A Trial to Evaluate the Long-term Safety and Tolerability of TAK-861

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

27 Apr 2023 → ongoing

Decision date (initial)

2025-02-23

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Takeda Development Center Americas, Inc.

External identifiers

EU CT number

2023-508462-15-00

EudraCT number

2022-002965-13

WHO UTN

U1111-1283-1888

ClinicalTrials.gov

NCT05816382

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy

To evaluate the long-term safety and tolerability of TAK-861.

Secondary objectives 3

1. To assess the effect of TAK-861 on excessive daytime sleepiness (EDS) as assessed by the mean sleep latency from the Maintenance of Wakefulness Test (MWT).
2. To assess the effect of TAK-861 on EDS as measured by the Epworth Sleepiness Scale (ESS) total score.
3. To assess the effect of TAK-861 on cataplexy as assessed by the weekly cataplexy rate (WCR)

Conditions and MedDRA coding

Narcolepsy with Cataplexy (Narcolepsy Type 1)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Participant with a diagnosis of NT1 who has completed a controlled Trial with TAK-861, who meet all inclusion criteria and does not meet any exclusion criteria, and for whom the investigator has no clinical objection to their enrollment.

Exclusion criteria 12

1. Participant has a treatment-related adverse event (TEAE) that remains severe at the time of rollover related to the study drug from the parent study or discontinued because of TEAEs in the parent study.
2. The participant has a positive urine screen result for drugs of abuse and/or positive alcohol test result at screening or at Day -2 if a dosing gap > 7 days is present. An exception at screening is made for stimulants or other drugs the participant has been prescribed.
3. Participant has a risk of suicide according to endorsement of item 4 or 5 on the Columbia Suicide Severity Rating Scale Since Last Visit (C-SSRS) on any visit in the prior study or has positive answers on item 4 or 5 on the C-SSRS Screening/Baseline (based on the past year) during the screening assessment for participants with a dosing gap.
4. Participant has alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >1.5 times the upper limit of normal (ULN) at multiple visits in the parent study and the findings are of clinical significance, per investigator or sponsor opinion, or ALT/AST >1.5 times ULN during the screening period for participants with a dosing gap.
5. Participant has a current medical disorder, other than narcolepsy with or without cataplexy, associated with excessive daytime sleepiness (EDS).
6. Participant has current active major depressive episode (MDE) or has had an active MDE in the past 6 months.
7. Participant has developed (within the last 6 months) gastrointestinal disease that is expected to influence the absorption of drugs (i.e., a history of malabsorption, esophageal reflux, peptic ulcer disease, erosive esophagitis, frequent [more than once per week] occurrence of heartburn, or any surgical intervention).
8. Participant has epilepsy or history of seizure.
9. Participant has any other medical condition, such as anxiety, depression, heart disease, or significant hepatic, pulmonary, or renal disease, that requires them to take excluded medications.
10. Participant has a history of cerebral ischemia, transient ischemic attack (<5 years ago), or cerebral hemorrhage.
11. Participant has a history of myocardial infarction, clinically significant coronary artery disease, clinically significant angina, clinically significant cardiac rhythm abnormality, or heart failure.
12. Participant has a history of cancer in the past 5 years (does not apply to participants with carcinoma in situ that has been resolved without further treatment, or basal cell skin cancer; these participants may be included after approval by the sponsor or designee).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of at least 1 treatment-emergent adverse event (TEAE).

Secondary endpoints 3

1. Change from baseline in the parent study in MWT mean sleep latency.
2. Change from baseline in the parent study in ESS total score
3. Change from baseline in the parent study in WCR using the patient reported Cataplexy Diary

Investigational products

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

Test 2

Placebo 1

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 10

Locations

11 EU/EEA countries · 32 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 111 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

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