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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04321980




Registration number
NCT04321980
Ethics application status
Date submitted
23/03/2020
Date registered
26/03/2020
Date last updated
14/06/2021

Titles & IDs
Public title
A Clinical Study to Measure the Effect of OP-101 After Being Administered Subcutaneous in Healthy Volunteers
Scientific title
A Phase 1 Open-Label Single-Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 (Dendrimer N-acetyl-cysteine) After Subcutaneous Administration in Healthy Volunteers
Secondary ID [1] 0 0
OP-101-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Healthy 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - OP-101

Experimental: Cohort 1 - Subjects in Cohort 1 will be administered 4 mg/kg OP-101 as a subcutaneous (SC) injection.

Experimental: Cohort 2 - Subjects in Cohort 2 will be administered 8 mg/kg OP-101 as a SC injection.


Treatment: Drugs: OP-101
Subcutaneous injection of OP-101 in healthy volunteers
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events
Timepoint [1] 0 0
Up to Day 15
Secondary outcome [1] 0 0
Pharmacokinetics: Maximum Observed Plasma Concentration (Cmax) of OP-101
Timepoint [1] 0 0
Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose
Secondary outcome [2] 0 0
Pharmacokinetics: Time to Reach Maximum Plasma Concentration (Tmax) of OP-101
Timepoint [2] 0 0
Pre-dose, 0.5 hours and at 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose
Secondary outcome [3] 0 0
Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to the Last Quantifiable Concentration (AUC0-last) of OP-101
Timepoint [3] 0 0
Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose
Secondary outcome [4] 0 0
Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 48 Hour Post Dose Time Point (AUC0-48) of OP-101
Timepoint [4] 0 0
Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose
Secondary outcome [5] 0 0
Pharmacokinetics: Renal Clearance (CLR) for OP-101
Timepoint [5] 0 0
Pre-dose, 0 to 4, 4 to 8, 8 to 12, 12 to 18, 18 to 24, and 24 to 48 hours post dose

Eligibility
Key inclusion criteria
- Is a healthy man or woman age 18 to 65 years, inclusive, at the Screening Visit;

- Has the ability to understand and sign the written Informed Consent Form (ICF) and
local medical privacy authorization forms, which must be obtained prior to any study
related procedures being completed;

- Body mass index (BMI) between 18 and 32 kg/m2, inclusive;

- Is in general good health, based upon the results of a medical history assessment,
physical examination, vital signs, and laboratory profile, as judged by the
Investigator;

- Female subjects of non-childbearing potential must be either surgically sterile
(hysterectomy, bilateral tubal ligation, salpingectomy, and/or bilateral oophorectomy
at least 26 weeks before the Screening Visit) or postmenopausal, defined as
spontaneous amenorrhea for at least 2 years, with follicle-stimulating hormone (FSH)
in the postmenopausal range at screening, based on the central laboratory's ranges;

- Female subjects of childbearing potential (ie, ovulating, premenopausal, and not
surgically sterile) and all male subjects must use a medically accepted contraceptive
regimen (including hormonal contraceptives) during their participation in the study
and for 30 days after the last administration of study drug. Medically accepted
contraceptive methods are defined as those with 90% or greater efficacy;

- Acceptable methods of contraception for male subjects enrolled in the study include
the following:

• Condoms or surgical sterilization of subject at least 26 weeks before the Screening
Visit (vasectomy);

- Acceptable methods of contraception for female subjects enrolled in the study include
the following:

- Surgical sterilization of subject at least 26 weeks before the Screening Visit
(includes hysterectomy or bilateral tubal ligation, oophorectomy, or
salpingectomy);

- Intrauterine device for at least 12 weeks before the Screening Visit;

- Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12
weeks before the Screening Visit; or

- Diaphragm;

- If male, subjects must agree to abstain from sperm donation through 90 days after
administration of the last dose of study drug;

- Female subjects may not be pregnant, lactating, or breastfeeding;

- Female subjects of childbearing potential must have negative result for pregnancy test
at screening and Check-in;

- Subjects must have a negative test result for hepatitis B surface antigen (HBsAg),
hepatitis C virus antibody (HCVab), and human immunodeficiency virus (HIV) antibody at
screening;

- Subjects must have an estimated glomerular filtration rate (eGFR) of =90 mL/min/1.73m2
at screening;

- Subjects must have a negative urine test for drugs of abuse (opiates, benzodiazepines,
amphetamines, cannabinoids, cocaine, barbiturates, and phencyclidine), cotinine, and
breath alcohol test at screening and Check-in; and

- Subjects must be willing and able to abide by all study requirements and restrictions.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Evidence of clinically significant hematologic, renal, endocrine, pulmonary, cardiac,
gastrointestinal (GI), hepatic, psychiatric, neurologic, immunologic, allergic disease
(including multiple or clinically significant drug allergies), or any other condition
that, in the opinion of the Investigator, might significantly interfere with the
absorption, distribution, metabolism, or excretion of study drug, or place the subject
at an unacceptable risk as a participant in this study;

- History of malignancy (other than successfully treated basal cell or squamous cell
skin cancer);

- History or presence of an abnormal ECG that, in the opinion of the Investigator, is
clinically significant;

- Laboratory results (serum chemistry, hematology, coagulation, and urinalysis) outside
the normal range at screening and Check-in and considered clinically significant in
the opinion of the Investigator. Any elevation of aspartate transaminase (AST) and
alanine transaminase (ALT) above the upper limit of normal at screening and/or
Check-in is exclusionary. One retest of an exclusionary laboratory result is allowed
at the discretion of the Investigator;

- Has had an acute illness considered clinically significant by the Investigator within
30 days prior to screening;

- History of alcoholism or drug abuse within 2 years prior to screening;

- Has used any product containing nicotine within 90 days prior to screening or intends
to use any product containing nicotine during the course of the study;

- Has had any immunizations (live vaccines) in the 4 weeks prior to screening;

- Has used medications that affect GI motility or gastric emptying; such as
metoclopramide, proton pump inhibitors, and H2 blockers; within 30 days prior to Day
1;

- Has used any prescription or over-the-counter medication (with exception of
acetaminophen), vitamins/herbal supplements (with the exception of hormonal
contraceptives) within 14 days prior to Day 1;

- Has used any other study drug within 30 days or 5 half-lives of the drug (whichever is
longer) prior to Day 1;

- Has lost or donated >450 mL of whole blood or blood products within 30 days prior to
screening;

- Investigator has reason to believe that the subject may be unable to fulfill the
protocol visit schedule or requirements;

- Has any finding that, in the view of the Investigator or Medical Monitor, would
compromise the subject's safety requirements; or

- Is employed by the Sponsor, the Contract Research Organization (CRO), or the study
site (permanent, temporary contract worker, or designee responsible for the conduct of
the study), or is a family member (spouse, parent, sibling, or child) of the Sponsor,
CRO, or study site employee.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/05/2020
Sample size
Target
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
CMAX - Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Orpheris, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A clinical study to measure the Safety, Tolerability, and Pharmacokinetics of OP-101 After
Subcutaneous Administration in Healthy Volunteers
Trial website
https://clinicaltrials.gov/ct2/show/NCT04321980
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04321980