Ornithine Transcarbamylase DeficiencyPhase 1NCT04416126
Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
A Phase 1 study testing ARCT-810 in people with Ornithine Transcarbamylase Deficiency, run by Arcturus Therapeutics, Inc. at 1 site in New Zealand.
Trial summary
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Ornithine Transcarbamylase Deficiency
- What is being tested?
- ARCT-810
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteNew Zealand
- Study dates
- 1 Jun 2020to 9 Dec 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Healthy males or females aged 18 to 65 at the time of informed consent.
- Body weight ≤ 100Kg and body mass index <35 kg/m2
- Willing to refrain from strenuous exercise/activity (for example heavy lifting, weight training, intense aerobics classes etc.) and alcohol for at least 72 hours prior to study visits and willing to refrain from taking protein supplements for the duration of the study.
- Willing and able to comply with protocol-defined procedures and complete all study visits
- Males must be surgically sterile or, if engaged in sexual relations with a female of child-bearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent form until at least 30 days after the last dose of Study Drug. Females: must be non-pregnant and non-lactating and either: i. surgically sterile or ii. post-menopausal
Who cannot join
- Clinically significant abnormalities in medical history
- Screening laboratory results as follows:
- ALT, AST, GGT, total bilirubin or alkaline phosphatase, > ULN.
- Random blood glucose and/or HbA1c > ULN
- Hemoglobin < LLN
- Platelet count < 100x109/L
Show all 14Show fewer
- Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2 calculated by Modification to Diet in Renal Disease [MDRD] study equation.
- Urine protein:creatinine ratio (UPCR) > 50 mg/mmol
- Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed within 7 days prior to Study Day 1
- Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
- Uncontrolled hypertension (BP > 160/100 mm Hg)
- Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational drug, whichever is longer
- Recent (within 1 year) history of, or current drug or alcohol abuse
- Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
Read the detailed description
This is a single ascending dose study of ARCT-810 in which approximately 30 subjects are planned to be enrolled. The length of each study participant is approximately 8 weeks from screening to last study visit.
Study participants will be allocated to one of the five different study groups (also called cohorts), to test different doses of ARCT-810. There will be 6 participants in each group. Within each cohort, subjects will be randomized 2:1 to receive ARCT-810 or placebo as an IV infusion.
- Conditions
- Ornithine Transcarbamylase Deficiency
- Being tested
- ARCT-810, Placebo
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARCT-810Biological
ARCT-810 is an investigational medicinal product comprising OTC mRNA formulated in a lipid nanoparticle (LNP) under development.
PlaceboOther
The placebo for this study is 0.9% sterile saline.
| Study group | Role | What they receive |
|---|---|---|
| ARCT-810 | Experimental | Ascending single doses of ARCT-810 administered intravenously Receives: Biological: ARCT-810 |
| Placebo | Placebo comparator | Single doses of 0.9% Saline administered intravenously Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity and dose-relationship of AEs
Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
Measured: 4 weeks
Secondary outcomes (2)
Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810
The plasma pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose
Measured: Up to 15 days
Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810
The urine pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose
Measured: Up to 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2020
- Study start1 Jun 2020
- Primary completion9 Dec 2020
- Study completion9 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ornithine Transcarbamylase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arcturus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Christian Schwabe, MD, Principal investigator, Auckland Clinical Studies (ACS) Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04416126
- Sponsor's protocol ID
- ARCT- 810-01
- Status last verified
- Nov 2023
- First submitted
- 28 May 2020
- Last updated
- 8 Nov 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects
- ClinicalTrials.gov ID
- NCT04416126
- Acronym / sponsor ID
- ARCT- 810-01
- Conditions
- Ornithine Transcarbamylase Deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Arcturus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 9 Dec 2020 (actual)
- Study completion
- 9 Dec 2020 (actual)
- First posted
- 4 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
Detailed description
This is a single ascending dose study of ARCT-810 in which approximately 30 subjects are planned to be enrolled. The length of each study participant is approximately 8 weeks from screening to last study visit.
Study participants will be allocated to one of the five different study groups (also called cohorts), to test different doses of ARCT-810. There will be 6 participants in each group. Within each cohort, subjects will be randomized 2:1 to receive ARCT-810 or placebo as an IV infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ARCT-810 | Experimental | Biological: ARCT-810Ascending single doses of ARCT-810 administered intravenously |
| Placebo | Placebo comparator | Other: PlaceboSingle doses of 0.9% Saline administered intravenously |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Healthy males or females aged 18 to 65 at the time of informed consent.
- Body weight ≤ 100Kg and body mass index <35 kg/m2
- Willing to refrain from strenuous exercise/activity (for example heavy lifting, weight training, intense aerobics classes etc.) and alcohol for at least 72 hours prior to study visits and willing to refrain from taking protein supplements for the duration of the study.
- Willing and able to comply with protocol-defined procedures and complete all study visits
- Males must be surgically sterile or, if engaged in sexual relations with a female of child-bearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent form until at least 30 days after the last dose of Study Drug. Females: must be non-pregnant and non-lactating and either: i. surgically sterile or ii. post-menopausal
Key exclusion criteria (14 listed)
- Clinically significant abnormalities in medical history
- Screening laboratory results as follows:
- Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed within 7 days prior to Study Day 1
- Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
- Uncontrolled hypertension (BP > 160/100 mm Hg)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARCT-810ARCT-810 is an investigational medicinal product comprising OTC mRNA formulated in a lipid nanoparticle (LNP) under development. | Biological | ARCT-810 |
| PlaceboThe placebo for this study is 0.9% sterile saline. | Other | Placebo |
Primary outcome
Incidence, severity and dose-relationship of AEs
Time frame: 4 weeks
Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
2 secondary outcomes
Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810
Time frame: Up to 15 days
Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810
Time frame: Up to 24 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christian Schwabe, MD principal investigator
Auckland Clinical Studies (ACS) Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ARCT-810 in people with Ornithine Transcarbamylase Deficiency, run by Arcturus Therapeutics, Inc. at 1 site in New Zealand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity and dose-relationship of AEs (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2023.