Can a new drug ease nerve pain in rare heart disease?
NCT ID NCT05023889
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the drug patisiran in 10 adults with a rare condition called ATTR amyloidosis that causes nerve damage. Participants received an IV infusion every 21 days for 24 months. Researchers measured changes in nerve function, autonomic symptoms, and quality of life to see if the drug could help control the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- patisiran (also known as ONPATTRO)
- What this could lead to
- If successful, this could point toward a treatment that slows or improves nerve damage and quality of life in people with ATTR amyloidosis.
- What could go wrong
- This is a very small, early-phase study with only 10 participants, so results may not apply broadly. The drug also requires lifelong dosing and premedication, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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10 people
The number who actually took part.
- Started
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Aug 2022
- Finished
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Apr 2026
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Male or female \>18 2. Diagnosis of symptomatic polyneuropathy 3. wtATTR based on cardiac biopsy or Tc99m PYP 4. Negative hATTR sequencing 5. 0 to 0.5 gram/dl serum monoclonal protein. 6. No history of other secondary causes of neuropathy. 7. Have adequate complete blood counts and liver function tests 8. Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV) Exclusion Criteria: 1. Other Causes of neuropathy as determined by the principle investigator. 2. Has known human immunodeficiency virus (HIV) infection; 3. Primary AL. 4. NYHA Class IV at the Screening visit. 5. Has any of the following laboratory parameter assessments at screening: 1. Aspartate transaminase (AST) or alanine transaminase (ALT) levels ˃2.0 × the upper limit of normal (ULN). 2. Total bilirubin ˃ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if total bilirubin \<2 × ULN. 3. International normalized ratio (INR) ˃1.5 (unless patient is on anticoagulant therapy, in which case excluded if INR ˃3.5). 6\. Has eGFR \< 30 mL/min/1.73 m2 (using the modification of diet in renal disease \[MDRD\] formula). 7. Is currently taking diflunisal; if previously on this agent, must have at least a 6-month wash-out prior to dosing (Day 1). 8\. Is currently taking doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents must have completed a 30-day wash-out prior to dosing (Day 1). 9\. Received prior TTR-lowering treatment or participated in a gene therapy trial for amyloidosis. 10. Current or future participation in another investigational device or drug study, Scheduled to occur during this study, or has received an investigational agent or device within 30 days (or 5 half-lives of the investigational drug, whichever is longer) prior to dosing (Day 1). In the case of investigational TTR stabilizer drugs, washout for 6 months prior to dosing (Day 1) is required; this does not apply to patients who are on tafamidis at baseline (per inclusion Criterion 4). 11\. Requires treatment with calcium channel blockers (eg, verapamil, diltiazem) or digitalis. 12\. Other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease. 13\. Has non-amyloid disease affecting exercise testing (eg, severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation). 14\. Had acute coronary syndrome or unstable angina within the past 3 months. 15. Has history of sustained ventricular tachycardia or aborted ventricular fibrillation. 16\. Has persistent elevation of systolic (˃180 mmHg) and diastolic (˃100 mmHg) blood pressure that is considered uncontrolled by physician. 17-Has untreated hypo- or hyperthyroidism. 18-Prior or planned heart, liver, or other organ transplant. 19. Had a malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated. 20\. Has other medical conditions or comorbidities which, in the opinion of the Investigator would interfere with study compliance or data interpretation. 21. Female Is not willing to comply with the contraceptive requirements during the study period. 22\. History of illicit drug abuse within the past 5 years that in the opinion of the Investigator would interfere with compliance with study procedures or follow-up visits. \-
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Austin Neuromuscler Center/National Neuromuscular Research Institute
Austin, Texas, 78759, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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