Clinical Trials Register

Summary
EudraCT Number:	2013-004384-31
Sponsor's Protocol Code Number:	SAG/0211PFC-11S1
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2016-02-04
Trial results	View 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

Poland - Office for Medicinal Products

A.2

EudraCT number

2013-004384-31

A.3

Full title of the trial

A Multicentre, Long-term Safety, Efficacy and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged ≥6 years to <18 years with Functional Constipation

A.3.1

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

Long-term safety extension study of lubiprostone for the treatment of paediatric functional constipation in subjects aged ≥6 to <18 years

A.4.1

Sponsor's protocol code number

SAG/0211PFC-11S1

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/190/2012

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Sucampo Pharma Americas, LLC
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	Sucampo Pharma Americas, LLC
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	Sucampo AG
B.5.2	Functional name of contact point	Senior Director Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	Baarerstrasse 22
B.5.3.2	Town/ city	Zug
B.5.3.3	Post code	6300
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	41417263045
B.5.5	Fax number	41417263031
B.5.6	E-mail	hschulze@sucampo.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	Yes
D.2.1.1.1	Trade name	Amitiza 24 microgram soft capsules
D.2.1.1.2	Name of the Marketing Authorisation holder	Sucampo Pharma Europe Ltd.
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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.3.4	Pharmaceutical form	Capsule, soft
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	Lubiprostone
D.3.9.1	CAS number	136790-76-6
D.3.9.2	Current sponsor code	SPI-0211
D.3.9.3	Other descriptive name	LUBIPROSTONE
D.3.9.4	EV Substance Code	SUB32077
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	24
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.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	Lubiprostone capsules 12 mcg
D.3.2	Product code	SPI-0211, RU-0211
D.3.4	Pharmaceutical form	Capsule, soft
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	Lubiprostone
D.3.9.1	CAS number	136790-76-6
D.3.9.2	Current sponsor code	SPI-0211
D.3.9.3	Other descriptive name	LUBIPROSTONE
D.3.9.4	EV Substance Code	SUB32077
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	12
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

functional constipation in paediatric patients

E.1.1.1

Medical condition in easily understood language

constipation in children and adolescents

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.0
E.1.2	Level	PT
E.1.2	Classification code	10010774
E.1.2	Term	Constipation
E.1.2	System Organ Class	10017947 - Gastrointestinal 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 assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent obtained from subject and/or parent/legal guardian (and assent from subject where applicable).
2. Subject must have completed the entire 12-week treatment period from the preceding study (SAG/0211PFC-1131) prior to enrolment.
3. Subject must continue to abstain from taking concomitant medication (prescribed or over-the-counter) that affects gastrointestinal motility; these medications include:
a. Cholinesterase inhibitors; anti-spasmodic, anti-diarrheal, anti-constipation, or prokinetic agents; laxative agents (e.g., PEG 3350), including homeopathic remedies;
b. Tricyclic antidepressants; or
c. Any medication, at the discretion of the Investigator, known to relieve or cause constipation or constipation-related symptoms, and which the Investigator, based on medical history of the subject, suspects to be a contributing factor to the patient's chronic constipation, or may otherwise confound the evaluation of treatment response.
Exceptions: Treatment with anticholinergic agents, SSRIs, SNRIs, or MAO inhibitors is allowed if a stable dose has been used for at least 30 days prior to the Baseline Visit (of the preceding study SAG/0211PFC-1131) and not likely to change during the study.
4. Subject (and if necessary, parent/legal guardian) must be willing and able to use or administer recommended (rectal and/or oral) rescue medications if needed.
5. If subject is taking a fibre supplement (e.g., Metamucil®, PerDiem®, Fybogel), usage must have been at a stable dose not likely to change during the study.
6. Subject and his/her parent/legal guardian must be willing and able to fill out his/her own diary

E.4

Principal exclusion criteria

1. Subject has current evidence of untreated faecal impaction.
2. Subject has experienced an adverse event during the SAG/0211PFC-1131 study which the Investigator considers to be clinically significant and would limit the subject’s ability to participate in the trial.
3. Subject has had a significant change in their medical status, newly diagnosed and uncontrolled cardiovascular, liver or lung disease, neurologic or psychiatric disorder, or other systemic disease, which the Investigator considers to be clinically significant and would limit the subject’s ability to participate in the trial.
4. Subject has developed abnormal laboratory test (haematology, urinalysis, or blood chemistry), which in the Investigator’s opinion is clinically significant, unexplained, and would limit the subject’s ability to participate in the trial.
5. Subject (female of childbearing potential) has a positive pregnancy test or refuses/is unwilling to undergo pregnancy testing, and/ does not agree to use protocol-specified contraceptive measures for the duration of the study.
6. Subject demonstrated non-compliance with study protocol (i.e., dosing schedule, visit schedule, diary completion, or study procedures) during the SAG/0211PFC-1131 study

E.5 End points

E.5.1

Primary end point(s)

The efficacy endpoints are as follows:
• Overall and monthly changes from baseline in BM and SBM frequency rate
• Overall and monthly assessments of the average degree of, and changes from baseline in:
o Stool consistency of SBMs
o Straining associated with SBMs
o Abdominal pain associated with SBMs
o Constipation severity
• Monthly SBM Response
o A monthly responder is defined as a subject who is a weekly responder for 3 of 4 weeks per month.
o A weekly responder is defined as a subject who has a frequency rate of ≥ 3 SBMs/week and an increase from baseline of ≥ 1 SBM/week for that week.
• Overall and monthly assessment of average treatment effectiveness rating
• Overall Health-related quality of life (PedsQL™)
• Overall and monthly change from baseline in incontinence episodes frequency (analysis performed for subset of subjects presenting with incontinence at baseline)
• Overall and monthly change from baseline in the production of large diameter stool (a stool that clogs the toilet) frequency
• Overall and monthly change from baseline in frequency of faecal impaction
• Overall and monthly change from baseline in proportion of BMs and SBMs in toilet overall
• Overall and monthly change from baseline in frequency of retentive posturing or excessive volitional stool retention

The safety endpoints are as follows:
• Incidence of adverse events grouped by MedDRA System Organ Class (SOC) and Preferred Term
• Changes from baseline in clinical laboratory parameters (haematology, serum chemistry, urinalysis)
• Changes from baseline in physical examination
• Changes from baseline in vital sign measurements, including height and weight
o Height will be measured using a wall-mounted stadiometer, if available
• For those subjects who were aged 6 to 9 or 14 to 17 at the time of enrolment into the SAG/0211PFC-1131 study, and who were qualified to participate and enrolled in the dual-energy X-ray absorptiometry (DXA) substudy:
o Percent changes from baseline in bone mineral density (BMD) and bone mineral content (BMC)
o Changes from baseline in BMD Z-scores and in height-adjusted Z-scores, as assessed by DXA for DXA Subgroup
o Changes from baseline in height and weight Z-scores for DXA Subgroup
o Incidence of clinical fractures

E.5.1.1

Timepoint(s) of evaluation of this end point

throughout the entire 36 weeks treatment period (e-diary)

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

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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

Yes

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

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

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

Belgium

Canada

France

Netherlands

Poland

Spain

United Kingdom

United States

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

LPLV

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

415

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

205

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

210

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Children and adolescents below 18 years.

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

150

F.4.2.2

In the whole clinical trial

415

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)

Subjects will return to standard of care

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

2016-04-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-02-11

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2017-05-01

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