study of spg302 in healthy volunteers and als participants

Conditions: Amyotrophic Lateral Sclerosis

Summary: the first-in-human phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of spg302 in healthy volunteers and als participants

this study is a phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in hv with food effect cohort, and a repeat dose expansion cohort(s) in participants with als. the study consists of 3 parts, as follows: * part 1: sad in hv with up to 6 cohorts including a food effect cohort. * part 2: mad over 5 days in hv with up to 5 cohorts * part 3: als cohorts with once daily (qd) dosing over 28 day cycles

Eligibility criteria:
inclusion criteria: * age 18-55 * must be in good health with no significant medical history * clinical laboratory values within normal range or \< 1.2 times uln * bmi 18-32 (inclusive) * contraceptive use by men or women consistent with local regulations * able and willing to provide written informed consent exclusion criteria: * any physical or psychological condition that prohibits study completion * known cardiac disease * active or history of malignancy in the past 5 years * serious infection within 1 month of screening * acute illness within 30 days of day 1 * surgery, bone fracture, or major musculoskeletal injury in the past 3 months * history of suicidal behavior or suicidal ideation * active cigarette smokers and users of nicotine-containing products * hiv, hepatitis b and hepatitis c positive * sbp \>140 or \<90 * dbp \>90 or \<40 * hr \ 100 * qtcf \>450ms, cardiac arrhythmia, or clinically significant abnormal ecg * prescriptions, over-the-counter, or herbal medication within 7 days * vaccines within 14 days * other investigational products within 30 days * blood donation within 30 days * plasma donation within 7 days * pregnant or breastfeeding * otherwise unfit, on metabolic-altering lifestyle/diet, positive urine drug screen or intake of alcohol or caffeine-containing products als cohort inclusion criteria: * age 18-80 * als tricals risk score * stable dose of standard of care treatment * contraception use by men or women consistent with local regulations * able and willing to provide written informed consent als cohort exclusion criteria: * underlying physical or psychological condition prohibiting study completion * known cardiac disease * active or history of malignancy in the past 5 years * serious infection within 1 month of screening * acute illness within 30 days of day 1 * history of suicidal behavior or suicidal ideation * active cigarette smokers and users of nicotine-containing products * neurodegenerative disease * external respiratory support or supplemental oxygen requirement * hiv, hepatitis b and hepatitis c positive * sbp \>140 or \<90 * dbp \>90 or \<40 * hr \ 100 * qtcf \>450ms, cardiac arrhythmia, or clinically significant abnormal ecg * vaccines within 14 days * other investigational products within 30 days * blood donation within 30 days * plasma donation within 7 days * pregnant or breastfeeding * otherwise unfit

Keywords: amyotrophic lateral sclerosis regenerative synapse