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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to month 12
Awards & highlights
Study Summary
This trial will test a new drug, patisiran, for safety and effectiveness in treating ATTR amyloidosis, a heart condition.
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.Check my eligibility
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.See study design
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 belowSelect...
I have been hospitalized for heart failure before or currently show signs of it.
Select...
I can walk more than 150 meters in 6 minutes.
Select...
I haven't been hospitalized for heart issues in the last 6 weeks.
Select...
I have been diagnosed with ATTR amyloidosis affecting my heart.
Select...
I have never used tafamidis or my condition worsened while on it for 6 months or more.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to month 12
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to month 12
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change From Baseline at Month 12 in Six-Minute Walk Test (6-MWT)
Secondary outcome measures
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 moreSide effects data
From 2022 Phase 3 trial • 211 Patients • NCT0251026132%
Nasopharyngitis
24%
Oedema Peripheral
24%
Limb Injury
20%
Flushing
20%
Urinary Tract Infection
20%
Fall
20%
Cataract
20%
Cough
16%
Dental Caries
16%
Vertigo
16%
Pain in Extremity
16%
Influenza
16%
Back Pain
16%
Infusion Related Reaction
12%
Skin Ulcer
12%
Constipation
12%
Vitamin D Deficiency
12%
Diarrhoea
12%
Erysipelas
12%
Vomiting
12%
Respiratory Tract Infection
12%
Tonsillitis
12%
Pyrexia
12%
Infected Skin Ulcer
12%
Nausea
12%
Musculoskeletal Pain
8%
Renal Failure
8%
Ear Pain
8%
Fatigue
8%
Post-traumatic Pain
8%
Urinary Retention
8%
Malaise
8%
Tendonitis
8%
Anxiety
8%
Traumatic Haematoma
8%
Toothache
8%
Hypertension
8%
Sinusitis
8%
Wound Infection
8%
Neuralgia
8%
Tibia Fracture
8%
Anaemia
8%
Cardiac Failure
8%
Osteoarthritis
8%
Ankle Fracture
8%
Fungal skin infection
8%
Paraesthesia
8%
Gait Disturbance
8%
Gastrooesophageal Reflux Disease
8%
Muscle Spasms
8%
Skin Lesion
8%
Oropharyngeal Pain
8%
Visual Impairment
8%
Localised Infection
8%
Vertigo Positional
8%
Atrial Flutter
8%
Depression
8%
Arthralgia
8%
Orthostatic Hypotension
8%
Syncope
8%
Dermatitis
4%
Thermal Burn
4%
Polyp
4%
Dehydration
4%
Headache
4%
Bone Cancer
4%
Weight Decreased
4%
Osteomyelitis
4%
Metastases to Meninges
4%
Seizure
4%
Confusional State
4%
Asthenia
4%
Conjunctival Haemorrhage
4%
Hypokalaemia
4%
Bronchitis
4%
Ligament Sprain
4%
Dysphonia
4%
Erythema
4%
Epistaxis
4%
Atrioventricular Block Complete
4%
Abdominal Pain Upper
4%
Upper Respiratory Tract Infection
4%
Sciatica
4%
Neck Pain
4%
Acute Kidney Injury
4%
Chest Discomfort
4%
Cerebral Infarction
4%
Brain Cancer Metastatic
4%
Metastatic Malignant Melanoma
4%
Benign Prostatic Hyperplasia
4%
Rash
4%
Atrial Tachycardia
4%
Coronary Artery Stenosis
4%
Conversion Disorder
4%
Restrictive Cardiomyopathy
4%
Rhinitis
4%
Cerebrovascular Accident
4%
Abdominal Pain
4%
Dizziness
4%
Myalgia
4%
Dysuria
100%
80%
60%
40%
20%
0%
Study treatment Arm
Prior Placebo Group of Study 004
Prior Patisiran Group of Study 003
Prior Patisiran Group of Study 004
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
Find a Location
Who is running the clinical trial?
Alnylam PharmaceuticalsLead Sponsor
72 Previous Clinical Trials
14,458 Total Patients Enrolled
5 Trials studying Transthyretin Amyloidosis
5,713 Patients Enrolled for Transthyretin Amyloidosis
Medical DirectorStudy DirectorAlnylam Pharmaceuticals
2,777 Previous Clinical Trials
8,063,120 Total Patients Enrolled
2 Trials studying Transthyretin Amyloidosis
188 Patients Enrolled for Transthyretin Amyloidosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My heart condition is serious and considered high risk.I have been hospitalized for heart failure before or currently show signs of it.I can walk more than 150 meters in 6 minutes.I need a cane or wheelchair to walk due to nerve damage.I have been diagnosed with primary or leptomeningeal amyloidosis.I have a condition like severe lung disease or arthritis that makes it hard for me to walk.I have a heart condition not related to ATTR amyloidosis.My kidney function is severely reduced.I have received treatment to lower TTR levels before.Your blood test for NT-proBNP shows levels above 300 ng/L and below 8500 ng/L. If you have atrial fibrillation, your NT-proBNP levels should be above 600 ng/L and below 8500 ng/L.I haven't been hospitalized for heart issues in the last 6 weeks.I have been diagnosed with ATTR amyloidosis affecting my heart.My heart failure is classified as severe.I have had or am planning to have an organ transplant.My liver isn't working properly.I have hepatitis B, C, or HIV.I have never used tafamidis or my condition worsened while on it for 6 months or more.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo
- Group 2: Patisiran
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Could you please provide a short list of other Patisiran-based research studies?
"Patisiran was first trialled in 2019, and to date there have been 18248 completed studies. Right now, there are 3 active studies, with a considerable amount of these taking place in Kansas City, Kansas."
Answered by AI
Do patients who take Patisiran experience any adverse effects?
"Patisiran's safety has been well-supported by efficacy data in Phase 3 trials, and it received a score of 3 from our team."
Answered by AI
Are people over the age of 40 able to sign up for this research project?
"Eligibility for this clinical trial includes being between 18-85 years old."
Answered by AI
Is this research pioneering in the medical field?
"First researched in 2019, Patisiran has made significant progress. The first study, sponsored by Alnylam Pharmaceuticals, enrolled 164 patients. After the success of the first study, Patisiran received approval for Phase 3 trials. Now, 3 years later, there are 20 cities and 27 countries with active Patisiran trials."
Answered by AI
Are there any current vacancies in this research project for individuals?
"As of right now, this particular clinical trial is not looking for any more patients. It was originally posted on September 4th, 2019, and the most recent edit was on October 3rd, 2022. However, there are still 251 other trials for amyloidosis enrolling patients and 3 for Patisiran that are actively searching for participants."
Answered by AI
How many individuals are given the chance to participate in this clinical trial?
"Unfortunately, this study is not currently enrolling patients. The listing was created on September 4th, 2019 and updated on October 3rd, 20212. There are, however, other trials which may be of interest. There are 251 studies recruiting patients with amyloidosis and 3 trials for Patisiran that are presently enrolling participants."
Answered by AI
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