Narcolepsy Type 1 (NT 1) Clinical Trial
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)
| Verified date | December 2023 |
| Source | Takeda |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
Adults with narcolepsy who have completed the TAK-994-1501 study will be able to take part in this study. The main aim of this study is to check if participants have side effects from TAK-994. Participants will take one of 3 different TAK-994 dose for 8 weeks. Then, half the participants will continue with their dose of TAK-994 and half will take a placebo. In this study, a placebo will look like a TAK-994 tablet but will not have any medicine in it. Participants will take TAK-994 or placebo for 4 weeks. Participants will visit the clinic for a final check-up 2 weeks after their last dose of TAK-994 or placebo. The study doctors will check for side effects from TAK-994 and placebo throughout the study. Participants will continue to record any narcolepsy symptoms as they did in Part B of the TAK 994-1501 study.
| Status | Terminated |
| Enrollment | 26 |
| Est. completion date | November 3, 2021 |
| Est. primary completion date | November 3, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 65 Years |
| Eligibility | Inclusion 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. Exclusion Criteria: 1. Participant has a clinically significant moderate or severe ongoing AE related to the study drug from the prior study. |
| Country | Name | City | State |
|---|---|---|---|
| Canada | West Ottawa Sleep Centre | Ottawa | Ontario |
| Canada | Jodha Tishon Inc. | Toronto | Ontario |
| Canada | Toronto Sleep Institute | Toronto | Ontario |
| Czechia | Fakultni nemocnice Hradec Kralove Dept of Neurologicka klinika | Hradec Kralove | |
| Czechia | Vseobecna fakultni nemocnice v Praze Dept of Neurologicka klinika 1.LF UK a VFN v Praze | Praha 2 | |
| Finland | Terveystalo Helsinki Uniklinikka 300186257 | Helsinki | |
| Finland | Turku University Hospital | Turku | |
| France | Hopital Roger Salengro - CHU Lille service de neurologie D | Lille Cedex | Nord |
| France | Hopital Gui de Chauliac Service de Neurologie | Montpellier | Herault |
| Hungary | SomnoCenter Budapest | Budapest | |
| Italy | Universita di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche | Bologna | |
| Italy | Ospedale San Raffaele (San Raffaele Turro) Clinica Neurologica- Div Malattie del Sonno | Milano | |
| Italy | Azienda Ospedaliera Universitaria Policlinico Tor Vergata U.O.C. Neurologia | Roma | |
| Italy | IRCCS Oasi Maria SS 300206751 | Troina | Enna |
| Japan | Koishikawa Tokyo Hospital Dept of Psychiatry | Bunkyo-ku | Tokyo-To |
| Japan | SOUSEIKAI PS Clinic Dept of Internal Medicine | Fukuoka-shi | Fukuoka-Ken |
| Japan | Jinyukai Kotorii Isahaya Hospital Dept of Psychiatry | Isahaya-shi | Nagasaki-Ken |
| Japan | Nihon University Itabashi Hospital Dept of Neuropsychiatry | Itabashi-ku | Tokyo-To |
| Japan | You Ariyoshi Sleep Clinic Dept of Psychiatry | Kitakyushu-shi | Fukuoka-Ken |
| Japan | Howakai Kuwamizu Hospital Dept of Internal Medicine | Kumamoto-shi | Kumamoto-Ken |
| Japan | Kurume University Hospital Dept of Neuropsychiatry | Kurume-shi | Fukuoka-Ken |
| Japan | Shunkaikai Inoue Hospital Dept of Respiratory Medicine | Nagasaki-shi | Nagasaki-Ken |
| Japan | Gokeikai Osaka Kaisei Hospital Dept of Sleep Medicine | Osaka-shi | Osaka-Fu |
| Japan | Kyowakai Hannan Hospital Dept of Psychiatry | Sakai-shi | Osaka-Fu |
| Japan | Yoyogi Sleep Disorder Center Dept of Psychiatry | Shibuya-ku | Tokyo-To |
| Japan | Sleep & Stress Clinic Dept of Psychiatry | Shinagawa-ku | Tokyo-To |
| Japan | Sleep Support Clinic Dept of Psychosomatic Medicine/Psychiatry | Shinagawa-ku | Tokyo-To |
| Japan | Sumida Hospital Phase I | Sumida-ku | Tokyo-To |
| Japan | Kaiseikai Kita Shin Yokohama Internal Medicine Clinic Dept of Internal Medicine | Yokohama-shi | Kanagawa-Ken |
| Korea, Republic of | Keimyung University Dongsan Hospital 300144594 | Daegu | |
| Korea, Republic of | The Catholic University of Korea, St. Vincent's Hospital 300187879 | Suwon-si | Gyeonggi-do |
| Spain | Hospital Clinic de Barcelona Servicio de Neurologia | Barcelona | |
| Spain | Hospital General de Castellon Servicio de Neurofisiologia | Castellon de la Plana | Castellon |
| Spain | Hospital Vithas Nuestra Senora de America Neurofisiologia Clinica | Madrid | |
| Spain | Hospital Universitario Araba Sede Santiago Sleep Unit | Vitoria | Alava |
| United States | Wright Clinical Research | Alabaster | Alabama |
| United States | NeuroTrials Research, Inc. 300116336 | Atlanta | Georgia |
| United States | CITrials - Bellflower | Bellflower | California |
| United States | Beth Israel Deaconess Medical Center CardioVascular Institute | Boston | Massachusetts |
| United States | Alpine Clinical Research Center 1024762 | Boulder | Colorado |
| United States | Medical University of South Carolina (MUSC) PARENT | Charleston | South Carolina |
| United States | Helene A. Emsellem, MD PC trading as "The Center for Sleep & Wake Disorders" 150119420 | Chevy Chase | Maryland |
| United States | CTI Clinical Research Center | Cincinnati | Ohio |
| United States | Intrepid Research | Cincinnati | Ohio |
