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

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

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

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: ACT-1004-1239; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 50
• Phase: Phase 1

Contacts and Locations

Study Locations

• Portugal
  • Porto, Portugal, 4250-449
    • BlueClinical Phase 1 Hospital de Prelado

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ACT-1004-1239 Dose level 1 (30 mg) to 5; 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; ACT-1004-1239 administered as hard capsules for oral use.
Placebo Comparator: Placebo; 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; Matching placebo administered as hard capsules for oral use.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Idorsia Pharmaceuticals Ltd.

Publications and helpful links

General Publications

• Pouzol L, Baumlin N, Sassi A, Tunis M, Marrie J, Vezzali E, Farine H, Mentzel U, Martinic MM. ACT-1004-1239, a first-in-class CXCR7 antagonist with both immunomodulatory and promyelinating effects for the treatment of inflammatory demyelinating diseases. FASEB J. 2021 Mar;35(3):e21431. doi: 10.1096/fj.202002465R.

Study record dates

Study Major Dates

• Study Start (Actual): July 11, 2020
• Primary Completion (Actual): September 25, 2020
• Study Completion (Actual): September 25, 2020

Study Registration Dates

• First Submitted: February 25, 2020
• First Submitted That Met QC Criteria: February 25, 2020
• First Posted (Actual): February 27, 2020

Study Record Updates

• Last Update Posted (Actual): January 20, 2021
• Last Update Submitted That Met QC Criteria: January 19, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Other Study ID Numbers: ID-086-102; 2019-004262-17 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No