Study in Healthy Subjects to Examine the Safety and Tolerability of ACT-1004-1239 Given as Multiple, Gradually Increasing Doses and to Examine the Effects of ACT-1004-1239 on the Body and the Way the Body Takes up, Distributes, and Gets Rid of ACT-1004-1239

Study Overview
=================
Brief Summary
-----------------
Study in healthy subjects to examine the safety and tolerability of ACT-1004-1239 given as multiple, gradually increasing doses and to examine the effects of ACT-1004-1239 on the body and the way the body takes up, distributes, and gets rid of ACT-1004-1239

Official Title
-----------------
Randomized, Double-blind, Placebo-controlled, Phase 1 Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple-ascending Doses of ACT-1004-1239 in Healthy Subjects

Conditions
-----------------
Healthy

Intervention / Treatment
-----------------
* Drug: ACT-1004-1239
* Drug: Placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Signed informed consent prior to any study-mandated procedure. Healthy male or female subject aged between 18 and 55 years (inclusive) at Screening. A female subject of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day - 1 and must agree to consistently and correctly use a highly effective method of contraception with a failure rate of ≤1% per year, be sexually inactive, or have a vasectomized partner. A female subject of non-childbearing potential must be postmenopausal, or must have a medical history of previous bilateral salpingectomy, bilateral salpingo-oophorectomy, hysterectomy, premature ovarian failure, XY genotype, Turner syndrome, or uterine agenesis. A male subject must use adequate contraception from first study treatment administration up to at least 90 days after last administration unless he is vasectomized. A male subject must agree to refrain from donation of semen from first study treatment administration up to at least 90 days after last administration. Exclusion Criteria: Previous exposure to ACT-1004-1239. Known hypersensitivity to ACT-1004-1239, or any of its excipients. 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. Acute, ongoing, recurrent, or chronic systemic disease able to interfere with the evaluation of the study. Previous treatment with any prescribed medications (including vaccines) or over-the-counter (OTC) medications (including herbal medicines such as St John's Wort, homeopathic preparations, vitamins, and minerals) within 3 weeks prior to first study treatment administration. Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol. Pregnant or lactating woman. Any cardiac condition or illness that may jeopardize the safety of the study subject per investigator judgment based on medical history or 12-lead ECG measured at Screening. Any immunosuppressive treatment within 6 weeks or 5x terminal half-life (t½), whichever is longer, before study treatment administration.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
Accepts Healthy Volunteers

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Other
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ACT-1004-1239 Dose level 1 (30 mg) to 5<br>Each dose level will be investigated in a group of 10 male and female subjects (ratio 1:1, male:female), with 8 subjects being administered ACT-1004-239 and 2 subjects matching placebo (ratio 1:1, male:female). Sentinel dosing will be applied at each dose level, i.e., in each group two subjects (ratio 1:1, male:female) will initially receive study treatment (1 on active treatment and 1 on placebo). | Drug: ACT-1004-1239<br>* ACT-1004-1239 administered as hard capsules for oral use.<br>|
| Placebo Comparator: Placebo<br>Each dose level will be investigated in a group of 10 male and female subjects (ratio 1:1, male:female), with 8 subjects being administered ACT-1004-239 and 2 subjects matching placebo (ratio 1:1, male:female). Sentinel dosing will be applied at each dose level, i.e., in each group two subjects (ratio 1:1, male:female) will initially receive study treatment (1 on active treatment and 1 on placebo). | Drug: Placebo<br>* Matching placebo administered as hard capsules for oral use.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent (serious) adverse events |  | From first study treatment administration up to End of Study (EOS). Duration: up to 6 weeks |