HealthyPhase 1NCT04452006
A Study in Healthy Subjects to Investigate the Safety and Tolerability of ACT- 541478 as Well as What ACT-541478 Does to the Body and the Way the Body Takes up, Distributes, and Gets Rid of ACT-541478
A Phase 1 study testing ACT-541478 10 mg, ACT-541478 30 mg and ACT-541478 100 mg in people with Healthy, run by Idorsia Pharmaceuticals Ltd. at 1 site in Germany.
Trial summary
A study in healthy subjects to investigate the safety and tolerability of ACT-541478 as well as what ACT-541478 does to the body and the way the body takes up, distributes, and gets rid of of ACT-541478 Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy
- What is being tested?
- ACT-541478 10 mg, ACT-541478 30 mg and ACT-541478 100 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 1 siteGermany
- Study dates
- 20 Jul 2020to 20 Apr 2021
- 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 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Part A/B/C:
- Signed informed consent in a language understandable to the subject prior to any study-mandated procedure.
- BMI of 18.0 to 29.9 kg/m2 (inclusive) at Screening.
- SBP 100-139 mmHg, DBP 50-89 mmHg, and pulse rate 50-90 bpm (inclusive), measured on either arm, after 5 min in the supine position at Screening and on Day -1 in the first period, if applicable.
- Fertile male subjects (defined as physiologically capable of conceiving a child according to the investigator's judgment) must agree to refrain from fathering a child and:
- be sexually abstinent with women of child-bearing potential or use condoms during sexual intercourse with women of child-bearing potential from (first) study treatment administration up to at least 90 days after (last) study treatment administration. Moreover, it is recommended that women of child-bearing potential partners of male subjects consistently and correctly use for the same period a highly effective method of contraception with a failure rate of < 1% per year.
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- to not donate sperm from (first) study treatment administration up to at least 90 days after (last) study treatment administration.
- Body temperature in the range of 35.5° to 37.5 °C at Screening and on Day -1 in the first period, if applicable.
- 12-lead safety ECG: QTcF ≤ 450 ms, QRS ≤ 110 ms, PR ≤ 220 ms, and resting HR 50-90 bpm (inclusive) with no clinically relevant abnormalities on 12-lead safety ECG after at least 5 min in the supine position at Screening and on Day -1 in the first period, if applicable.
- Normal renal function as confirmed by an estimated glomerular filtration rate ≥ 80 mL/min/1.73 m2 determined at Screening using the Chronic Kidney Disease Epidemiology Collaboration formula.
- Part A/B:
- Healthy male subjects aged between 18 and 55 years (inclusive) at Screening.
- Parts C1 to C3 (Adult subjects):
- Healthy male and female subjects aged between 18 and 55 years (inclusive) at Screening.
- Parts E1 and E2 (Elderly subjects):
- Healthy male and female subjects aged between 65 and 80 years (inclusive) at Screening.
- Parts C1 to C3, E1 and E2 (Female subjects):
- Women of child-bearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day
- 1. They must consistently and correctly use (from Screening, during the entire study, and for at least 30 days after last study treatment administration) a highly effective method of contraception with a failure rate of < 1% per year, or be sexually inactive, or have a vasectomized partner. If a hormonal contraceptive is used, it must have been initiated at least 1 month before first study treatment administration.
- Women of child-bearing potential (i.e., postmenopausal [defined as 12 consecutive months with no menses without an alternative medical cause, confirmed by an FSH test > 40 mIU/mL], or with documented previous bilateral salpingectomy, bilateral salpingo oophorectomy or hysterectomy, or with premature ovarian failure [confirmed by a specialist], XY genotype, Turner syndrome, uterine agenesis).
Who cannot join
- Part A/B/C
- History of major medical or surgical disorders, which, in the opinion of the investigator, are likely to interfere with the absorption, distribution, metabolism, or excretion of the study treatment (appendectomy and herniotomy allowed, cholecystectomy not allowed).
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
- History or presence of cardiac rhythm disorders (e.g., sinoatrial heart block, sick-sinus syndrome, 2nd or 3rd degree AV block, long QT syndrome, symptomatic bradycardia, atrial flutter, or atrial fibrillation).
- Any illness with a potential to increase the risk of the subject based on medical history.