| United States | St. Francis Medical Institute | Clearwater | Florida |
| United States | The Cleveland Clinic Foundation 100428 | Cleveland | Ohio |
| United States | Delta Waves Sleep Disorders and Research Center 300148510 | Colorado Springs | Colorado |
| United States | Bogan Sleep Consultants, LLC 150711087 | Columbia | South Carolina |
| United States | Research Carolina Elite | Denver | North Carolina |
| United States | Ohio Sleep Medicine and Neuroscience Institute 186 | Dublin | Ohio |
| United States | Fort Wayne Neurological Center 150711262 | Fort Wayne | Indiana |
| United States | Clinical Research of Gastonia | Gastonia | North Carolina |
| United States | Hawaii Pacific Neuroscience | Honolulu | Hawaii |
| United States | University of Kansas Medical Center Research Institute, Inc. University of Kansas Hospital | Kansas City | Kansas |
| United States | Santa Monica Clinical Trials | Los Angeles | California |
| United States | Sleep Practitioners, LLC Macon | Macon | Georgia |
| United States | Clinical Site Partners, LLC | Miami | Florida |
| United States | Sleep Medicine Specialists of South Florida | Miami | Florida |
| United States | Mayo Clinic Arizona 300151190 | Phoenix | Arizona |
| United States | Raleigh Neurology Associates 300209729 | Raleigh | North Carolina |
| United States | Raleigh Neurology Associates,300209729 | Raleigh | North Carolina |
| United States | Stanford School of Medicine | Redwood City | California |
| United States | Sleep Therapy & Research Center 300151246 | San Antonio | Texas |
| United States | Pacific Research Network, Inc 150118105 | San Diego | California |
| United States | SDS Clinical Trials, Inc. | Santa Ana | California |
| United States | Clinical Research Institute 300169881 | Stockbridge | Georgia |
| United States | Comprehensive Sleep Medicine Associates | Sugar Land | Texas |
| United States | JSV Clinical Research Study, Inc | Tampa | Florida |
| United States | Florida Pulmonary Research Institute, LLC 300127039 | Winter Park | Florida |
| United States | Respiratory Specialists Berks Schuylkill Respiratory Specialists Ltd | Wyomissing | Pennsylvania |
| Lead Sponsor | Collaborator |
|---|---|
| Takeda |
United States, Canada, Czechia, Finland, France, Hungary, Italy, Japan, Korea, Republic of, Spain,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Participants With at Least One Treatment Emergent Adverse Event (TEAE) During the Active Drug Extension Period | 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. | Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8) | |
| Primary | Number of Participants With at Least One Post-dose Markedly Abnormal Value (MAV) in Laboratory Test During the Active Drug Extension Period | 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. | Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8) | |
| Primary | Number of Participants With at Least One Post-dose MAV for Vital Signs During the Active Drug Extension Period | 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. | Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8) | |
| Primary | Number of Participants With at Least One Post-dose MAV for Electrocardiogram (ECG) Parameters During the Active Drug Extension Period | 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. | Up to 8 weeks in the Active Drug Extension Period (Weeks 1 to 8) | |
| Secondary | Number of Participants With at Least One TEAE During the Double-blind Randomized Withdrawal Period | 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. | Up to 4 weeks in the Double-blind Randomized Withdrawal Period (Weeks 9 to 12) | |
| Secondary | Number of Participants With at Least One Post-dose MAV in Laboratory Test During the Double-blind Randomized Withdrawal Period | Clinical laboratory tests included hematology, serum chemistry, and urinalysis. MAV criteria: Hemoglobin <0.8×LLN, >1.2×ULN; Hematocrit <0.8×LLN, >1.2×ULN; RBC count <0.8×LLN, >1.2×ULN; WBC count <0.5xLLN, >1.5xULN; Platelet count <75x10^9/L, >600x10^9/L; ALT >3xULN; AST >3xULN; GGT >3xULN; Alkaline phosphatase >3xULN; Total bilirubin >1.5xULN; Albumin <25 g/L; Total protein <0.8x LLN, >1.2xULN; Creatinine >1.5xULN; Blood urea nitrogen >40 mg/dL; Sodium <130 mEq/L, >150 mEq/L; Potassium <3.0 mmol/L, >5.3 mmol/L; CPK >3xULN; Glucose <50 mg/dL, >300 mg/dL; Calcium <7.7 mg/dL, >11.1 mg/dL. | Up to 4 weeks in the Double-blind Randomized Withdrawal Period (Weeks 9 to 12) | |
| Secondary | Number of Participants With at Least One Post-dose MAV for Vital Signs During the Double-blind Randomized Withdrawal Period | MAV criteria for vital signs were: Pulse <40 bpm, >115 bpm; Systolic blood pressure <90 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 Double-blind Randomized Withdrawal Period (Day 57 of this study). | Up to 4 weeks in the Double-blind Randomized Withdrawal Period (Weeks 9 to 12) | |
| Secondary | Number of Participants With at Least One Post-dose MAV for ECG Parameters During the Double-blind Randomized Withdrawal Period | MAV criteria for ECG were: Heart rate <40 bpm, >115 bpm; PR interval =80 msec, =200 msec; 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. | Up to 4 weeks in the Double-blind Randomized Withdrawal Period (Weeks 9 to 12) |