Clinical Trials Register

Summary
EudraCT Number:	2022-002965-13
Sponsor's Protocol Code Number:	TAK-861-2003
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-12-14
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2022-002965-13

A.3

Full title of the trial

A Long-term Extension Study to Evaluate the Safety and Tolerability of TAK-861 in Participants With Selected Central Hypersomnia Conditions

Studio di estensione a lungo termine volto a valutare la sicurezza e la tollerabilità di TAK 861 in partecipanti con condizioni selezionate di ipersonnia di origine centrale

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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

Uno studio per valutare la sicurezza e la tollerabilità a lungo termine di TAK-861

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

TAK-861-2003

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1283-1888

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	TAKEDA DEVELOPMENT CENTER AMERICAS INC.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Takeda Development Center Americas, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Takeda Development Center Americas, Inc.
B.5.2	Functional name of contact point	Study Registration Call Center
B.5.3	Address:
B.5.3.1	Street Address	95 Hayden Avenue
B.5.3.2	Town/ city	Lexington
B.5.3.3	Post code	MA 02421
B.5.3.4	Country	United States
B.5.4	Telephone number	+18778253327
B.5.5	Fax number	000000
B.5.6	E-mail	medinfoUS@takeda.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	TAK-861
D.3.2	Product code	[TAK-861]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	TAK-861
D.3.2	Product code	[TAK-861]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	TAK-861
D.3.2	Product code	[TAK-861]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 4
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	TAK-861
D.3.2	Product code	[TAK-861]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	TAK-861
D.3.9.1	CAS number	2460722-04-5
D.3.9.2	Current sponsor code	TAK-861
D.3.9.4	EV Substance Code	SUB293348
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Narcolepsy with Cataplexy (Type 1) and Narcolepsy without Cataplexy (Type 2)

Narcolessia con cataplessia (Tipo 1) e narcolessia senza cataplessia (Tipo 2)

E.1.1.1

Medical condition in easily understood language

Narcolepsy with Cataplexy (Type 1) and Narcolepsy without Cataplexy (Type 2)

Narcolessia con cataplessia (Tipo 1) e narcolessia senza cataplessia (Tipo 2)

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10028713
E.1.2	Term	Narcolepsy
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

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

Valutazione della sicurezza e della tollerabilità a lungo termine di TAK-861

E.2.2

Secondary objectives of the trial

• 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)
• To assess the effect of TAK-861 on EDS as measured by the Epworth Sleepiness Scale (ESS) total score.
• To assess the effect of TAK-861 on cataplexy as assessed by the weekly cataplexy rate (WCR) (participants with narcolepsy type 1 [NT1] only).

• Valutazione dell’effetto di TAK-861 sull’eccessiva sonnolenza diurna (excessive daytime sleepiness, [EDS])
valutata dalla latenza media del sonno mediante il test di mantenimento dello stato di veglia (maintenance of wakefulness test, [MWT])
• Valutazione dell’effetto di TAK-861 sull’EDS misurato dal punteggio totale della scala di sonnolenza di Epworth (Epworth Sleepiness Scale, [ESS]).
• Valutazione dell’effetto di TAK-861 sulla cataplessia valutato dal tasso di cataplessia settimanale (weekly cataplexy rate, [WCR]) (solo partecipanti con narcolessia di tipo 1 [NT1]).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Informed Consent
1. Participant is willing and able to understand and fully comply with
study procedures and requirements (including digital tools and
applications), in the opinion of the investigator.
2. Participant has provided informed consent (that is, in writing,
documented via a signed and dated informed consent form (ICF) and/or
electronic consent [eConsent]) and any required privacy authorization
before the initiation of any study procedures.
Type of Participant and Disease Characteristics
3. Participant with a diagnosis of narcolepsy who has completed a
controlled study with TAK-861 (including participants diagnosed with
NT1 or NT2) and for whom the investigator has no clinical objection to
their enrollment. Additionally, a rare exception may be granted by the
sponsor or designee for a participant who was unable to complete a
previous TAK-861 controlled study conducted in participants with
narcolepsy.
Contraception
4. The participant agrees to follow the birth control requirements

Consenso informato
1. Secondo il parere dello sperimentatore, il partecipante è disposto e in grado di comprendere e rispettare pienamente le procedure e i requisiti dello studio (inclusi gli strumenti e le applicazioni digitali).
2. Il partecipante ha fornito il consenso informato (ovvero, per iscritto, documentato tramite un modulo di consenso informato (informed consent form, [ICF]) firmato e datato e/o tramite consenso elettronico [eConsent]) e qualsiasi autorizzazione alla privacy richiesta prima dell’inizio di qualsiasi procedura di studio.
Tipologia di partecipante e caratteristiche della malattia
3. - Partecipante con diagnosi di narcolessia che ha completato uno studio controllato con TAK-861 (compresi i partecipanti con diagnosi di NT1 o NT2) e per il quale lo sperimentatore non abbia obiezioni cliniche al suo arruolamento. Inoltre, lo sponsor o il designato può concedere una rara eccezione per un partecipante che non sia stato in grado di completare un precedente studio controllato con TAK-861 condotto su partecipanti con narcolessia.

