What side effects are associated with this type of intervention?

Common treatments for Transthyretin Amyloidosis (ATTR) include Patisiran and Tafamidis. Patisiran, an RNA interference therapeutic, has been associated with side effects such as peripheral edema and infusion-related reactions. Tafamidis, a transthyretin stabilizer, has shown side effects including urinary tract infections, vaginal infections, and diarrhea. Both treatments have been generally well-tolerated, but patients should be monitored for these potential adverse effects.

What prior FDA approvals exist?

Patisiran is an RNA interference therapeutic that targets and degrades transthyretin (TTR) mRNA, reducing the production of the TTR protein. The FDA has approved patisiran (Onpattro) for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. Another similar FDA-approved drug is inotersen (Tegsedi), an antisense oligonucleotide that also reduces TTR protein levels by inhibiting TTR mRNA. These treatments aim to mitigate the effects of TTR amyloidosis by decreasing the accumulation of amyloid deposits.

What other types of research exist?

Promising alternatives to Patisiran for Transthyretin Amyloidosis patients may include other RNA interference therapies, antisense oligonucleotides like Inotersen, and small molecules such as Tafamidis, which stabilizes the TTR protein. These treatments offer different mechanisms to reduce or stabilize TTR protein levels, potentially providing effective options for patients.

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RNAi Therapeutics

Patisiran for Cardiomyopathy

Phase 3

Waitlist Available

Research Sponsored by Alnylam Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure)

Able to complete ≥150 m on the 6-minute walk test

Must not have

New York Heart Association heart failure classification of III and at high risk

Neuropathy requiring cane or stick to walk, or is wheelchair bound

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to month 12

Awards & highlights

Summary

This trial is testing a medication called patisiran. It aims to help people with a heart condition caused by abnormal protein buildup. The medication works by lowering the levels of these harmful proteins in the body. Patisiran has been shown to significantly reduce symptoms and improve quality of life in patients.

Who is the study for?

This trial is for adults with a heart condition called ATTR amyloidosis with cardiomyopathy. They should have had heart failure symptoms or hospitalization, be stable without recent cardiovascular hospitalizations, able to walk a short distance in 6 minutes, and not severely affected by other diseases like severe lung disease or arthritis. People who've had certain organ transplants or infections like hepatitis B/C or HIV can't join.

What is being tested?

The study tests the effectiveness and safety of Patisiran compared to a placebo in treating ATTR amyloidosis affecting the heart. Participants will either receive Patisiran or an inactive substance without knowing which one they're getting to see if there's an improvement in their heart condition.

What are the potential side effects?

Patisiran may cause side effects such as infusion-related reactions (like flushing, back pain), vision issues, nausea, muscle/joint pain, and swelling. It might also affect liver function and could lead to abnormal blood test results.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been hospitalized for heart failure before or currently show signs of it.

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I can walk more than 150 meters in 6 minutes.

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I haven't been hospitalized for heart issues in the last 6 weeks.

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I have been diagnosed with ATTR amyloidosis affecting my heart.

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I have never used tafamidis or my condition worsened while on it for 6 months or more.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart condition is serious and considered high risk.

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I need a cane or wheelchair to walk due to nerve damage.

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I have been diagnosed with primary or leptomeningeal amyloidosis.

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I have a condition like severe lung disease or arthritis that makes it hard for me to walk.

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I have a heart condition not related to ATTR amyloidosis.

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My kidney function is severely reduced.

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I have received treatment to lower TTR levels before.

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My heart failure is classified as severe.

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I have had or am planning to have an organ transplant.

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My liver isn't working properly.

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I have hepatitis B, C, or HIV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to month 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)

Secondary study objectives

Change From Baseline at Month 12 in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score

Composite Endpoint of All-Cause Mortality and Frequency of All-Cause Hospitalizations and Urgent HF Visits in Participants Not on Tafamidis at Baseline

Composite Endpoint of All-Cause Mortality, Frequency of Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) and Change From Baseline in 6-MWT Analyzed by Win Ratio

