Healthy Volunteers - Male and Female
Conditions
Brief summary
The purpose of this study is to assess the safety and tolerability of ORN252 in healthy adult participants.
Interventions
BIOLOGICALORN252
ORN252 is a lipid nanoparticle encapsulating a circular RNA encoding an anti-CD19 CAR protein.
Sponsors
Orna Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Male and female participants who are healthy as determined by the Investigator based on review of medical history, physical examination, and laboratory tests * Participant is willing and able to comply with study visits and other protocol requirements * Other protocol specific inclusion criteria may apply
Exclusion criteria
* Clinically significant history or presence of organ dysfunction * Use of any investigational product within 3 months, or 5 half-lives, whichever longer * Other protocol specific
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The incidence and severity of safety, clinical, and laboratory abnormalities in healthy adult participants. | 12 weeks | To evaluate the safety and tolerability of ORN252 administered to healthy adult participants. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic (PK) parameters of ORN252 components | 29 days | Area under the concentration versus time curve from time zero to the time of the last quantifiable concentration \[AUClast\] |
| Pharmacodynamic (PD) effects of of ORN252 | 12 weeks | Changes in circulating B cell counts and lymphocytes |
| Immunogenicity of ORN252 | 12 weeks | To assess the presence of anti-drug antibodies in healthy adult participants |
Countries
Australia
Contacts
CONTACTOrna Clinical Trials
Outcome results
None listed