- Clinically relevant findings in clinical laboratory tests (hematology, clinical chemistry, and urinalysis) at Screening (and on Day -1 in Part A and C) or any of the following laboratory parameters out of normal range: ALT, AST, total bilirubin, creatinine, TSH, and/or hemoglobin, at Screening and on Day -1 in the first period, if applicable.
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- Any signs or symptoms of active, ongoing infection judged to be clinically relevant by the investigator (special attention should be given to clinical signs and symptoms consistent with COVID-19).
- Part A (A3, effect of food)
- Known lactose intolerance.
- Known hereditary problems of galactose intolerance, Lapp lactase deficiency, or glucose-galactose malabsorption.
- Inability or unwillingness to completely consume the required high-fat meal.
- Part C
- Pregnant or lactating women.
- Relevant history of a suicide attempt or suicidal behavior. Any recent suicidal ideation within the last 6 months (categories 4 or 5), or any suicidal behavior within the last 2 years, except for "Non-Suicidal Self-Injurious Behavior" (item also included in the Suicidal Behavior section), as judged by the investigator using the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening.
- Result ≤ 27 in the Mini-Mental State Examination (MMSE®2™), assessed at Screening (E1 and E2 only).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Single-center, Double-blind, Randomized, Placebo-controlled, Three-part, First-in-human, Phase 1 Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACT-541478 in Healthy Subjects
A study in healthy subjects to investigate the safety and tolerability of ACT-541478 as well as what ACT-541478 does to the body and the way the body takes up, distributes, and gets rid of of ACT-541478
- Conditions
- Healthy
- Being tested
- ACT-541478 10 mg, ACT-541478 30 mg, ACT-541478 100 mg, ACT-541478 300 mg, ACT-541478 1000 mg
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACT-541478 10 mgDrug
ACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 10 mg.
ACT-541478 30 mgDrug
ACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 10 mg.
ACT-541478 100 mgDrug
ACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 50 mg.
ACT-541478 300 mgDrug
ACT-541478 will be provided in HPMC capsules for oral administration at dose strengths of 50 mg and 250 mg.
ACT-541478 1000 mgDrug
ACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 250 mg.
ACT-541478 high or low dose (or placebo)Drug
Cross-over design: ACT-541478 will be provided in HPMC capsules for oral administration at high or low dose strengths (to be defined after completion of Part A).
ACT-541478 dose E1Drug
E1 is a dose level that has been investigated and well tolerated in the adult dose level groups C1 to C3. ACT-541478 will be provided in HPMC capsules for oral administration at dose strengths of 10, 50, and 250 mg.
Matching placeboDrug
Matching placebo will be provided in HPMC capsules for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Part A (SAD) - A1, ACT-541478 10 mg fasted | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 10 mg; Drug: Matching placebo |
| Part A (SAD) - A2, ACT-541478 30 mg fasted | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 30 mg; Drug: Matching placebo |
| Part A (SAD) - A3 (Period 1), ACT-541478 100 mg fasted | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 100 mg; Drug: Matching placebo |
| Part A (SAD) - A3 (Period 2), ACT-541478 100 mg fed | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 100 mg; Drug: Matching placebo |
| Part A (SAD) - A4, ACT-541478 300 mg fasted | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 300 mg; Drug: Matching placebo |
| Part A (SAD) - A5, ACT-541478 1000 mg fasted | Experimental | SAD = single ascending dose Receives: Drug: ACT-541478 1000 mg; Drug: Matching placebo |
| Part B - B1-3, ACT-541478 low or high dose | Experimental | Not described Receives: Drug: ACT-541478 high or low dose (or placebo); Drug: Matching placebo |
| Part C (MAD) - C1, ACT-541478 30 mg, fasted | Experimental | MAD = multiple ascending dose Receives: Drug: ACT-541478 30 mg; Drug: Matching placebo |
| Part C (MAD) - C2, ACT-541478 100 mg, fasted | Experimental | MAD = multiple ascending dose Receives: Drug: ACT-541478 100 mg; Drug: Matching placebo |
| Part C (MAD) - C3, ACT-541478 300 mg, fasted | Experimental | MAD = multiple ascending dose Receives: Drug: ACT-541478 300 mg; Drug: Matching placebo |
| Part C (Elderly) E1, ACT-541478, fasted | Experimental | Not described Receives: Drug: ACT-541478 dose E1; Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent (serious) AEs
Measured: From (first) study treatment administration up to 96 h after last study treatment administration in the corresponding period (if applicable). Total duration: up to 5 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2020
- Study start20 Jul 2020
- Primary completion20 Apr 2021
- Study completion20 Apr 2021
Why it stopped: Based on interim analysis, the Sponsor decided to terminate the study prematurely.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HealthyStudies 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
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Part A: Single-ascending dose study in healthy male subjects (including investigation of the effect of food); Part B: Single-dose, three-way crossover study in healthy male subjects to investigate specific/additional parameters related to safety and tolerability; Part C: Multiple-ascending dose study in healthy male and female subjects (including healthy male and female elderly subjects).
