A Study of TAK-994 in Adults with Narcolepsy

JRCT ID: jRCT2071210015

Registered date:28/04/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Narcolepsy Type 1 (NT 1)
• Date of first enrollment: 30/04/2021
• Target sample size: 26
• Countries of recruitment: United States,Japan,Canada,Japan,Italy,Japan,France,Japan,Spain,Japan,Hungary,Japan,Czech Republic,Japan,Finland,Japan,Netherlands,Japan,South Korea,Japan
• Study type: Interventional

Intervention(s)

Active Drug Extension Period: TAK-994 Dose 1 - 3
TAK-994 dose 1 - 3, tablets, orally, from Day 1 (Day 57 of previous study) to Day 56.

Double-blind Randomized Withdrawal Period: TAK-994 Dose 1 - 3
Following the Active Drug Extension Period, participants randomized to active treatment Dose 1 - 3 will continue to receive same dose (TAK-994, dose 1-3, tablets, orally) from Day 57 to Day 84.

Double-blind Randomized Withdrawal Period: Placebo
Following the Active Drug Extension Period participants will receive placebo-matching tablets for 4 weeks (from Day 57 to Day 84).

Outcome(s)

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 Week 8 in the Active Drug Extension Period (Weeks 1 to 8)
An 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 and serum chemistry, 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 <0.5*LLN, >1.5*ULN; Platelet count <75*10^9/litre (L), >600*10^9/L; alanine aminotransferase (ALT) >3*ULN; aspartate aminotransferase (AST) >3*ULN; gamma-glutamyl transferase (GGT) >3*ULN; Alkaline phosphatase >3*ULN; Total bilirubin >1.5*ULN; Albumin <25 grams per litre (g/L); Total protein <0.8* LLN, >1.2*ULN; Creatinine >1.5*ULN; Blood urea nitrogen >40 milligrams per decilitres (mg/Dl); Sodium <130 milliequivalents per litre (mEq/L), >150 mEq/L; Potassium <3.0 millimoles per litre (mmol/L), >5.3 mmol/L; creatine phosphokinase (CPK) >3*ULN; Glucose <50 mg/dL, >300 mg/dL; Calcium <7.7 mg/dL, >11.1 mg/dL.
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 >20, >30 mmHg, Body temperature >38.5 degree Celsius, Respiratory Rate >21 breath/minute.

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.

Secondary Outcome

1.Number of Participants with at Least One TEAE During the Randomized Withdrawal Period
Time Frame: Up to 4 weeks in the Randomized Withdrawal Period (Weeks 9 to 12)
An 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, 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 MAV for Laboratory Test During the Randomized Withdrawal Period
Time Frame: Up to 4 weeks in the Randomized Withdrawal Period (Weeks 9 to 12)
Clinical laboratory tests included hematology and serum chemistry, 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 <0.5*LLN, >1.5*ULN; Platelet count <75*10^9/litre (L), >600*10^9/L; alanine aminotransferase (ALT) >3*ULN; aspartate aminotransferase (AST) >3*ULN; gamma-glutamyl transferase (GGT) >3*ULN; Alkaline phosphatase >3*ULN; Total bilirubin >1.5*ULN; Albumin <25 grams per litre (g/L); Total protein <0.8* LLN, >1.2*ULN; Creatinine >1.5*ULN; Blood urea nitrogen >40 milligrams per decilitres (mg/Dl); Sodium <130 milliequivalents per litre (mEq/L), >150 mEq/L; Potassium <3.0 millimoles per litre (mmol/L), >5.3 mmol/L; creatine phosphokinase (CPK) >3*ULN; Glucose <50 mg/dL, >300 mg/dL; Calcium <7.7 mg/dL, >11.1 mg/dL.

3.Number of Participants with at Least One Post-dose MAV for Vital Signs During the Randomized Withdrawal Period
Time Frame: Up to 4 weeks in the Randomized Withdrawal Period (Weeks 9 to 12)
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 >20, >30 mmHg, Body temperature >38.5 degree Celsius, Respiratory Rate >21 breath/minute. Baseline for this outcome measure is Day 1 of the Double-blind Randomized Withdrawal Period.

4.Number of Participants with at Least One Post-dose MAV for ECG Parameters During the Randomized Withdrawal Period
Time Frame: Up to 4 weeks in the Randomized Withdrawal Period (Weeks 9 to 12)
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.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 65age old
• Gender: Both
• Include criteria: 1. Participant with a diagnosis of Narcolepsy Type 1 (NT1) who has completed TAK-994-1501 Part B before enrollment (which will occur immediately following the final TAK-994-1501 assessments), and for whom the investigator has no clinical objection they be enrolled.
• Exclude criteria: 1. Participant has a clinically significant moderate or severe ongoing adverse event (AE) related to the study drug from the prior study.

Related Information

• Primary Sponsor: Nonomura Hidenori
• Secondary ID(s): NCT04820842,2021-000251-39