Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPG302 in Healthy Volunteers and ALS Participants

Sponsor

Spinogenix (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05882695

Collaborator

(none)

112

Enrollment

Location

Arms

17.1

Anticipated Duration (Months)

6.6

Patients Per Site Per Month

Study Details

Study Description

Brief 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

Condition or Disease	Intervention/Treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: SPG302 Drug: Placebo	Phase 1

Detailed 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 days

Study Design

Study Type:

Interventional

Anticipated Enrollment :

112 participants

Allocation:

Randomized

Intervention Model:

Sequential Assignment

Intervention Model Description:

Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV and a repeat dose expansion in cohort(s)Phase 1 randomized, double-blind, placebo-controlled, single, and multiple ascending dose study in HV and a repeat dose expansion in cohort(s)

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

Double blinded

Primary Purpose:

Treatment

Official Title:

A Phase 1, Randomized, Double Blind, Placebo Controlled, Single and Multiple Dose Escalation Study in Healthy Volunteers and an Expansion Cohort in Adult Participants With Amyotrophic Lateral Sclerosis (ALS) to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SPG302

Anticipated Study Start Date :

Jul 1, 2023

Anticipated Primary Completion Date :

May 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Experimental Part 1: Active SPG302 to be administered to healthy volunteers (SAD) 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	Drug: SPG302 synthetic small molecule
Placebo Comparator: Placebo Comparator Part 1: Placebo comparator to be administered to healthy volunteers (SAD) 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	Drug: Placebo Placebo
Experimental: Experimental Part 2: Active SPG302 to be administered to healthy volunteers (MAD) 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).	Drug: SPG302 synthetic small molecule
Placebo Comparator: Placebo Comparator Part 2: Placebo comparator to be administered to healthy volunteers (MAD) 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).	Drug: Placebo Placebo
Experimental: Experimental Part 3: Active SPG302 to be administered to participants with ALS 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.	Drug: SPG302 synthetic small molecule
Placebo Comparator: Placebo Comparator Part 3: Placebo comparator to be administered to participants with ALS 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.	Drug: Placebo Placebo

Outcome Measures

Primary Outcome Measures

Safety and tolerability in healthy volunteers (SAD cohort) [7 days]
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in healthy volunteers (SAD food effect cohort) [15 days]
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in healthy volunteers (MAD cohort) [12 days]
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and tolerability in participants with ALS [60 days]
• Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort) [7 days]
PK parameters of SPG302 on concentrations in plasma
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort) [15 days]
Effects of food on SPG302 PK profile
Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort) [12 days]
PK parameters of SPG302 on concentrations in plasma
Plasma pharmacokinetics of SPG302 in participants with ALS [60 days]
PK parameters of SPG302 on concentrations in plasma
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS [60 days]
Spirometry

Other Outcome Measures

Effect of repeated dosing of SPG302 on electroencephalogram in healthy volunteers (MAD cohort) [12 days]
Change from baseline in EEG parameters
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS [60 days]
Spirometry
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS [60 days]
Number of respiratory complications
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS [60 days]
Functional outcomes
Effect of SPG302 on proteins and biomarkers in participants with ALS [60 days]
Multiple protein and immunological biomarkers

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Nucleus Melbourne	Melbourne	Victoria	Australia	3004

Sponsors and Collaborators

Spinogenix

Investigators

Principal Investigator: Ofer M Goren, MD, Nucleus Network

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Spinogenix

ClinicalTrials.gov Identifier:

NCT05882695

Other Study ID Numbers:

SPG302-ALS-001

First Posted:

May 31, 2023

Last Update Posted:

May 31, 2023

Last Verified:

May 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Spinogenix

Amyotrophic Lateral Sclerosis

regenerative

synapse

Additional relevant MeSH terms:

Motor Neuron Disease

Amyotrophic Lateral Sclerosis

Sclerosis

Study Results

No Results Posted as of May 31, 2023