Contraccezione
4. Il partecipante si impegna a seguire i requisiti relativi alla contraccezione

E.4

Principal exclusion criteria

Medical Conditions
1. Participant has a moderate or severe ongoing (TEAE) related to the
study drug from the parent study or discontinued because of TEAEs in
the parent study.
Prior/Concomitant Therapy
2. Participant used disallowed medication during the parent study and is
unable to refrain from or anticipates using excluded medications.
Diagnostic Assessments
3. Participant has a (BP) >160 mm Hg (systolic) or >100 mm Hg
(diastolic) at the final collection point in any prior controlled study
without a dosing gap. The participant may have a history of hypertension
and be on antihypertensive medication treatment as long as the BP
values are below these criteria. BP measurements should be obtained
after the participant has been semirecumbent (lying down with the head
of the bed at 30 degrease) for a minimum of 5 minutes. BP will be
repeated 3 times, with a minimum of 2 minutes between assessments.
The median BP (of 3 assessments) obtained will be used for assessing
participant eligibility.
4. Participant has a resting HR outside of the range of 40 to 100 beats
per minute, confirmed on repeat testing within a maximum of 30
minutes at assessment at screening.
5. Participant has an ECG with a (QTcF) interval>450 ms (men) or >470
ms (women) at screening.
6. Participant has (ALT) and (AST) >1.5 times the (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.
7. Participant's renal creatinine clearance is =50 mL/min.
8. Participant has a positive urine screen for drugs of abuse (findings
confirmed) and/or positive alcohol test during any visit in their prior
TAK-861 study, or during the screening period for participants with a
dosing gap. Products containing cannabidiol are allowed throughout the
study, at the discretion of the investigator.
9. Participant has a positive pregnancy test at screening or is a
lactating/breastfeeding woman.
10. Participant has a risk of suicide according to endorsement of item 4
or 5 on the C-SSRS Since Last Visit (C-SSRS) on any visit in the prior
study, or has positive answers on item 4 or 5 on the Screening/Baseline
C-SSRS Lifetime (based on the past year) during the screening
assessment for participants with a dosing gap.
Other Exclusion Criteria
11. Participant consumes excessive amounts of caffeine, defined as
greater than 600 mg of caffeine of coffee, tea, cola, energy drinks, or
other caffeinated beverages per day (1 cup of coffee is approximately
120 mg).
12. Participant is unwilling to refrain from drivingand/or operating
dangerous or hazardous machinery during times of heightened
sleepiness or fatigue as well as during times of medication
weaning/changes, or is unwilling to adhere to local regulations and any
PI guidance restricting driving.
Participants with a Dosing GapUp to 3 Months
Additional exclusion criteria for participants who have completed their
parent study and who are not able to directly roll over into the current
study (ie, for whom there is a dosing gap), are as follows:
13. Participant has participated in another investigational drug study, in
which they received the investigational drug other than TAK-861, within
60 days (or 6 months if participant may have received an investigational
biologic product). The interval window from the parent study will be
derived from the date of the last study procedure in the parent study to
the screening visit of TAK-861-2003.
14. Participant is unable to discontinue, or refrain from using excluded
medications for the duration of the study, including those used for the
treatment of narcolepsy. Participant must be willing to complete
specified washout periods before the first dose of study drug
Medical Conditions

