New shot every 6 months could slow rare nerve disease

NCT ID NCT07223203

Recruiting now Disease control Sponsor: Alnylam Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 4 times

Summary

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to an existing treatment and a placebo group from a previous study.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Nucresiran (a drug given as an injection under the skin every 6 months)
What this could lead to: If successful, nucresiran could offer a more convenient treatment option for people with hereditary ATTR amyloidosis with polyneuropathy, potentially slowing nerve damage and improving quality of life.
What could go wrong: This is an early-stage Phase 3 trial with only 125 participants, so results may not apply to everyone. The drug may not prove superior to the existing treatment vutrisiran, and side effects are still being studied.

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Conditions

The condition(s) this trial relates to.

amyloidosis familial amyloid neuropathy polyneuropathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HATTR-PN HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS WITH POLYNEUROPATHY

Contacts and locations

Locations

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Aurora, Colorado, 80045, United States
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Chicago, Illinois, 60611, United States
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Baltimore, Maryland, 21287, United States
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Boston, Massachusetts, 02118, United States
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Rochester, Minnesota, 55905, United States
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New York, New York, 10032, United States
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Chapel Hill, North Carolina, 27514, United States
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Nashville, Tennessee, 37232, United States
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Dallas, Texas, 75246, United States
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Houston, Texas, 77030, United States
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Darlinghurst, 2010, Australia
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Woolloongabba, 4102, Australia
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Ribeirão Preto, 14051-140, Brazil
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São Paulo, 04038-002, Brazil
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Vancouver, V5Z 1M9, Canada
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Égkomi, 2371, Cyprus
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Créteil, 94000, France
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Le Kremlin-Bicêtre, 94270, France
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Marseille, 13005, France
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Berlin, 10117, Germany
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Heidelberg, 69120, Germany
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Münster, 48149, Germany
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Athens, 115 28, Greece
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Heraklion, 715 00, Greece
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Florence, 50134, Italy
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Milan, 20133, Italy
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Pavia, 27100, Italy
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Roma, 00168, Italy
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Bunkyo C, 113-8519, Japan
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Kumamoto, 860-8556, Japan
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Matsumoto-shi, 390-8621, Japan
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Suita, 565-0871, Japan
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Kuala Lumpur, 59100, Malaysia
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Lisbon, 1649-035, Portugal
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Porto, 4099-001, Portugal
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Seoul, 05030, South Korea
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Seoul, 06351, South Korea
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Barcelona, 08035, Spain
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Huelva, 21005, Spain
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L'Hospitalet de Llobregat, 8907, Spain
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Palma de Mallorca, 07198, Spain
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Stockholm, 113 61, Sweden
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Umeå, 907 37, Sweden
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Taipei, 10002, Taiwan
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Taipei, 112, Taiwan
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Taoyuan City, 33305, Taiwan
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Istanbul, 34093, Turkey (Türkiye)
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Izmir, 35330, Turkey (Türkiye)