Open-label single dose trial to evaluate pharmacokinetics, safety, and acceptability/palatability of oveporexton in children from 6 years to less than 18 years of age with NT1

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

7 Jan 2026 → 22 May 2026

Decision date (initial)

2025-11-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Takeda Development Center Americas, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Others

To determine the plasma pharmacokinetic (PK) parameters of oveporexton in pediatric participants.

Secondary objectives 2

1. To evaluate the safety of oveporexton as measured by TEAE incidence.
2. To evaluate the acceptability/palatability of the oveporexton tablet as assessed by the Acceptability/Palatability Questionnaire.

Conditions and MedDRA coding

Narcolepsy with Cataplexy (Narcolepsy Type 1)

Study design 1 period

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003553-PIP01-23

Plan to share IPD

Yes

IPD plan description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. The participant has an ICSD-3 or ICSD-3-TR diagnosis of NT1.
2. Male or female participants aged 6 to 17 years, inclusive, at the time of signing the ICF.
3. The participant has a body mass index within the range 18 to 30 kg/m2
4. The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from radioimmunoassay indicate the participant’s CSF OX/hypocretin-1 concentration is <110 pg/mL
5. The participant is judged by the investigator to be sufficiently healthy to participate in the trial based on clinical evaluations including laboratory safety tests, medical history, physical examination, 12-lead ECG, and vital sign measurements performed at the screening visit and before the first dose of trial intervention.
6. Parent or legal guardian – and participants old enough to understand - have expressed a willingness to participate in and complete the trial and signed and dated the ICF after getting acquainted with the participant’s information sheet, prior to beginning protocol-required procedures.

Exclusion criteria 4

1. Participant is unable to refrain from using excluded medications
2. The participant has a positive urine screen result for drugs of abuse and/or positive alcohol test result at screening or at Day -1.
3. Participant has a positive pregnancy test result at screening or is breastfeeding.
4. Participant has a known hypersensitivity to any component of the formulation of oveporexton or related compounds.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Maximum observed concentration (Cmax). Time to reach maximum observed concentration (tmax). Area under the concentration-time curve from time 0 to infinity (AUC∞), calculated using the observed value of the last quantifiable concentration.

Secondary endpoints 2

1. Occurrence of at least 1 TEAE.
2. Participants rating acceptability and palatability.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

Sponsor organisation

Takeda Development Center Americas Inc.

Address

500 Kendall Street

City

Cambridge

Postcode

02142-1108

Country

United States

Scientific contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 6

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Serious breaches 1 · Art. 52 CTR

Application history

1 submissions — initial application plus substantial / non-substantial modifications