Healthy VolunteersPhase 1NCT03263507
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
A Phase 1 study testing Donidalorsen in people with Healthy Volunteers, run by Ionis Pharmaceuticals, Inc. at 1 site in Canada.
Trial summary
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen administered subcutaneously to Healthy Volunteers Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteers
- What is being tested?
- Donidalorsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 1 siteCanada
- Study dates
- 28 Aug 2017to 9 Oct 2018
- 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
- Must have given written informed consent and be able to comply with all study requirements
- Healthy males or females aged 18-65 inclusive at the time of Informed Consent
- Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
- Males must be surgically sterile, abstinent or using an acceptable contraceptive method
- BMI <35 kg/m2
Who cannot join
- Clinically-significant abnormalities in medical history including acute coronary syndrome, major surgery within 3 months of Screening, planned surgery that would occur during the study or physical examination or other screening results such as ECGs at Screening
- Clinically significant hematologic, chemistry, and urine abnormalities
- Treatment with another Study Drug, biological agent, or device within 4 weeks of Screening
- History of bleeding diathesis or coagulopathy
- Smoking >10 cigarettes per day
- Considered unsuitable for inclusion by the Principal Investigator
Show all 9Show fewer
- Current use of concomitant medications (including herbal or OTC medications) unless approved by Sponsor Medical Monitor
- Known history or positive test for HIV, hepatitis C or chronic hepatitis B
- Blood donation within 30 days of screening
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Double-Blind, Placebo-Controlled, Dose-Escalation Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 721744, an Antisense Oligonucleotide Inhibitor of PKK, Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen administered subcutaneously to Healthy Volunteers
- Conditions
- Healthy Volunteers
- Being tested
- Placebo, Donidalorsen
- Keywords
- IONIS PKK-LRx
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Saline .9%
DonidalorsenDrug
Also called ISIS 721744, IONIS-PKK-LRx
Donidalorsen administered subcutaneously
| Study group | Role | What they receive |
|---|---|---|
| Donidalorsen | Experimental | Ascending single and multiple doses of Donidalorsen administered subcutaneously Receives: Drug: Donidalorsen |
| Placebo (sterile saline 0.9%) | Placebo comparator | Calculated volume to match active comparator Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events that are related to treatment with Donidalorsen
The safety and tolerability of multiple doses of Donidalorsen will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with Donidalorsen.
Measured: Up to 176 days
Peak plasma Concentration (Cmax) of Donidalorsen
Maximum Donidalorsen plasma concentration, Cmax (ug/mL) will be assessed following SC administration
Measured: Up to 176 days
Time to peak plasma Concentration (Tmax) of Donidalorsen
Time to peak Donidalorsen plasma concentration, Tmax (hours) will be assessed following SC administration
Measured: Up to 176 days
Effects of Donidalorsen on plasma PKK concentration
Effects of Donidalorsen on plasma PKK concentration after multiple doses of Donidalorsen compared to baseline
Measured: Up to 176 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2017
- Study start28 Aug 2017
- Primary completion9 Oct 2018
- Study completion9 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteersStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03263507
- Sponsor's protocol ID
- ISIS 721744-CS1
- Status last verified
- Aug 2022
- First submitted
- 17 Aug 2017
- Last updated
- 4 Aug 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled, Dose-Escalation Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 721744, an Antisense Oligonucleotide Inhibitor of PKK, Administered Subcutaneously to Healthy Volunteers
- ClinicalTrials.gov ID
- NCT03263507
- Acronym / sponsor ID
- ISIS 721744-CS1
- Conditions
- Healthy Volunteers
- Keywords
- IONIS PKK-LRx
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Aug 2017 (actual)
- Primary completion
- 9 Oct 2018 (actual)
- Study completion
- 9 Oct 2018 (actual)
- First posted
- 28 Aug 2017 (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.
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen administered subcutaneously to Healthy Volunteers
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Donidalorsen | Experimental | Drug: DonidalorsenAscending single and multiple doses of Donidalorsen administered subcutaneously |
| Placebo (sterile saline 0.9%) | Placebo comparator | Drug: PlaceboCalculated volume to match active comparator |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Must have given written informed consent and be able to comply with all study requirements
- Healthy males or females aged 18-65 inclusive at the time of Informed Consent
- Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
- Males must be surgically sterile, abstinent or using an acceptable contraceptive method
- BMI <35 kg/m2
Key exclusion criteria (9 listed)
- Clinically-significant abnormalities in medical history including acute coronary syndrome, major surgery within 3 months of Screening, planned surgery that would occur during the study or physical examination or other screening results such as ECGs at Screening
- Clinically significant hematologic, chemistry, and urine abnormalities
- Treatment with another Study Drug, biological agent, or device within 4 weeks of Screening
- History of bleeding diathesis or coagulopathy
- Smoking >10 cigarettes per day
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 |
|---|---|---|
| PlaceboSaline .9% | Drug | Placebo (sterile saline 0.9%) |
| DonidalorsenAlso: ISIS 721744, IONIS-PKK-LRxDonidalorsen administered subcutaneously | Drug | Donidalorsen |
Primary outcome
Incidence and severity of adverse events that are related to treatment with Donidalorsen
Time frame: Up to 176 days
The safety and tolerability of multiple doses of Donidalorsen will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with Donidalorsen.
Peak plasma Concentration (Cmax) of Donidalorsen
Time frame: Up to 176 days
Maximum Donidalorsen plasma concentration, Cmax (ug/mL) will be assessed following SC administration
Time to peak plasma Concentration (Tmax) of Donidalorsen
Time frame: Up to 176 days
Time to peak Donidalorsen plasma concentration, Tmax (hours) will be assessed following SC administration
Effects of Donidalorsen on plasma PKK concentration
Time frame: Up to 176 days
Effects of Donidalorsen on plasma PKK concentration after multiple doses of Donidalorsen compared to baseline
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 4 Aug 2022.
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Screening Protocol
Pain
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.
The record lists no contact. Use the official record to reach the sponsor.
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 Donidalorsen in people with Healthy Volunteers, run by Ionis Pharmaceuticals, Inc. at 1 site in Canada.
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 9 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 4 Aug 2022. 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 Canada. 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 and severity of adverse events that are related to treatment with Donidalorsen (time frame: Up to 176 days). The study lists 4 primary outcomes in all. 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 4 Aug 2022.