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Idorsia Pharmaceuticals Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04452006
- Sponsor's protocol ID
- ID-088-101
- Status last verified
- Jul 2021
- First submitted
- 26 Jun 2020
- Last updated
- 9 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-center, Double-blind, Randomized, Placebo-controlled, Three-part, First-in-human, Phase 1 Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACT-541478 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT04452006
- Acronym / sponsor ID
- ID-088-101
- Conditions
- Healthy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Based on interim analysis, the Sponsor decided to terminate the study prematurely.
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Jul 2020 (actual)
- Primary completion
- 20 Apr 2021 (actual)
- Study completion
- 20 Apr 2021 (actual)
- First posted
- 30 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.
A study in healthy subjects to investigate the safety and tolerability of ACT-541478 as well as what ACT-541478 does to the body and the way the body takes up, distributes, and gets rid of of ACT-541478
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 11
- Model description
- Part A: Single-ascending dose study in healthy male subjects (including investigation of the effect of food); Part B: Single-dose, three-way crossover study in healthy male subjects to investigate specific/additional parameters related to safety and tolerability; Part C: Multiple-ascending dose study in healthy male and female subjects (including healthy male and female elderly subjects).
| Arm | Type | Receives |
|---|---|---|
| Part A (SAD) - A1, ACT-541478 10 mg fasted | Experimental | Drug: ACT-541478 10 mg, Drug: Matching placeboSAD = single ascending dose |
| Part A (SAD) - A2, ACT-541478 30 mg fasted | Experimental | Drug: ACT-541478 30 mg, Drug: Matching placeboSAD = single ascending dose |
| Part A (SAD) - A3 (Period 1), ACT-541478 100 mg fasted | Experimental | Drug: ACT-541478 100 mg, Drug: Matching placeboSAD = single ascending dose |
| Part A (SAD) - A3 (Period 2), ACT-541478 100 mg fed | Experimental | Drug: ACT-541478 100 mg, Drug: Matching placeboSAD = single ascending dose |
| Part A (SAD) - A4, ACT-541478 300 mg fasted | Experimental | Drug: ACT-541478 300 mg, Drug: Matching placeboSAD = single ascending dose |
| Part A (SAD) - A5, ACT-541478 1000 mg fasted | Experimental | Drug: ACT-541478 1000 mg, Drug: Matching placeboSAD = single ascending dose |
| Part B - B1-3, ACT-541478 low or high dose | Experimental | Drug: ACT-541478 high or low dose (or placebo), Drug: Matching placebo |
| Part C (MAD) - C1, ACT-541478 30 mg, fasted | Experimental | Drug: ACT-541478 30 mg, Drug: Matching placeboMAD = multiple ascending dose |
| Part C (MAD) - C2, ACT-541478 100 mg, fasted | Experimental | Drug: ACT-541478 100 mg, Drug: Matching placeboMAD = multiple ascending dose |
| Part C (MAD) - C3, ACT-541478 300 mg, fasted | Experimental | Drug: ACT-541478 300 mg, Drug: Matching placeboMAD = multiple ascending dose |
| Part C (Elderly) E1, ACT-541478, fasted | Experimental | Drug: ACT-541478 dose E1, Drug: Matching placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Signed informed consent in a language understandable to the subject prior to any study-mandated procedure.
- BMI of 18.0 to 29.9 kg/m2 (inclusive) at Screening.
- SBP 100-139 mmHg, DBP 50-89 mmHg, and pulse rate 50-90 bpm (inclusive), measured on either arm, after 5 min in the supine position at Screening and on Day -1 in the first period, if applicable.
- Fertile male subjects (defined as physiologically capable of conceiving a child according to the investigator's judgment) must agree to refrain from fathering a child and:
- Body temperature in the range of 35.5° to 37.5 °C at Screening and on Day -1 in the first period, if applicable.
