Ignite Creation Date:
2024-05-06 @ 11:27 AM
Last Modification Date:
2024-10-26 @ 12:45 PM
Study NCT ID:
NCT03520751
Status:
SUSPENDED
Last Update Posted:
2024-05-01
First Post:
2018-04-20
Brief Title:
Phase IIIa Trial of scAAV1tMCKNTF3 for Treatment of CMT1A
Sponsor:
Nationwide Childrens Hospital
Organization:
Nationwide Childrens Hospital
Study Overview
Official Title:
Phase IIIa Trial Evaluating scAAV1tMCKNTF3 for Treatment of Charcot-Marie-Tooth Neuropathy Type 1A CMT1A
Status:
SUSPENDED
Status Verified Date:
2024-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Vector has not been produced
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This clinical trial is an open-label one-time injection dose study in which scAAV1tMCKNTF3 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 887e11 vgkg will be enrolled
Detailed Description:
This clinical trial is an open-label one-time injection study in which scAAV1tMCKNTF3 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 887e11 vgkg based on a qPCR titer using linearized standards equivalent to 400x1012 vgkg based on a qPCR titer using supercoiled standards distributed bilaterally between both limbs in Cohort 1 Post-gene transfer monitoring will include follow up visits on days 7 14 30 60 90 120 and months 6 9 12 15 18 and 24 following gene transfer Safety is the primary endpoint for this clinical gene transfer trial Stopping criteria are based on development of unacceptable toxicity defined as the occurrence of any one Grade III or higher unanticipated treatment-related toxicity The secondary endpoint is efficacy defined as halting of the decline in functional and sensory abilities measured by the CMT Pediatric Scale CMTPedS at 2 years post gene transfer The CMTPedS is an 11-item scale comprised of the Functional Dexterity Test Nine-Hole Peg Test 9HPT hand grip foot plantar flexion foot dorsiflexion pinprick sensation vibration sensation the Bruininks Oseretsky Test- Balance assessment gait assessment long jump and six-minute walk test 6MWT Exploratory outcome measures will include 10 meter runwalk timed test 10M 100 meter timed test 100M peroneal and ulnar CMAP amplitude and sensory and motor conduction velocities a revised sensory testing to increase sensitivity for pinprick touch-test and vibration assessments visual analogue scales for pain and fatigue Short Form Health Survey SF-36 as Quality of Life measure and circulating NT-3 levels
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None