physical Performance Battery
Month 12
Change in patient-reported sexual function when on beta-blocker versus when off beta-blocker, as measured by Patient-Reported Outcome Measurement Information System-Sexual Function (PROMIS-Sexual Function)
Month 12
Change in patient-reported cognitive function when on beta-blocker versus when off beta-blocker, as measured by Patient-Reported Outcome Measurement Information System-Short Form 6a (PROMIS SF-6a)
Change in patient-reported quality of life when on beta-blocker, as measured by the Patient-Reported Outcome Measurement Information System-29 (PROMIS-29)
Week 8
Change in exercise capacity when on beta-blocker and off-beta blocker, as measured by peak oxygen consumption (VO2) during cardiopulmonary exercise test (CPET)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Heart Diseases

Trial Design

0 Treatment Group

This trial requires 16 total participants across 0 different treatment group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline; end of intervention period 1 (weeks 2, 3, 4, 5, or 6), period 2 (weeks 8, 9, 10, 11, or 12), period 3 (weeks 14, 15, 16,17, or 18), and period 4 (weeks 20, 21, 22, 23, or 24); month 6, month 9, month 12
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline; end of intervention period 1 (weeks 2, 3, 4, 5, or 6), period 2 (weeks 8, 9, 10, 11, or 12), period 3 (weeks 14, 15, 16,17, or 18), and period 4 (weeks 20, 21, 22, 23, or 24); month 6, month 9, month 12 for reporting.

Closest Location

Weill Cornell Medicine - New York, NY

Eligibility Criteria

This trial is for patients born any sex aged 65 and older. There are 2 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
should be considered for prophylactic cardiac pacemaker (PCP) placement Based on the findings of this study, it is recommended that any adult aged 65 or older who is believed to have TTR-cardiac amyloidosis be considered for a prophylactic cardiac pacemaker. show original
Taking beta-blocker

Patient Q&A Section

Can amyloidosis be cured?

"Patients with primary amyloidosis typically die of complications. Prognosis for patients who survive the first 2 years depends on the extent of disease at diagnosis, as well as treatment." - Anonymous Online Contributor

Unverified Answer

How many people get amyloidosis a year in the United States?

"Only the most severe forms of AL amyloidosis were underreported in Medicare enrollment in the United States during the 1990s. This surveillance deficiency probably accounts for an underreporting of all the cases of AL amyloidosis during that period. The AL prevalence in the United States was approximately 21.4 per 100,000 per year by the end of 1995, which is higher than in the European Union, where an AL prevalence of 0.3 per 100,000 per year has been reported.. Physicians caring for patients with this disease should be aware of AL amyloidosis and treat it with the same urgency as AL amyloidosis of the kidney or cardiac muscle." - Anonymous Online Contributor

Unverified Answer

What are the signs of amyloidosis?

"Signs of AL amyloidosis include dyspnea, pleural effusion and a restrictive cardiac filling pattern by echocardiography. Signs of AA amyloidosis include enlarged liver and spleen and high serum amyloid A." - Anonymous Online Contributor

Unverified Answer

What causes amyloidosis?

"Although it is possible to formulate a concise definition of amyloidoses, no single causative agent may cause all forms of amyloidoses, so each amyloidosis requires a definition of its own. We suggest that the best definition of amyloidosis may lie in our recognition that amyloidoses are a group of syndromes that are all caused by abnormal deposits of a fibrillar component of a protein." - Anonymous Online Contributor

Unverified Answer

What are common treatments for amyloidosis?

"Common treatments for AL are medication and supportive care. However, specific therapies such as autologous stem cell transplantation have not shown to improve the prognosis as of 2019." - Anonymous Online Contributor

Unverified Answer

What is amyloidosis?

"Amyloidosis is an insidious disease that is diagnosed at an average of 3 years prior to death. The incidence and prevalence of amyloidosis continues to grow each year." - Anonymous Online Contributor

Unverified Answer

Does treatment improve quality of life for those with amyloidosis?

"Quality of life for patients with AL amyloidosis is impaired in relation to both subjective and objective parameters. Treatment in this patient group is necessary." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in treatment for therapeutic use?

"There have been many advances in treatment options for various types of amyloid disease, in terms of their selectivity and safety. There is still much more research to be done in order to determine therapeutic efficacy and clinical outcome. However, most patients can be treated by a combination of drugs. There has been much refinement on the different therapeutic options, as well as new and safe drugs that are tailored to target treatment to particular subtypes of AL and AA amyloidosis." - Anonymous Online Contributor

Unverified Answer

How serious can amyloidosis be?

"In severe cases, a poor response to therapy is known to occur with a high rate of death, although the prognosis is still considered better than that of systemic light chains. An early diagnosis is recommended to detect this condition before irreversible organ damage occurs." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of amyloidosis?

"At present the primary or initiating cause in amyloidosis remains unclear. In most cases the cause of amyloidosis was found to be associated or associated with systemic diseases. However, other causes such as genetic mutations in the amyloidogenic transthyretin are being increasingly recognised. Thus our understanding of the genesis of amyloidosis is growing richer. In most cases a combination of several factors such as genetics, environmental factors, aging status and nutritional state seem to be necessary for the development of amyloidosis." - Anonymous Online Contributor

Unverified Answer

What is the latest research for amyloidosis?

"Treatment with tetrandrine has yielded satisfactory results in clinical trials performed at the National Cancer Institute, in combination with and without autologous stem cell transplantation. The most promising results were achieved in patients with AL amyloidosis, especially when the patient was young and had no underlying disease other than myeloma. Although this treatment is in clinical trials, it may prove to be as effective as, and maybe more so than, melphalan plus autologous stem cell transplantation in the treatment of patients with AL amyloidosis." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of treatment?

"The most common side effects of chemotherapy treatment are nausea, hair loss and anemia. The two most common side effects of melphalan treatment were hair loss and bruising. Nausea and vomiting with chemotherapy are common, and can be treated with antiemetic and laxatives and a low-lactating diet, but do not appear to be related to melphalan dosage. Hair loss, nail problems and peeling can be minimized by avoiding excessive light, and this is especially important with melphalan. Hair growth will usually recover after the chemotherapeutic treatment, and may take several months. Bleeding and anemia have been reported to occur in patients receiving melphalan, as a consequence of myelosuppression." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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