Neoplasms
Conditions
Brief summary
Because AVI-4020 Injection was found to cross the blood-brain barrier, and because AVI-4126 (RESTEN-NG) has potential medical benefit to people with a variety of diseases, including cancers, this study is being performed to find out if AVI-4126 also crosses the blood-brain barrier. If it does, then additional investigations could be performed, such as in people with certain types of cancer.
Interventions
DRUGAVI-4126 Injection (RESTEN-NG®)
Sponsors
Sarepta Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 64 Years
Healthy volunteers
Yes
Inclusion criteria
* Adult males 18 years to 64 years of age; * Good general health (as evidenced by no chronic conditions, normal physical exam, vital signs within normal limits; laboratory evaluations within normal range) * Signed and dated written informed consent form; and * Willing to participate in all study activities and all requirements, including effective contraception (viz., a double-barrier method) during the 7-day study surveillance period.
Exclusion criteria
* Hematology, serum chemistry (exclusive of electrolytes), and urinalysis laboratory test values \>2 times upper limits of normal or anemia (hemoglobulin \<11 g/dL), leukopenia (total white blood count \<3,000/µL or total neutrophils \<1,500/ µL) or thrombocytopenia (platelets \<100,000/µL). Electrolytes and coagulation values exceeding normal ranges are to be excluded. * Body Mass Index (BMI) \>35. * Calculated creatinine clearance (by the Cockroft and Gault Formula) \<70 mL/min, based on age and gender. * Positive HIV-1 or HIV-2 serology. * Positive HCV serology and/or positive plasma HCV-RNA status. * Positive HBsAg or HBcAb status. * Solid or hematopoetic organ transplant recipient. * Active illness or recent illness within 30 days of the first dose of study drug. * History of any of the following: brain injury, neoplasm, chronic or migraine headaches, cancer, meningitis or hydrocephalus. * Usage of any prescribed, over-the-counter or illicit drug(s) within 30 days of study drug administration. Use of herbal remedies and/or supplements at the discretion of the Investigator. * Unwilling to practice effective contraception during the study period. * Participation in any clinical interventional trial within the previous 6 months. * Positive drug urine screen.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine if the study drug penetrates the blood brain barrier following a single intravenous dose, and if so, the associated CSF, plasma and urine pharmacokinetics. | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety | — |
| Tolerability | — |
Countries
United States
Outcome results
None listed