Study of SPG302 in Healthy Volunteers and ALS Participants

Spinogenix

Status and phase: Active, not recruiting; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: SPG302

Study type: Interventional

Funder types: Industry

Identifiers

NCT05882695

SPG302-ALS-001

Details and patient eligibility

About

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

Full description

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

Enrollment: 112 estimated patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

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 <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 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 <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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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112 participants in 7 patient groups, including a placebo group

Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD)

Experimental group

Description:

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

Treatment:

Drug: SPG302

Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD)

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD)

Experimental group

Description:

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).

Treatment:

Drug: SPG302

Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD)

Placebo Comparator group

Description:

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).

Treatment:

Drug: Placebo

Experimental Part 3: Active SPG302 to be administered to participants with ALS

Experimental group

Description:

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.

Treatment:

Drug: SPG302

Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS

Experimental group

Description:

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 for up to 3 cycles in the USA and up to 12 cycles in Australia. A follow-up safety visit will be conducted 30 days after last dose (±7 days).

Treatment:

Drug: SPG302