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Clinical trial

First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations

NCT06351592 is registered in ClinicalTrials.gov with Regeneron Pharmaceuticals as lead sponsor.

NCT NCT06351592
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Registry record

As filed in ClinicalTrials.gov.

Title
First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations
Lead sponsor
Regeneron Pharmaceuticals
Phase
Phase 1/2
Overall status
Recruiting
Start date
August 28, 2024
Completion date
March 21, 2032
Enrollment
54 (estimated)
Sites
22

Conditions

As the registry lists them; a condition with its own page is linked.

amyotrophic lateral sclerosis (als), mutation in the superoxide dismutase-1 (sod1) gene