Healthy VolunteersPhase 1NCT04934891
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547
A Phase 1 study testing ION547 in people with Healthy Volunteers, run by Ionis Pharmaceuticals, Inc. at 1 site in Canada.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of the antisense inhibitor ION547 administered subcutaneously (SC) in healthy participants. 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?
- ION547
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 1 siteCanada
- Study dates
- 24 Mar 2021to 14 Sep 2022
- 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 (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
- Healthy males or females 18 to 65, inclusive, at the time of informed consent.
- Satisfy the following:
- Females: Women of childbearing potential are not allowed to participate in this study.
- b. Males must be abstinent, surgically sterile (vasectomy with negative semen analysis at Follow-up, or a surgically sterile non-pregnant female partner) or if engaged in sexual relations with a woman of childbearing potential (WOCBP), participant is utilizing a highly effective contraceptive method
- Body mass index (BMI) ≤ 35 kilograms per square meter (kg/m^2).
Who cannot join
- Clinically significant abnormalities in medical history or physical examination.
- Screening laboratory results as follows, or any other clinically significant (CS) abnormalities in screening laboratory values that would render a participant unsuitable for inclusion:
- Urine protein/creatinine (P/C) ratio greater than or equal to (≥) 0.2 milligrams (mg)/mg.
- Positive test (including trace) for blood on urinalysis.
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, alkaline phosphatase (ALP), serum creatinine, and blood urea nitrogen (BUN) > upper limit of normal (ULN).
- Hemoglobin A1c (HbA1c) > ULN
Show all 19Show fewer
- Platelet count < lower limit of normal (LLN)
- aPTT > ULN
- INR > 1.4
- Evidence of uncorrected hypothyroidism or hyperthyroidism based on thyroid function results at Screening.
- Uncontrolled hypertension (blood pressure [BP] > 160/100 millimeters of mercury [mmHg]).
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid [siRNA]) within 4 months of screening if single dose received, or within 12 months of screening if multiple doses received. Exclusion does not apply to Covid-19 messenger ribonucleic acid (mRNA) vaccines.
- History of bleeding diathesis or coagulopathy.
- Regular use of alcohol within 6 months prior to screening (> 7 drinks/week for females, > 14 drinks/week for males [1 drink = 5 ounces (150 mL) of wine or 12 ounces (360 mL) of beer or 1.5 ounces (45 mL) of hard liquor]), or use of soft drugs (such as marijuana) within 3 months prior to screening, or hard drugs (such as cocaine and phencyclidine [PCP]) within 1 year prior to screening, or positive urine drug screen at Screening.
- Concomitant medication restrictions: the use of prescription and over-the-counter medications including nonsteroidal anti-inflammatory drugs (with the exception of occasional acetaminophen or ibuprofen) and herbal remedies at Screening and throughout the Study Period.
- Smoking limitations: more than 10 cigarettes/day at Screening and those with a significant change in smoking habits within 1 month prior to Screening should be excluded.
- Blood donation of 50 to 499 mL within 30 days of Screening or of > 499 mL within 60 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 Single and Multiple Doses of ION547 Administered Subcutaneously to Healthy Subjects
The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of the antisense inhibitor ION547 administered subcutaneously (SC) in healthy participants.
Read the detailed description
This is a Phase 1, double-blind, randomized, placebo-controlled, dose escalation study of ION547 in up to 76 participants. Participants will be randomized to receive single and multiple doses of ION547 or placebo SC. The maximum length of participation in the study will be approximately 34 weeks.
- Conditions
- Healthy Volunteers
- Being tested
- ION547, Placebo
- Keywords
- Healthy Participants
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ION547Drug
ION547 will be administered by SC injection.
