Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

This 2-part first in human (FIH) study consists of an open-label, single ascending dose Part 1, which may identify the optimal biologically active dose (OBD) of NTLA-2001, followed by Part 2, if applicable, an open-label, single-dose expansion at the OBD to further characterise activity of NTLA-2001, provide an initial assessment of the effect of NTLA-2001 on clinical measures of neuropathy and neurologic function, and obtain additional safety data at the OBD.

Information provided by (Responsible Party):

For more details on study and if recruitment is still underway, click HERE.

To contact Auckland clinical trial centre, please email ‘’

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