Back to resultsSafety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPG302 in Healthy Volunteers and ALS Participants
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis, regenerative, synapse
Eligibility Criteria
Inclusion Criteria: Age 18-55 Must be in good health with no significant medical history Clinical laboratory values within normal range 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 <40 or >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 FVC>80% ALSFRS-R >=12 points 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 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 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 <40 or >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
Sites / Locations
- Nucleus MelbourneRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Placebo Comparator
Experimental
Placebo Comparator
Experimental
Placebo Comparator
Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD)
Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD)
Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD)
Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD)
Experimental Part 3: Active SPG302 to be administered to participants with ALS
Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS
8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule
8 participants will be randomized in a 3:1 ratio to active or placebo. Study intervention will be administered orally once. Randomization to each SAD cohort will be done in a staggered manner; initially 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed and after a safety evaluation period after the dose without clinically significant adverse events (AEs) and investigator approval, then, 6 additional participants will be randomized and dosed (5 active and 1 placebo) at the discretion of the Investigator according to the randomization schedule
8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).
8 participants will be randomized in a 3:1 ratio to active or placebo. Participants will receive study intervention QD over 5 days and will be discharged on Day 6. A follow-up safety visit will be conducted on Day 12 (±3 days).
12 participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.
12 participants with ALS will be randomized to receive SPG302 or placebo at a 3:1 ratio. Study intervention will be administered QD over 28 days. A follow-up safety visit will be conducted 30 days after last dose (±7 days). Participants who complete Part 3 may be offered to participate in an open-label extension.
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
2. Study Status
3. Sponsor/Collaborators
4. Oversight
5. Study Description
6. Conditions and Keywords
7. Study Design
8. Arms, Groups, and Interventions
10. Eligibility
12. IPD Sharing Statement
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