+1 more

Side effects data

From 2022 Phase 3 trial • 211 Patients • NCT02510261

26%

Oedema Peripheral

26%

Diarrhoea

24%

Fall

21%

Pain in Extremity

20%

Nasopharyngitis

20%

Urinary Tract Infection

16%

Nausea

15%

Back Pain

15%

Dizziness

15%

Cough

15%

Upper Respiratory Tract Infection

14%

Headache

14%

Constipation

13%

Influenza

13%

Pyrexia

12%

Hypotension

12%

Infusion Related Reaction

12%

Hypertension

11%

Vertigo

11%

Arthralgia

10%

Vomiting

10%

Limb Injury

10%

Atrial Fibrillation

10%

Cataract

Muscle Spasms

COVID-19

Musculoskeletal Pain

Neuralgia

Rash

Insomnia

Thermal Burn

Syncope

Foot Fracture

Anaemia

Cellulitis

Rhinitis

Ligament Sprain

Traumatic Haematoma

Skin Abrasion

Abdominal Pain

Asthenia

Haematuria

Peripheral Swelling

Toothache

Depression

Gastroenteritis

Dysuria

Sciatica

Orthostatic Hypotension

Contusion

Decreased Appetite

Erythema

Haemorrhoids

Dysphonia

Conjunctivitis

Gastritis

Somnolence

Bronchitis

Skin Ulcer

Decubitus Ulcer

Dehydration

Post-traumatic Pain

Rhinorrhoea

Gastrooesophageal Reflux Disease

Sinusitis

Epistaxis

Dyspnoea

Eczema

Osteoarthritis

Dental Caries

Flushing

Pneumonia

Fatigue

Erysipelas

Pruritus

Neck pain

Cerebrovascular Accident

Vitamin D Deficiency

Conjunctival Haemorrhage

Myalgia

Paraesthesia

Oropharyngeal Pain

Cardiac Amyloidosis

Cardiac Failure

Atrial Flutter

Abdominal Pain Upper

Malaise

Fungal skin infection

Benign Prostatic Hyperplasia

Conduction Disorder

Abdominal Discomfort

Weight Decreased

Hip Fracture

Dry Mouth

Respiratory Tract Infection

N-terminal Prohormone Brain Natriuretic Peptide Increased

Vertigo Positional

Hypokalaemia

Urinary Retention

Flatulence

Atrioventricular Block Complete

Tachycardia

Generalised Oedema

Acute Kidney Injury

Choking

Device Physical Property Issue

Localised Infection

Cholecystitis

Pneumonia Aspiration

Tonsillitis

Familial Amyloidosis

Lower Limb Fracture

Carpal Tunnel Syndrome

Vitreous Opacities

Rhabdomyolysis

Rotator Cuff Syndrome

Breast Cancer

Infected Skin Ulcer

Stoma Site Extravasation

Sudden Hearing Loss

Bile Duct Stone

Cholecystitis Acute

Gangrene

Herpes Zoster

Facial Bones Fracture

Hyponatraemia

Autonomic Nervous System Imbalance

Aspiration

Anxiety

Burns Second Degree

Fractured Sacrum

Device Power Source Issue

Respiratory Acidosis

Visual Impairment

Dermatitis

Large Intestine Polyp

Hypoglycaemia

Foot Deformity

Metastases to Lymph Nodes

Basal Ganglia Infarction

Sudden Cardiac Death

Peritonitis

Metabolic Acidosis

Gastric Cancer

Prostatitis

Ventricular Arrhythmia

Femur Fracture

Lumbar Vertebral Fracture

Intraductal Proliferative Breast Lesion

Hydronephrosis

Acute Respiratory Failure

Neurogenic Shock

Arrhythmia

Skin Lesion

Inguinal Hernia

Cholangitis

Urosepsis

Road Traffic Accident

Amyloid Arthropathy

Lumbar Spinal Stenosis

Bladder Cancer Recurrent

Invasive Ductal Breast Carcinoma

Hereditary Neuropathic Amyloidosis

Abdominal Distension

Corneal Perforation

Device Capturing Issue

Infusion Site Cellulitis

Rib Fracture

Benign Renal Neoplasm

Ocular Surface Squamous Neoplasia

Dysarthria

Subarachnoid Haemorrhage

Hypoventilation

Pulmonary Embolism

Disability

Dysphagia

Nephrolithiasis

Neurogenic Bladder

Poor Peripheral Circulation

Cardiogenic Shock

Glaucoma

Retinal Detachment

Atrial Tachycardia

Atrioventricular Block

Bradycardia

Eye Haemorrhage

Keratitis

Retinal Ischaemia

Rhegmatogenous Retinal Detachment

Ulcerative Keratitis

Vitreous Haemorrhage

Colitis

Gastrointestinal Disorder

Gastrointestinal Haemorrhage

Extravasation

Gait Disturbance

General Physical Health Deterioration

Implant site injury

COVID-19 Pneumonia

Acute Myocardial Infarction

Pyelonephritis Acute

Septic Shock

Wound Infection

Ankle Fracture

Joint Dislocation

Atrioventricular Block Second Degree

Intraocular Pressure Increased

Cachexia

Electrolyte Imbalance

Restrictive Cardiomyopathy

Sinus Node Dysfunction

Cardiac Failure Congestive

Myocardial Infarction

Cholangiocarcinoma

Lethargy

Psychomotor Skills Impaired

Calculus Bladder

Chronic Kidney Disease

Sleep Apnoea Syndrome

Varicose Vein

Tendonitis

100%

80%

60%

40%

20%

Study treatment Arm

Prior Placebo Group of Study 004

Prior Patisiran Group of Study 004

Prior Patisiran Group of Study 003

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PatisiranExperimental Treatment1 Intervention

Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period.

Group II: PlaceboPlacebo Group2 Interventions

Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Patisiran

2015

Completed Phase 3

~340

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Transthyretin Amyloidosis (ATTR) include RNA-targeted therapies such as Patisiran and Inotersen. Patisiran is a small interfering RNA (siRNA) that degrades TTR mRNA, thereby reducing the production of both mutant and wild-type transthyretin (TTR) protein in the liver. This reduction in TTR protein decreases the formation of amyloid deposits in tissues, which is crucial for managing the disease's progression. Inotersen, another RNA-targeted therapy, works similarly by inhibiting TTR synthesis. These treatments are significant for ATTR patients as they directly target the root cause of amyloid deposition, potentially stabilizing or improving organ function and quality of life.

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