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A Phase 3 Study of NTLA-2001 in ATTRv-PN

ConditionsNeuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative DiseaseNeurodegenerative Disease, HereditaryNeurodegenerative DiseasesNeuromuscular DiseasesNerve DisordersNervous System DiseaseNervous System DiseasesGenetic Disease, InbornAmyloidosis, FamilialAmyloidosis, HereditaryAmyloidosisPolyneuropathiesAmyloid NeuropathiesAmyloid Neuropathies, FamilialPeripheral Nervous System DiseasePeripheral Nervous System DiseasesMetabolism, Inborn ErrorsMetabolic Diseases
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-85
SponsorIntellia Therapeutics

About this trial

This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of ATTRv-PN

Karnofsky Performance Status (KPS) ≥ 60

Disqualifiers

Other causes of amyloidosis (amyloidosis caused by non-TTR protein)

Other known causes of sensorimotor or autonomic neuropathy

Diabetes mellitus

New York Heart Association Class III or IV heart failure

Trial design

Design model

Parallel

Treatments tested in this trial

  • nexiguran ziclumeran

    Biological/Vaccine

    nexiguran ziclumeran 55 mg by single IV infusion

  • Normal Saline as Placebo

    Drug

    Normal saline (0.9% NaCl) by single IV infusion

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: nexiguran ziclumeranExperimental treatment 1 intervention
Group B: Normal SalinePlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Modified Neuropathy Impairment Score +7 (mNIS+7)

Time frame
18 Months
2

Serum TTR

Time frame
29 Days

Secondary outcomes

1

Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire

Time frame
18 Months
2

Modified Body Mass Index (mBMI)

Time frame
18 Months
3

Serum TTR

Time frame
18 Months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Intellia Therapeutics

Lead sponsor

Regeneron Pharmaceuticals

Collaborator

Source ClinicalTrials.gov
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