For further exclusion criteria no 15-27 please refer to the protocol

Condizioni mediche
1. - Il partecipante ha in atto un moderato o grave evento avverso emergente dal trattamento (treatment-emergent adverse event, [TEAE]) correlato al farmaco in studio dallo studio originario o ha interrotto l’assunzione del farmaco a causa di TEAE nello studio originario.
Terapia pregressa/concomitante
2. Il partecipante ha utilizzato farmaci non consentiti durante lo studio originario e non è in grado di rinunciare a farmaci esclusi o prevede di utilizzarli. Valutazioni diagnostiche
3. - Il partecipante ha una pressione arteriosa (blood pressure, [BP]) >160 mm Hg (sistolica) o >100 mm Hg (diastolica) al momento del prelievo finale in eventuali precedenti studi controllati senza un intervallo nella somministrazione. Il partecipante può avere una anamnesi di ipertensione ed essere in trattamento con farmaci antipertensivi fintanto che i valori della BP risultino inferiori a questi criteri. Le misurazioni della BP devono essere ottenute dopo che il partecipante è stato semisdraiato (sdraiato con la testiera del letto a 30 gradi) per un minimo di 5 minuti. La misurazione della BP verrà ripetuta 3 volte, con un minimo di 2 minuti tra le valutazioni. Per valutare l’idoneità dei partecipanti si utilizzerà il valore mediano di BP (di 3 valutazioni) ottenuto.
4. - Il partecipante ha una frequenza cardiaca (heart rate, [HR]) a riposo al di fuori dell’intervallo dei 40-100 battiti al minuto, confermata da test ripetuti entro un massimo di 30 minuti alla valutazione allo screening.
5. Il partecipante ha un ECG con un intervallo (QTcF)> 450 ms (uomini) o > 470 ms (donne) allo screening.
6. - Il partecipante ha valori di alanina aminotransferasi (alanine aminotransferase, [ALT]) e aspartato aminotransferasi (aspartate aminotransferase, [ALT]) >1,5 volte il limite superiore della norma (upper limit of normal, [ULN]) in più visite nello studio originario e i risultati sono di rilevanza clinica, secondo l’opinione dello sperimentatore o dello sponsor, o ALT/AST >1,5 volte l’ULN durante il periodo di screening per partecipanti con un intervallo nella somministrazione.
7. La clearance renale della creatinina del partecipante è =50 mL/min.
8. Il partecipante ha un esame tossicologico sulle urine positivo per droghe d’abuso (risultati confermati) e/o un alcol test positivo durante qualsiasi visita nel precedente studio con TAK-861 o durante il periodo di screening per i partecipanti con un intervallo nella somministrazione. I prodotti contenenti cannabidiolo sono consentiti durante lo studio, a discrezione dello sperimentatore.
9. - La partecipante ha un test di gravidanza positivo allo screening o è una donna che produce latte/allatta al seno.
10. Il partecipante ha, in qualsiasi visita nello studio precedente, un rischio di suicidio secondo quanto sostenuto al punto 4 o 5 della Scala della Columbia University per la valutazione della gravità del rischio di suicidio (Columbia-Suicide Severity Rating Scale, [C-SSRS]) “Dall’ultima visita” (C-SSRS), o, durante la valutazione allo screening per i partecipanti con un intervallo nella somministrazione, ha risposto positivamente al punto 4 o 5 sulla C-SSRS “Nel corso della vita” allo Screening/al basale (sulla base dell’anno passato).
Altri criteri di esclusione
11. Il partecipante consuma quantità eccessive di caffeina, definite come superiori a 600 mg di caffeina al giorno, da caffè, tè, cola, bevande energetiche o altre bevande contenenti caffeina (1 tazza di caffè è di circa 120 mg).
12. Il partecipante non è disposto a rinunciare alla guida e/o a utilizzare
13. Il partecipante ha preso parte a un altro studio su farmaco sperimentale, in cui gli è stato somministrato il farmaco sperimentale diverso da TAK-861, entro 60 giorni (o 6 mesi se il partecipante potrebbe aver ricevuto un prodotto biologico sperimentale). La finestra dell’intervallo dallo studio originario sarà desunta dalla data dell’ultima procedura di studio nello studio originario alla visita di screening di TAK-861-2003.
14. Il partecipante non è in grado di interrompere o di rinunciare a utilizzare i farmaci esclusi per tutta la durata dello studio, compresi quelli utilizzati per il trattamento della narcolessia. Prima di assumere la prima dose del farmaco in studio, il partecipante deve essere disposto a completare i periodi di sospensione specificati Condizioni mediche

Per ulteriori criteri di esclusione N. 15-27 si rimanda al protocollo

E.5 End points

E.5.1

Primary end point(s)

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

• Insorgenza di almeno 1 evento avverso emergente dal trattamento (TEAE).

E.5.1.1

Timepoint(s) of evaluation of this end point

Day 395, 425, 485, 515, 575, 605, 665, 695

Giorno 395, 425, 485, 515, 575, 605, 665, 695

E.5.2

Secondary end point(s)

• Change from baseline in the parent study in MWT mean sleep latency.
• Change from baseline in the parent study in ESS total score
• Change from baseline in the parent study in WCR using the patientreported
cataplexy diary (participants with NT1 only)

• Variazione rispetto al basale nello studio originario della latenza media del sonno misurata mediante l’MWT.
• Variazione rispetto al basale nello studio originario nel punteggio totale della ESS
• Variazione rispetto al basale nello studio originario del WCR utilizzando il diario della cataplessia riportato dal paziente (solo partecipanti con NT1)

E.5.2.1

Timepoint(s) of evaluation of this end point

- Day 182
- Day 14, 28, 56, 84, 182, 266, 364, 448, 546, 630. 728
- Day 69 to 84; 167 to 182; 251 to 266; 350to 364; 531 to 546; 715 to
728; 728 to 735

- Giorno 182
- Giorno 14, 28, 56, 84, 182, 266, 364, 448, 546, 630. 728
- Dal Giorno 69 al Giorno 84; da 167 a 182; da 251 a 266; da 350 a 364; da 531 a 546; da 715 a 728; 728 a 735

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

tolerability

tollerabilità

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

Yes

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

dose blind

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

placebo to blind dose

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Japan

United States

Finland

France

Sweden

Netherlands

Spain

Switzerland

Germany

Italy

Norway

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The end of study is defined as the final date on which data were or are expected to be collected, ie, the last visit of the last participant in the study

La fine dello studio è definita come la data finale in cui i dati sono stati raccolti o dovrebbero essere stati raccolti, cioè l’ultima visita dell’ultimo partecipante allo studio

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

135

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

160

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Best standard of care at investigator's discretion

Miglior standard di cura a discrezione dello sperimentatore

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-03-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-02-16

P. End of Trial
P.	End of Trial Status	Ongoing

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