A Phase 2 interventional study of Neurosteroid enantiomer in Mild Traumatic Brain Injury, Concussion, sponsored by Oragenics, Inc.. Not yet recruiting at 1 site in Australia. Open to participants aged 18 Months to 60 Months. Per ClinicalTrials.gov, last updated 2025-03-11.
Sponsored by Oragenics, Inc. · Phase 2, Interventional, and Treatment
This is a Phase IIa, randomized, double-blind, placebo-controlled pilot study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ONP-002 in adults with mild traumatic brain injury (mTBI).
Broadly, a mTBI, often referred to as a concussion, is a type of head trauma that causes temporary disruption to brain function, usually resulting from a blow to the head, fall, or sudden movement, and is often characterised by symptoms of headache, dizziness, confusion, memory problems, and difficulty concentrating, without significant loss of consciousness or structural brain damage on imaging tests.
The study will enroll up to 40 participants into 2 parallel treatment arms, of 20 patients each, who will receive either 16 mg (8 mg twice daily [BID]) intranasal (IN) ONP-002 or placebo, at 8-12-hour intervals consecutively for 5 days with a total of up to 9 doses.
Up to 40 participants will be randomized 1:1 to receive either ONP-002 or placebo, using a block randomization algorithm.
If participants receive their first dose on the morning of Day1, they will receive a total of 9 doses. If participants receive their first dose on the evening of Day 1, they will receive a total of 8 doses. The last dose of study drug for all participants will be administered in the morning on Day 5.
Objectives Endpoints To determine the feasibility of administering ONP-002 in acute mTBI patients within 12 hours of injury. • Time to enrolment post injury and first treatment with ONP-002 To investigate the safety and tolerability of multiple IN doses of ONP-002 in patients with mTBI. • Incidence and severity of AEs
To establish a reproducible protocol for the combination of IN ONP-002 dosing and blood biomarker draws in mTBI patients for up to 5-days post-injury by;
Assess correlation of the below scores with initial positive blood biomarker findings prior to first dose:
Blood samples will be collected throughout the study for analysis of ONP-002 levels, and mTBI biomarkers including (but not limited to):
Efficacy endpoints will include the following assessment of patient reported symptom severity and clinical assessments of cognitive and visual-motor integrity:
Observed values and changes from baseline in vital signs (including oxygen saturation levels), macroscopic nasal examination, pulmonary function, ECG parameters and continuous clinical laboratory parameters will be summarized at each scheduled timepoint by dose group using descriptive statistics (n, mean, standard deviation, minimum, maximum, median). Categorical clinical laboratory data will be summarized at each scheduled timepoint using participant counts and percentages and tabulated by dose group. Abnormalities in clinical laboratory parameters and ECG parameters QTcF will be tabulated by dose group showing participant counts and percentages.
Physical examination findings will be listed only. Pharmacodynamics Individual participant blood biomarker concentrations at each timepoint will be listed and summarized by treatment using descriptive statistics (n, mean, standard deviation, coefficient of variation, minimum, maximum, median, and geometric mean) for each dose group.
Efficacy Observed values and changes from baseline of efficacy measures (RPQ, DANA 5-minute neurocognition test, K-D 3-minute visual motor test, GOS-E) will be summarized at each scheduled timepoint by treatment arm using descriptive statistics (n, mean, standard deviation, minimum, maximum, median).
Participants will be screened for study eligibility in the hospital Emergency Department on Day 1. Eligible participants will be transferred and confined at the clinical site until completion of post first dose assessments on Day 1 after which they will be discharged. Participants will receive a daily telephone call (reminder to administer) from clinic staff on days when they are required to self-administer at home and to complete their study diary. Study participants will then be required to attend the clinical research facility for further study assessments on Days 2, 5 and 10, and an end of study assessment on Day 30. Home nursing study assessments may be provided for participants unable to attend the clinical research facility for scheduled visits.
If participants experience any clinically significant adverse events (AEs) they may remain at the study site for further observation at the discretion of the Principal Investigator (PI).
The expected total maximum study duration for participants will be 30 days, which includes a screening on Day 1, a 5-day treatment period, and a 25-day follow-up period.
ONP-002 is being developed for the treatment of mTBI (concussion). A Phase 1 healthy adults study exploring the safety and tolerability of single and multiple ascending IN doses of ONP-002 has been completed. The current phase IIa study aims to establish that ONP-002 is safe and feasible for acute (first dose administered within 12 hours of injury) IN administration in patients with mTBI while establishing protocols for administration and proof of concept (POC) using blood biomarker panels, cognitive and visual motor assessments to be used in later phase studies.
2,113 studies on the registry are indexed under Brain Injuries; 385 are open to participants now.
This study's planned enrollment of 40 is below the median of 48 across 1,331 interventional studies indexed under Brain Injuries.
Browse Brain Injuries studies →Oragenics, Inc. is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Participants with suspected concussion presenting to the emergency department will be included in the study only if they satisfy all the following criteria:
Must be diagnosed with a mTBI by all the following measures:
Female patients if of childbearing potential (defined as any female who has experienced menarche and who has not undergone surgical sterilisation and is not postmenopausal):
Male participants must:
7.2.2 Exclusion Criteria
Participants will be excluded from the study if they meet any of the following criteria:
7.3 Screen Failures Participants who consent to participate in the study but fail to meet the eligibility criteria at any point during the Screening Period and until randomisation are defined as screening failures. The reason for each screening failure will be recorded on the appropriate screening and enrolment log.
7.4 Re-Screening Participants who fail screening are not permitted to be re-screened. 7.5 Participant Withdrawal Criteria
Participants will be advised that they are free to withdraw from the study at any time for any reason or, if necessary, the PI (or delegate) may discontinue a participant from the study to protect the participant's wellbeing. A participant may voluntarily withdraw or be withdrawn from the study for reasons including, but not limited to, the following:
The Sponsor will be notified as soon as possible of any participant withdrawals. The date and reasons for withdrawal will be recorded in the eCRF.
Any participant who prematurely discontinues study drug, should have all scheduled assessments performed and attend all scheduled follow-up visits. If a participant withdraws or is withdrawn from the study, an attempt should be made to perform the Early Termination Visit assessments detailed in the Schedule of Assessments (SoA) in Section 1.3.
7.6 Participant Replacement Final confirmation of eligibility will be conducted prior to administration of the first dose of study drug (Day 1).
Automatic replacement of participants is allowed if a participant withdraws or is withdrawn prior to administration of the first dose of study drug.
Participants who withdraw or are withdrawn from the study after administration of the first dose of study drug for reasons other than occurrence of a treatment-related SAE, may be replaced at the discretion of the PI (or delegate) and following consultation with the Sponsor.
Any participants enrolled as replacements will be allocated to the same study treatment (treatment arm) as the participant replaced.
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The placebo is 92mg of Hydroxy-propyl beta-cyclodextrin as a spray-dried powder
Drug: Neurosteroid enantiomer
ONP-002 is 8mg API and 92 Hydroxy-propyl beta-cyclodextrin as a spray-dried powder
Drug: Neurosteroid enantiomer
A non-natural synthetic neurosteroid enantiomer considered a new chemical entity
Patient symptom reporting
Neurological questionnaires for patient symptoms including Rivermead
Time frame: Days 2, 5, 10 and 30 post-injurt
This study is not yet recruiting, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
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Oragenics, Inc.