A Study of TAK-994 in Adults With Narcolepsy

Study at a glance

From the registry record, last updated by the sponsor on 26 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

A Dose-Blind Extension Study With Double-blind, Placebo-Controlled, Randomized Withdrawal Period to Evaluate the Safety and Explore the Pharmacokinetics and Pharmacodynamics of TAK-994 in Adults With Narcolepsy With Cataplexy (Narcolepsy Type 1)

ClinicalTrials.gov ID

NCT04820842

Acronym / sponsor ID

TAK-994-1504

Conditions

Narcolepsy Type 1 (NT 1)

Keywords

Drug Therapy

Phase

Phase 2

Study type

Interventional

Recruitment status

TerminatedReason stopped: A safety signal has emerged in Phase 2 studies of TAK-994. As an immediate precautionary measure, Takeda has suspended dosing of patients and has decided to stop Phase 2 studies early.

Enrolment

26 participants (actual)

Lead sponsor

TakedaIndustry (pharma, biotech, devices)

Study start

30 Apr 2021 (actual)

Primary completion

3 Nov 2021 (actual)

Study completion

3 Nov 2021 (actual)

First posted

29 Mar 2021 (actual)

Results

Posted 26 Dec 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Randomised

Intervention model

Parallel groups

Masking

Quadruple blind (participant, care provider, investigator, outcomes assessor)

Number of arms

7

Who may qualify

Ages

18 to 65 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Number of Participants With at Least One Treatment Emergent Adverse Event (TEAE) During the Active Drug Extension Period

   Time frame: Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8)

   An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participants administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug.

2. Number of Participants With at Least One Post-dose Markedly Abnormal Value (MAV) in Laboratory Test During the Active Drug Extension Period

   Time frame: Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8)

   Clinical laboratory tests included hematology, serum chemistry, and urinalysis. MAV criteria: Hemoglobin <0.8×lower limit of normal (LLN), >1.2×upper limit of normal (ULN); Hematocrit <0.8×LLN, >1.2×ULN; Red blood cells (RBC) count <0.8×LLN, >1.2×ULN; White blood cells (WBC) count <0.5xLLN, >1.5xULN; Platelet count <75x10^9/liter (L), >600x10^9/L; alanine aminotransferase (ALT) >3xULN; aspartate aminotransferase (AST) >3xULN; gamma-glutamyl transferase (GGT) >3xULN; Alkaline phosphatase >3xULN; Total bilirubin >1.5xULN; Albumin <25 grams per liter (g/L); Total protein <0.8xLLN, >1.2xULN; Creatinine >1.5xULN; Blood urea nitrogen >40 milligrams per deciliters (mg/dL); Sodium <130 milliequivalents per liter (mEq/L), >150 mEq/L; Potassium <3.0 millimoles per liter (mmol/L), >5.3 mmol/L; creatine phosphokinase (CPK) >3xULN; Glucose <50 mg/dL, >300 mg/dL; Calcium <7.7 mg/dL, >11.1 mg/dL. Only categories with at least one participant with event are reported.

3. Number of Participants With at Least One Post-dose MAV for Vital Signs During the Active Drug Extension Period

   Time frame: Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8)

   MAV criteria for vital signs were: Pulse <40 beats per minute (bpm), >115 bpm; Systolic blood pressure <90 millimeters of mercury (mmHg), ≥160 mmHg; Diastolic blood pressure <50 mmHg, ≥100 mmHg, Systolic or Diastolic blood pressure change of >20, >30 mmHg from Baseline, Body temperature >38.5 degree Celsius, Respiratory Rate >21 breath/minute. Only categories with at least one participant with event are reported. Baseline for this outcome measure is Day 1 of the Active Drug Extension Period.

4. Number of Participants With at Least One Post-dose MAV for Electrocardiogram (ECG) Parameters During the Active Drug Extension Period

   Time frame: Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8)

   MAV criteria for ECG were: Heart rate <40 bpm, >115 bpm; PR interval ≤80 milliseconds (msec), ≥200 msec; QT interval with Fridericia correction method (QTcF) Interval ≤300 msec, >500 msec or ≥30 msec change from baseline and >450 msec; QRS duration ≤80 msec, ≥180 msec. Only categories with at least one participant with event are reported.

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 26 Dec 2023.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• Derived from this study: PubMed 37494485Dauvilliers Y, Mignot E, Del Rio Villegas R, Du Y, Hanson E, Inoue Y, Kadali H, Koundourakis E, Meyer S, Rogers R, Scammell TE, Sheikh SI, Swick T, Szakacs Z, von Rosenstiel P, Wu J, Zeitz H, Murthy NV, Plazzi G, von Hehn C. Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1. N Engl J Med. 2023 Jul 27;389(4):309-321. doi: 10.1056/NEJMoa2301940.
• To obtain more information on the study, click here/on this linkLink provided by the sponsor.
• Individual participant dataSharing planned: Yes. 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.

Related trials for Narcolepsy Type 1 (NT 1)

• Active, not recruitingObservational study

  Radar Sleep Monitoring in Type 1 Narcolepsy: Feasibility

  Narcolepsy Type 1 (NT 1), Hypersomnolence Disorder

• RecruitingPhase 2

  Extended-release Sodium Oxybate in Children

  Narcolepsy Type 1 (NT 1)

• Recruiting

  A Novel Approach to Manage Symptoms of Narcolepsy and Idiopathic Hypersomnia

  Idiopathic Hypersomnia, Narcolepsy Type 1 (NT 1)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

70 sites in 10 countries: see every site and its status.