A Trial of TAK-861 for the Treatment of Narcolepsy With Cataplexy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: TAK-861, Placebo.
- Who it may be relevant to
- Registry conditions: Narcolepsy Type 1 (NT1), Narcolepsy With Cataplexy. Basic parameters: 16 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, France, Germany, Italy, Netherlands +2
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Double-blind, Placebo-Controlled, Randomized Withdrawal Trial to Evaluate the Efficacy and Safety of TAK-861 for the Treatment of Narcolepsy With Cataplexy (Narcolepsy Type 1)
Overview
The main aim of this study is to assess how effective TAK-861 is for treating narcolepsy type 1 and if this effect is maintained over time. Participants will take TAK-861 for a few months and if they meet certain criteria, they will be randomly assigned (by chance, like flipping a coin) to continue taking TAK-861 or take placebo (fake medicine) for up to 4 weeks to see if their narcolepsy symptoms return.
Detailed description
The drug being tested in this study is called TAK-861. This study will look at how effective TAK-861 is for the treatment of narcolepsy type 1 and how well this effect is maintained over time. This study also evaluates how safe TAK-861 is and what adverse events may be associated with taking the drug and stopping the drug in participants with NT1.
The study will enroll approximately 88 participants. All participants will take TAK-861 during the open-label (OL) treatment period. Participants who meet certain criteria at the end of the OL Treatment Period will be randomized to one of two treatment groups for the up to 4-week double-blind randomized withdrawal (RW) Period. Participants will be randomized to one of the following treatment groups during the Double-blind RW Period:
1. TAK-861 (same dose participant was taking at the end of the OL Treatment Period) 2. Placebo
The randomized withdrawal period may last up to 4 weeks. Participants whose NT1 symptoms get worse during the RW period and whose Epworth Sleepiness Scale score rises above a certain score will stop the treatment and be invited to continue in a separate long term extension study. Participants who choose not to take part in the extension study will be followed-up for 4 weeks after their last dose of study drug. This multi-center trial will be conducted globally.
Interventions
- Drug TAK-861
TAK-861 tablets - Drug Placebo
TAK-861-matching placebo tablets
Primary outcome measures
- Time to Loss of Response in the Epworth Sleepiness Scale (ESS) Score During the Up to 4-Week RW Period [Time frame: Up to 4-week RW period]
Secondary outcome measures (8)
- Change From the End of the OL Treatment Period to Week 2 of the RW Period on the Maintenance of Wakefulness Test (MWT) [Time frame: From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)]
- Weekly Cataplexy Rate (WCR) at Week 2 of the RW Period [Time frame: Week 2 of the RW period]
- Change from the End of the OL Treatment Period to Week 2 of the RW Period in Mean Number of Lapses on the Psychomotor Vigilance Test (PVT) [Time frame: From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)]
- Number of Participants Reporting Much or Very Much Worse in Patient Global Impression of Change (PGI-C) Score at Week 2 of the RW Period [Time frame: Week 2 of the RW period]
- Change from the End of the OL Treatment Period to Week 2 of the RW Period in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score [Time frame: From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)]
- Change from the End of the OL Treatment Period to Week 2 of the RW Period in Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores [Time frame: From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)]
- Change from the End of the OL Treatment Period to Week 2 of the RW Period in Number of Correct Responses on the International Digit Symbol Substitution Test-symbols (iDSST-s) [Time frame: From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)]
- Number of Participants With At Least One Treatment-Emergent Adverse Event (TEAE) [Time frame: From Screening to End of study (approximately 31 weeks)]
Eligibility criteria
Inclusion criteria
- The participant has a body mass index (BMI) within the range 18 to 40 kilograms per square meter (kg/m\^2).
- The participant has an International Classification of Sleep Disorders, Third Edition (ICSD-3) or International Classification of Sleep Disorders, Third Edition, Text Revision (ICSD-3-TR) diagnosis of NT1.
- The participant is positive for the human leukocyte antigen (HLA) genotype Major Histocompatibility Complex, Class II, DQ Beta 1 (HLA-DQB1\*06:02) or results from radioimmunoassay indicate the participant's CSF OX/hypocretin-1 concentration is ≤110 pg/mL (or less than one-third of the mean values obtained in normal participants within the same standardized assay).
Exclusion criteria
- The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.
- The participant a) has a history of myocardial infarction, b) has a history of clinically significant hepatic disease, thyroid disease, coronary artery disease, cardiac rhythm abnormality or heart failure, or c) has any medical condition (such as unstable cardiovascular, pulmonary, renal or gastrointestinal disease.
- The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs.
- The participant has a history of cancer in the past 5 years.
- The participant has a clinically significant history of head injury or head trauma.
- The participant has a history of epilepsy, seizure, or convulsion (except for a single febrile seizure in childhood).
- The participant has a history of cerebral ischemia, transient ischemic attack (less than 5 years from screening), intracranial aneurysm, or arteriovenous malformation.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 11 centers
- Takeda Site 15 — Redwood City
- Takeda Site 19 — Brandon
- Takeda Site 22 — Miami
- Takeda Site 23 — Southfield
- Takeda Site 20 — Middletown
- Takeda Site 24 — Durham
- Takeda Site 25 — Winston-Salem
- Takeda Site 16 — Cincinnati
- … and 3 more centers
Spain · 4 centers
- Takeda Site 12 — Vitoria-Gasteiz
- Takeda Site 11 — Alzira
- Takeda Site 10 — Barcelona
- Takeda Site 9 — Madrid
France · 3 centers
- Takeda Site 1 — Montpellier
- Takeda Site 2 — Paris
- Takeda Site 3 — Toulouse
Germany · 2 centers
- Takeda Site 4 — Schwerin
- Takeda Site 5 — Berlin
Italy · 2 centers
- Takeda Site 6 — Bologna
- Takeda Site 7 — Rome
Switzerland · 2 centers
- Takeda Site 13 — Barmelweid
- Takeda Site 14 — Bern
Netherlands · 1 center
- Takeda Site 8 — Heemstede
Identifiers
NCT: NCT07363720 · TAK-861-3003 · 2024-519466-44-00