Clinical trial NCT03832452: A Study in Healthy Subjects to Investigate Whether Administration of Lucerastat Can Affect Normal Heart Function
Official title: Prospective, Single-center, Randomized, Double-blind, Placebo-controlled, Two-part Phase 1 Study to Assess the Effect of Single Therapeutic and Supra-therapeutic Doses of Lucerastat on the QT/QTc Interval Duration in Healthy Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Sponsor class: INDUSTRY
First posted: 2019-02-06
Enrollment: 44
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Prospective, single-center, randomized, double-blind, placebo-controlled, two-part Phase 1 study (Part A: parallel, 2 treatments; Part B: crossover, 4 treatments)
Primary purpose: OTHER

Brief summary: Two-part Phase 1 study to assess the effect of single therapeutic and supra-therapeutic doses of lucerastat on the QT/QTc interval duration in healthy subjects (Part A: Pilot study; Part B: TQT study)


Eligibility criteria: Inclusion Criteria:

Part A only:

- Healthy male subjects aged between 18 and 55 years.

Part B only:

* Healthy male and female subjects aged between 18 and 55 years.
* Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 1 pre-dose of the first period. They must consistently and correctly use a highly effective method of contraception, or be sexually inactive, or have a vasectomized partner.
* Women of nonchildbearing potential (i.e., postmenopausal, XY genotype, Turner syndrome, uterine agenesis).

Part A and B:

* Signed informed consent prior to any study-mandated procedure.
* Body mass index of 18.0 to 30.0 kg/m2 (inclusive) at Screening. Body weight at least 50.0 kg at Screening and prior to first study treatment administration.
* Healthy on the basis of medical history, physical examination, cardiovascular assessments and clinical laboratory tests.

Exclusion Criteria:

Part B only:

* Known hypersensitivity to moxifloxacin or any of its excipients.
* Pregnant or lactating women.
* Women planning to become pregnant.

Part A and B:

* Previous exposure to lucerastat.
* 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 rhythm disorders.

Primary outcomes:
measure: Part B: Placebo-corrected, change-from-baseline QTcF (ΔΔQTcF)
timeFrame: From 1 h pre-dose to 36 hour after study treatment administration (duration: up to 37 hours)
Secondary outcomes:
Other outcomes:
Locations:
city: Groningen
country: Netherlands
facility: QPS Netherlands B.V.
Study officials:
affiliation: Idorsia Pharmaceuticals Ltd.
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Mueller MS, Sidharta PN, Voors-Pette C, Darpo B, Xue H, Dingemanse J. The effect of the glucosylceramide synthase inhibitor lucerastat on cardiac repolarization: results from a thorough QT study in healthy subjects. Orphanet J Rare Dis. 2020 Oct 27;15(1):303. doi: 10.1186/s13023-020-01582-7.