Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy
Study ID: STU-2019-0631
Study SRK-015-002 is an active treatment study of SRK-015 in patients age 2 through 21 years old with later-onset SMa. Patients may receive SRK-015 as monotherapy or in addition to an SMn upregulator therapy approved for SMa. The study will enroll approximately 55 SMa patients across 3 separate parallel subpopulations subsequently described as cohorts: * Cohort 1, n[?]20: ambulatory Type 3 patients, age 5 through 21 years old, at least 10 of whom are not receiving an approved SMa treatment, as well as patients already receiving an approved SMa treatment that had been started after the patient turned 5 years old. Cohort 1 includes 5 additional patients to ensure an adequate representation of patients not concomitantly receiving an approved SMa therapy. * Cohort 2, n[?]15: Type 2 or non-ambulatory Type 3 patients, age 5 through 21 years old, already receiving an approved SMa treatment that had been started after the patient turned 5 years old. * Cohort 3, n[?]20: Type 2 patients, age [GreaterThanorequalTo]2 years old, already receiving an approved SMa treatment that had been started before the patient turned 5 years old. Patient participation in the study will consist of 3 parts, Screening, Treatment and Follow-up: 1. During the screening period, all patient screening and eligibility determinations will be conducted after informed consent (and, as required by local authorities, patient informed assent) has been provided and within 28 days prior to first dose. Screening motor function outcome measures will be conducted no less than 7 days prior to first dose. Baseline motor function outcome measures will be conducted within 48 hours prior to first dose. 2. During the 52-week treatment period, patients enrolled in Cohorts 1 and 2 will receive high dose (20 mg/kg) SRK-015 and patients enrolled in Cohort 3 will be randomized (1:1) in a blinded manner to receive either low dose (2 mg/kg) or high dose (20 mg/kg) SRK-015 (Figure 1). all treatments will be administered by iV infusion over approximately 2 hours once every 4 weeks. The first dose will be administered on Day 0 and the final dose on Day 364, for a total of 14 doses. There will be a (+-)7-day window around each dosing visit, with a minimum of 21 days and a maximum of 35 days between doses. Patients will be monitored at the study site through approximately 2 hours post-dose. if there are no acute reactions following the first two doses for a patient, and if the investigator determines that it would be safe to do so, the infusion duration can be changed to less than two hours but no shorter than 1 hour.
- Cancer Related
- Healthy Volunteers
- UT Southwestern Principal Investigator
- DIANA PATRICIA CASTRO
Primary objectives: * To assess safety and tolerability of SRK-015 in patients with later-onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMa) * To assess the efficacy of SRK-015 by assessing changes in motor function outcome measures Secondary objectives: * To characterize the pharmacokinetics (PK) of SRK-015 * To evaluate the pharmacodynamic (PD) effects of SRK-015 * To evaluate time to therapeutic effect between low and high dose SRK-015 in a predefined cohort (Cohort 3) * To evaluate the immunogenicity of SRK-015 * To evaluate the effect of SRK-015 on quality of life