Clinical Trials Register

Summary
EudraCT Number:	2011-004520-35
Sponsor's Protocol Code Number:	VP-VEC-162-3204
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2012-08-27
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

Germany - BfArM

A.2

EudraCT number

2011-004520-35

A.3

Full title of the trial

AN EXTENSION OPEN-LABEL SAFETY STUDY OF A 24-MONTH 20MG DOSE REGIMEN OF TASIMELTEON FOR THE TREATMENT OF NON-24-HOUR SLEEP-WAKE DISORDER (N24HSWD) IN BLIND INDIVIDUALS WITH NO LIGHT PERCEPTION WHO HAVE ENROLLED IN OTHER TASIMELTEON CLINICAL TRIALS

A.3.1

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

Weiterführende, offene Verträglichkeitsstudie einer 24-monatigen Gabe von 20mg Tasimelteon zur Behandlung von Schlaf-Wach-Störungen mit Abweichung vom 24-Stunden-Rhythmus (N24HSWD) bei blinden Patienten ohne Lichtwahrnehmung, die an einer vorangegangenen klinischen Studie mit Tasimelteon teilgenommen haben

A.4.1

Sponsor's protocol code number

VP-VEC-162-3204

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01429116

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	Vanda Pharmaceuticals 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	Vanda Pharmaceuitcals 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	Vanda Pharmaceuticals Inc.
B.5.2	Functional name of contact point	Marlene Dressmann
B.5.3	Address:
B.5.3.1	Street Address	2200 Pennsylvania Ave NW Suite 300E
B.5.3.2	Town/ city	Washington
B.5.3.3	Post code	DC 20037
B.5.3.4	Country	United States
B.5.4	Telephone number	0012027343400
B.5.5	Fax number	0012022961450
B.5.6	E-mail	marlene.dressman@vandapharma.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/841
D.3 Description of the IMP
D.3.1	Product name	Tasimelteon
D.3.2	Product code	VEC-162
D.3.4	Pharmaceutical form	Capsule, hard
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	Tasimelteon
D.3.9.1	CAS number	609799-22-06
D.3.9.2	Current sponsor code	VEC-162
D.3.9.3	Other descriptive name	(1R, 2R)-N-[2-(2,3-dihydrobenzofuran-4-yl)-cyclopropylmethyl]propanamide
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	20
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Capsule, hard
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

Blind males or females with no conscious light perception and the
complaint of a sleep-wake disorder associated with Non-24 Hour Sleep-
Wake Disorder

Blinde Männer oder Frauen ohne Licht-Wahrnehmung und mit Störung des Schlaf-Wach-Rhythmus mit einer Abweichung vom 24-Stunden-Rhythmus des Tages (N24HSWD)

E.1.1.1

Medical condition in easily understood language

Blind males or females with no conscious light perception and the
complaint of a sleep-wake disorder associated with Non-24 Hour Sleep-
Wake Disorder

Blinde Männer oder Frauen ohne Licht-Wahrnehmung und mit Störung des Schlaf-Wach-Rhythmus mit einer Abweichung vom 24-Stunden-Rhythmus des Tages (N24HSWD)

E.1.1.2

Therapeutic area

Body processes [G] - Biological Phenomena [G16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	16.1
E.1.2	Level	PT
E.1.2	Classification code	10009191
E.1.2	Term	Circadian rhythm sleep disorder
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

The primary objective of this study is to characterize the effect of tasimelteon, 20 mg/night for 24 months, on standard measures of subject safety.

E.2.2

Secondary objectives of the trial

not appliacble

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Ability and acceptance to provide informed consent;
2. Men or women at least 18 years of age or older who meet one of the following:
a. Has enrolled in VP-VEC-162-3201 (with sponsor approval)
b. Has completed VP-VEC-162-3203 (for US participants only)
c. Did not have a qualifying tau for randomization in VP-VEC-162-3203 (for US participants only)
d. Has enrolled in VP-VEC-162-3203 (with sponsor approval - for US participants only)
3. Males, non-fecund females (i.e., surgically sterilized, if procedure was done 6 months before screening or subject is postmenopausal, without menses for 1 year before screening), or females of child-bearing potential using 2 independent barrier methods of birth control (i.e., condoms, diaphragm, spermicidal agents, cervical cap) for a period of 35 days before the first dosing, during the study and for one month after the last dose and must have a negative pregnancy test at the screening and baseline visits; Note: Hormonal contraception is not considered a reliable method of birth control in this study.
Note: For patients over 55 years of age at the screening visit absence of menses for 1 year before screening is sufficient to establish the postmenopausal status. The postmenopausal status of a patient under 55 years of age at the screening visit will be confirmed by measuring the following hormones:
• Follicle-stimulating hormone (FSH) ≥40 mIU/mL
• Estradiol ≤ 30 pg/mL (110.1 pmol/L)
4. Diagnosis of N24HSWD in a previous tasimelteon study;
5. Willing and able to comply with study requirements and restrictions

E.4

Principal exclusion criteria

1. History (within the 12 months prior to screening) of psychiatric disorders including Major Depressive Disorder, Generalized Anxiety Disorder, Axis II Disorders, delirium or any other psychiatric disorder that in the opinion of the clinical investigator would affect participation in the study or full compliance with study procedures;
2. History of intolerance and/or hypersensitivity to melatonin or melatonin agonists;
3. History of drug or alcohol abuse as defined in DSM-IV, Diagnostic Criteria for Drug and Alcohol Abuse, within the 12 months prior to screening and/or regular consumption of alcoholic drinks (> 40g/day);
4. Patients having any current suicidal ideation of type 4 or 5 on the C-SSRS at Screening or Baseline;
5. Patient is at risk of suicide, in the opinion of the Investigator. Evidence of suicide risk could include any suicide attempt within the past year or any other suicidal behavior within the past year;
6. Current clinically significant cardiovascular, respiratory, neurologic, hepatic, hematopoietic, renal, gastrointestinal or metabolic dysfunction unless currently controlled and stable;
7. Clinically significant deviation from normal in vital signs measurements, or physical examination findings at screening or baseline as determined by the clinical investigator;
8. Pregnant or lactating females;
9. Smoke more than 10 cigarettes/day;
10. Exposure to any investigational drug other than tasimelteon, including placebo, within 30 days, 5 half-lives, or the exclusion period given by a previous study in which the patient has participated in, whichever of the three scenarios is longer.
11. Unwilling or unable to discontinue usage of medication listed in Section 8.2.1;
12. Any other sound medical reason as determined by the clinical investigator.

E.5 End points

E.5.1

Primary end point(s)

Adverse events (AEs) including suicidal ideation or behavior, changes in vital signs, clinical laboratory evaluations, electrocardiograms (ECGs) and physical exam findings during treatment.

E.5.1.1

Timepoint(s) of evaluation of this end point

End of study

E.5.2

Secondary end point(s)

not applicable

E.5.2.1

Timepoint(s) of evaluation of this end point

not applicable

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

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)

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

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

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

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

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

United States

E.8.7

Trial has a data monitoring committee

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

LVLS

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:

133

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

Yes

F.3.3.7.1

Details of other specific vulnerable populations

blind patients

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

200

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)

No treatment different from the expected normal treatment planned

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

2012-12-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-12-03

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2015-01-30

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