Key exclusion criteria (15 listed)
- Part A/B/C
- History of major medical or surgical disorders, which, in the opinion of the investigator, are likely to interfere with the absorption, distribution, metabolism, or excretion of the study treatment (appendectomy and herniotomy allowed, cholecystectomy not allowed).
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
- History or presence of cardiac rhythm disorders (e.g., sinoatrial heart block, sick-sinus syndrome, 2nd or 3rd degree AV block, long QT syndrome, symptomatic bradycardia, atrial flutter, or atrial fibrillation).
- Any illness with a potential to increase the risk of the subject based on medical history.
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 |
|---|---|---|
| ACT-541478 10 mgACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 10 mg. | Drug | Part A (SAD) - A1, ACT-541478 10 mg fasted |
| ACT-541478 30 mgACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 10 mg. | Drug | Part A (SAD) - A2, ACT-541478 30 mg fasted, Part C (MAD) - C1, ACT-541478 30 mg, fasted |
| ACT-541478 100 mgACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 50 mg. | Drug | Part A (SAD) - A3 (Period 1), ACT-541478 100 mg fasted, Part A (SAD) - A3 (Period 2), ACT-541478 100 mg fed, Part C (MAD) - C2, ACT-541478 100 mg, fasted |
| ACT-541478 300 mgACT-541478 will be provided in HPMC capsules for oral administration at dose strengths of 50 mg and 250 mg. | Drug | Part A (SAD) - A4, ACT-541478 300 mg fasted, Part C (MAD) - C3, ACT-541478 300 mg, fasted |
| ACT-541478 1000 mgACT-541478 will be provided in HPMC capsules for oral administration at a dose strength of 250 mg. | Drug | Part A (SAD) - A5, ACT-541478 1000 mg fasted |
| ACT-541478 high or low dose (or placebo)Cross-over design: ACT-541478 will be provided in HPMC capsules for oral administration at high or low dose strengths (to be defined after completion of Part A). | Drug | Part B - B1-3, ACT-541478 low or high dose |
| ACT-541478 dose E1E1 is a dose level that has been investigated and well tolerated in the adult dose level groups C1 to C3. ACT-541478 will be provided in HPMC capsules for oral administration at dose strengths of 10, 50, and 250 mg. | Drug | Part C (Elderly) E1, ACT-541478, fasted |
| Matching placeboMatching placebo will be provided in HPMC capsules for oral administration. | Drug | Part A (SAD) - A1, ACT-541478 10 mg fasted, Part A (SAD) - A2, ACT-541478 30 mg fasted, Part A (SAD) - A3 (Period 1), ACT-541478 100 mg fasted, Part A (SAD) - A3 (Period 2), ACT-541478 100 mg fed, Part A (SAD) - A4, ACT-541478 300 mg fasted, Part A (SAD) - A5, ACT-541478 1000 mg fasted, Part B - B1-3, ACT-541478 low or high dose, Part C (Elderly) E1, ACT-541478, fasted, Part C (MAD) - C1, ACT-541478 30 mg, fasted, Part C (MAD) - C2, ACT-541478 100 mg, fasted, Part C (MAD) - C3, ACT-541478 300 mg, fasted |
Primary outcome
Treatment-emergent (serious) AEs
Time frame: From (first) study treatment administration up to 96 h after last study treatment administration in the corresponding period (if applicable). Total duration: up to 5 days.
Results and safety
The study was stopped early (reason given: Based on interim analysis, the Sponsor decided to terminate the study prematurely.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2021.
- Individual participant dataSharing planned: No
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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.
Clinical Trials study director
Idorsia Pharmaceuticals 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 ACT-541478 10 mg, ACT-541478 30 mg and ACT-541478 100 mg in people with Healthy, run by Idorsia Pharmaceuticals Ltd. at 1 site in Germany.
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 20 inclusion and 15 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: stopped early, as of the sponsor's last update on 9 Jul 2021. 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 Germany. 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. 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 was stopped early (reason given: Based on interim analysis, the Sponsor decided to terminate the study prematurely.). Results are not available in the source record.
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?
Treatment-emergent (serious) AEs (time frame: From (first) study treatment administration up to 96 h after last study treatment administration in the corresponding period (if applicable). Total duration: up to 5 days.). 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 9 Jul 2021.