PlaceboDrug
ION547-matching placebo will be administered by SC injection.
| Study group | Role | What they receive |
|---|---|---|
| ION547 | Experimental | Ascending single multiple doses of ION547 will be administered by SC injection. Receives: Drug: ION547 |
| Placebo | Placebo comparator | Ascending single multiple doses of ION547-matching placebo will be administered by SC injection. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
Measured: Up to Day 232
Cmax: Maximum Observed Plasma Concentration of ION547
Measured: Up to Day 232
Tmax: Time Taken to Reach Maximal Plasma Concentration of ION547
Measured: Up to Day 232
AUCt: Area Under the Plasma Concentration-time Curve From Time Zero to Time t for ION547
Measured: Up to Day 232
Urine 0-24 Hour (hr) Excretion of ION547
Measured: Up to 24 hours
Change from Baseline in FXII Antigen Levels
Measured: Up to Day 232
Change from Baseline in FXII Activity Levels
Measured: Up to Day 232
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2021
- Study start24 Mar 2021
- Primary completion14 Sep 2022
- Study completion14 Sep 2022
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
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 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
- NCT04934891
- Sponsor's protocol ID
- ION547-CS1
- Status last verified
- Sep 2022
- First submitted
- 8 Jun 2021
- Last updated
- 23 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 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 Single and Multiple Doses of ION547 Administered Subcutaneously to Healthy Subjects
- ClinicalTrials.gov ID
- NCT04934891
- Acronym / sponsor ID
- ION547-CS1
- Conditions
- Healthy Volunteers
- Keywords
- Healthy Participants
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Mar 2021 (actual)
- Primary completion
- 14 Sep 2022 (actual)
- Study completion
- 14 Sep 2022 (actual)
- First posted
- 22 Jun 2021 (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 of this study is to evaluate the safety and tolerability of single and multiple doses of the antisense inhibitor ION547 administered subcutaneously (SC) in healthy participants.
Detailed description
This is a Phase 1, double-blind, randomized, placebo-controlled, dose escalation study of ION547 in up to 76 participants. Participants will be randomized to receive single and multiple doses of ION547 or placebo SC. The maximum length of participation in the study will be approximately 34 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ION547 | Experimental | Drug: ION547Ascending single multiple doses of ION547 will be administered by SC injection. |
| Placebo | Placebo comparator | Drug: PlaceboAscending single multiple doses of ION547-matching placebo will be administered by SC injection. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
- Healthy males or females 18 to 65, inclusive, at the time of informed consent.
- Satisfy the following:
- b. Males must be abstinent, surgically sterile (vasectomy with negative semen analysis at Follow-up, or a surgically sterile non-pregnant female partner) or if engaged in sexual relations with a woman of childbearing potential (WOCBP), participant is utilizing a highly effective contraceptive method
- Body mass index (BMI) ≤ 35 kilograms per square meter (kg/m^2).
Key exclusion criteria (19 listed)
- Clinically significant abnormalities in medical history or physical examination.
- Screening laboratory results as follows, or any other clinically significant (CS) abnormalities in screening laboratory values that would render a participant unsuitable for inclusion:
- Uncontrolled hypertension (blood pressure [BP] > 160/100 millimeters of mercury [mmHg]).
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
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 |
|---|---|---|
| ION547ION547 will be administered by SC injection. | Drug | ION547 |
| PlaceboION547-matching placebo will be administered by SC injection. | Drug | Placebo |
Primary outcome
Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
Time frame: Up to Day 232
Cmax: Maximum Observed Plasma Concentration of ION547
Time frame: Up to Day 232
Tmax: Time Taken to Reach Maximal Plasma Concentration of ION547
Time frame: Up to Day 232
AUCt: Area Under the Plasma Concentration-time Curve From Time Zero to Time t for ION547
Time frame: Up to Day 232
Urine 0-24 Hour (hr) Excretion of ION547
Time frame: Up to 24 hours
Change from Baseline in FXII Antigen Levels
Time frame: Up to Day 232
Change from Baseline in FXII Activity Levels
Time frame: Up to Day 232
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 23 Sep 2022.
- Individual participant dataSharing planned: No
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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 ION547 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 6 inclusion and 19 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 23 Sep 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?
Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) (time frame: Up to Day 232). The study lists 7 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 23 Sep 2022.