Nationwide Children's Hospital
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.
This clinical trial is an open-label, one-time injection study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into the medial and lateral heads of gastrocnemius, tibialis anterior, and rectus femoris muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three CMT1A patients, 18 to 35 years of age will be enrolled into one cohort in this trial. These adult subjects will be enrolled at an effective dose (8.87e11 vg/kg) based on a qPCR titer using linearized standards, equivalent to 4.00x1012 vg/kg based on a qPCR titer using supercoiled standards) distri…
Inclusion Criteria: * Subjects 18- 35 years old inclusive with CMT1A will be enrolled * Must exhibit a 1.5 Mb duplication at 17p11.2 inclusive of the peripheral myelin protein 22 (PMP22) gene * Males and females of any ethnic or racial group * Must exhibit weakness of the ankle dorsiflexion muscle (but has full ROM against gravity and is able to stand on heels 3 seconds or greater) * Abnormal nerve conduction velocities * Ability to cooperate for clinical evaluation and repeat nerve conduction studies * Willingness of sexually active subjects to practice a reliable method of contraception dur…
gene vector
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