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Clinical trial

An Open Label, Single Arm IIT Clinical Study Evaluating the Safety, Tolerability, and Preliminary Efficacy of GCB-001 in the Treatment of Patients With Delayed Onset Type 2 SMA Who Can Sit Alone But Cannot Walk.

NCT06772402 is registered in ClinicalTrials.gov with Genecombio Ltd. as lead sponsor.

NCT NCT06772402
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open Label, Single Arm IIT Clinical Study Evaluating the Safety, Tolerability, and Preliminary Efficacy of GCB-001 in the Treatment of Patients With Delayed Onset Type 2 SMA Who Can Sit Alone But Cannot Walk.
Lead sponsor
Genecombio Ltd.
Phase
Not applicable
Overall status
Enrolling by invitation
Start date
January 15, 2025
Completion date
December 31, 2030
Enrollment
6 (estimated)
Sites
1

Conditions

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

spinal muscular atrophy type 2