Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05882695




Registration number
NCT05882695
Ethics application status
Date submitted
22/05/2023
Date registered
31/05/2023
Date last updated
1/10/2024

Titles & IDs
Public title
Study of SPG302 in Healthy Volunteers and ALS Participants
Scientific title
A Phase 1/2a, 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
Secondary ID [1] 0 0
SPG302-ALS-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SPG302
Treatment: Drugs - Placebo

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

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

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).

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).

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

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

Experimental: Experimental Part 3: Open Label Extension - Active SPG302 administered to participants with ALS - 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: Drugs: SPG302
synthetic small molecule

Treatment: Drugs: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability in healthy volunteers (SAD cohort)
Timepoint [1] 0 0
7 days
Primary outcome [2] 0 0
Safety and tolerability in healthy volunteers (SAD food effect cohort)
Timepoint [2] 0 0
15 days
Primary outcome [3] 0 0
Safety and tolerability in healthy volunteers (MAD cohort)
Timepoint [3] 0 0
12 days
Primary outcome [4] 0 0
Safety and tolerability in participants with ALS
Timepoint [4] 0 0
60 days
Secondary outcome [1] 0 0
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD cohort)
Timepoint [1] 0 0
7 days
Secondary outcome [2] 0 0
Plasma pharmacokinetics of SPG302 in healthy volunteers (SAD food effect cohort)
Timepoint [2] 0 0
15 days
Secondary outcome [3] 0 0
Plasma pharmacokinetics of SPG302 in healthy volunteers (MAD cohort)
Timepoint [3] 0 0
12 days
Secondary outcome [4] 0 0
Plasma pharmacokinetics of SPG302 in participants with ALS
Timepoint [4] 0 0
12mon
Secondary outcome [5] 0 0
Clinical outcomes of multiple oral doses of SPG302 in participants with ALS
Timepoint [5] 0 0
12 mon
Secondary outcome [6] 0 0
Clinical efficacy measures of SPG302 in participants with ALS
Timepoint [6] 0 0
12 mon

Eligibility
Key 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
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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

* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/07/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
112
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Macquarie University - North Ryde
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [3] 0 0
Flinders Medical center - Adelaide
Recruitment hospital [4] 0 0
Nucleus Melbourne (healthy volunteers) - Melbourne
Recruitment postcode(s) [1] 0 0
2109 - North Ryde
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
5042 - Adelaide
Recruitment postcode(s) [4] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Spinogenix
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Novotech (Australia) Pty Limited
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/study/NCT05882695
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ofer M Gonen, MD
Address 0 0
Nucleus Network (for healthy volunteers)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Public queries for healthy volunteers
Address 0 0
Country 0 0
Phone 0 0
+61 1800 243 733
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05882695