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

Pivotal Trial

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%

Urinary Tract Infection

20%

Nasopharyngitis

16%

Nausea

15%

Upper Respiratory Tract Infection

15%

Back Pain

15%

Dizziness

15%

Cough

14%

Constipation

14%

Headache

13%

Influenza

13%

Pyrexia

12%

Hypertension

12%

Infusion Related Reaction

12%

Hypotension

11%

Vertigo

11%

Arthralgia

10%

Atrial Fibrillation

10%

Cataract

10%

Vomiting

10%

Limb Injury

Muscle Spasms

COVID-19

Musculoskeletal Pain

Neuralgia

Rash

Syncope

Thermal Burn

Insomnia

Haematuria

Anaemia

Foot Fracture

Traumatic Haematoma

Skin Abrasion

Abdominal Pain

Asthenia

Cellulitis

Rhinitis

Ligament Sprain

Depression

Decreased Appetite

Sciatica

Peripheral Swelling

Contusion

Gastroenteritis

Dysuria

Toothache

Erythema

Orthostatic Hypotension

Decubitus Ulcer

Dehydration

Somnolence

Haemorrhoids

Bronchitis

Gastritis

Conjunctivitis

Dysphonia

Skin Ulcer

Gastrooesophageal Reflux Disease

Eczema

Osteoarthritis

Flushing

Pneumonia

Dental Caries

Fatigue

Erysipelas

Sinusitis

Post-traumatic Pain

Dyspnoea

Epistaxis

Rhinorrhoea

Pruritus

Fungal skin infection

Myalgia

Malaise

Neck pain

Cardiac Amyloidosis

Cardiac Failure

Atrial Flutter

Cerebrovascular Accident

Conjunctival Haemorrhage

Abdominal Pain Upper

Vitamin D Deficiency

Paraesthesia

Oropharyngeal Pain

Flatulence

N-terminal Prohormone Brain Natriuretic Peptide Increased

Weight Decreased

Abdominal Discomfort

Atrioventricular Block Complete

Conduction Disorder

Hip Fracture

Vertigo Positional

Dry Mouth

Respiratory Tract Infection

Hypokalaemia

Urinary Retention

Benign Prostatic Hyperplasia

Respiratory Acidosis

Neurogenic Shock

Disability

Autonomic Nervous System Imbalance

Facial Bones Fracture

Gait Disturbance

Sudden Cardiac Death

Psychomotor Skills Impaired

Metastases to Lymph Nodes

Arrhythmia

Retinal Detachment

Device Capturing Issue

Urosepsis

Hydronephrosis

Pneumonia Aspiration

Septic Shock

Ulcerative Keratitis

Device Power Source Issue

Anxiety

Acute Kidney Injury

Femur Fracture

Hyponatraemia

Metabolic Acidosis

Foot Deformity

Carpal Tunnel Syndrome

Lethargy

Subarachnoid Haemorrhage

Tendonitis

Infected Skin Ulcer

Herpes Zoster

Pyelonephritis Acute

Fractured Sacrum

Stoma Site Extravasation

Amyloid Arthropathy

Device Physical Property Issue

Pulmonary Embolism

Wound Infection

Dermatitis

Large Intestine Polyp

Chronic Kidney Disease

Rhegmatogenous Retinal Detachment

Retinal Ischaemia

Hypoglycaemia

Basal Ganglia Infarction

Calculus Bladder

Hypoventilation

Varicose Vein

Bladder Cancer Recurrent

Cholangiocarcinoma

Invasive Ductal Breast Carcinoma

Poor Peripheral Circulation

Breast Cancer

Nephrolithiasis

Gastric Cancer

Atrioventricular Block

Atrial Tachycardia

Acute Myocardial Infarction

Cardiac Failure Congestive

Myocardial Infarction

Tachycardia

Hereditary Neuropathic Amyloidosis

Atrioventricular Block Second Degree

Cardiogenic Shock

Restrictive Cardiomyopathy

Ventricular Arrhythmia

Eye Haemorrhage

Glaucoma

Keratitis

Sinus Node Dysfunction

Familial Amyloidosis

Bradycardia

Sudden Hearing Loss

Corneal Perforation

Vitreous Haemorrhage

Vitreous Opacities

Abdominal Distension

Colitis

Gastrointestinal Disorder

Gastrointestinal Haemorrhage

Inguinal Hernia

Extravasation

General Physical Health Deterioration

Generalised Oedema

Implant site injury

Bile Duct Stone

Cholangitis

Cholecystitis

Cholecystitis Acute

COVID-19 Pneumonia

Gangrene

Infusion Site Cellulitis

Peritonitis

Ankle Fracture

Burns Second Degree

Joint Dislocation

Lower Limb Fracture

Lumbar Vertebral Fracture

Rib Fracture

Road Traffic Accident

Intraocular Pressure Increased

Cachexia

Electrolyte Imbalance

Lumbar Spinal Stenosis

Rhabdomyolysis

Rotator Cuff Syndrome

Benign Renal Neoplasm

Intraductal Proliferative Breast Lesion

Ocular Surface Squamous Neoplasia

Dysarthria

Neurogenic Bladder

Prostatitis

Acute Respiratory Failure

Aspiration

Choking

Sleep Apnoea Syndrome

Visual Impairment

Dysphagia

Localised Infection

Tonsillitis

Skin Lesion

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

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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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