Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 11:39 PM
Ignite Modification Date:
2025-12-24 @ 11:39 PM
NCT ID:
NCT03520751
Eligibility Criteria:
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 during the study
Exclusion Criteria:
* Active viral infection based on clinical observations or serological evidence of HIV, or Hepatitis B or C infection, herpesvirus or adenovirus
* Ongoing immunosuppressive therapy or immunosuppressive therapy within 6 months of starting the trial (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin)
* Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count \< 1.5K/µL
* Subjects with AAV1 binding antibody titers ≥ 1:50 as determined by ELISA immunoassay
* Subjects with circulating anti-NT-3 titers ≥ 1:50 as determined by ELISA immunoassay
* Treat with any investigational medication within 30 days before the infusion of study drug
* Abnormal laboratory values considered clinically significant (GGT \> 3XULN, bilirubin ≥ 3.0 mg/dL, creatinine ≥ 1.8 mg/dL, Hgb \< 8 or \> 18 g/Dl; WBC \> 15,000 per cmm)
* Any medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability
* Ankle contractures or surgeries preventing proper muscle strength testing
* Pregnancy or lactation (females subjects will be tested for pregnancy)
* Limb surgery in the past six months
* Severe infection (e.g. pneumonia, pyelonephritis, or meningitis) within 4 weeks before gene transfer visit (enrollment may be postponed)
* Anyone unwilling to disclose study participation with primary care physician and other medical providers.
* Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
18 Years
Maximum Age:
35 Years
Study:
NCT03520751
Study Brief:
Protocol Section:
NCT03520751