Clinical Trials Register

Summary
EudraCT Number:	2017-000956-25
Sponsor's Protocol Code Number:	15-06
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	GB - no longer in EU/EEA
Date on which this record was first entered in the EudraCT database:	2018-05-25
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

UK - MHRA

A.2

EudraCT number

2017-000956-25

A.3

Full title of the trial

CELLEBRATE: An Adaptive, Two-Stage, Double-Blind, Stratified, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR with Placebo in Female Subjects with Stress Urinary Incontinence

A.3.1

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

Clinical Study Investigating Autologous Muscle Derived Cells for Treatment of Female Stress Urinary Incontinence Patients

A.3.2

Name or abbreviated title of the trial where available

CLBT

A.4.1

Sponsor's protocol code number

15-06

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03104517

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	Cook MyoSite, Incorporated
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	Cook MyoSite, Incorporated
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	IQVIA
B.5.2	Functional name of contact point	Project Manager, ID & Vaccines
B.5.3	Address:
B.5.3.1	Street Address	4820 Emperor Boulevard
B.5.3.2	Town/ city	Durham, NC
B.5.3.3	Post code	27703
B.5.3.4	Country	United States
B.5.6	E-mail	Christina.Benjes@iqvia.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	Autologous Muscle Derived Cells
D.3.2	Product code	AMDC-USR
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	AMDC
D.3.9.2	Current sponsor code	AMDC
D.3.9.3	Other descriptive name	AMDC
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
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)	Yes
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	Yes
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	Yes
D.3.11.3.5.1	CAT classification and reference number	Tissue Engineered Product, not combined; Committee on Advance Therapies, 7 October 2012, EMA/627170/2010
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	Suspension for injection
D.8.4	Route of administration of the placebo	Intramuscular use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Female Stress Urinary Incontinence

E.1.1.1

Medical condition in easily understood language

Accidental loss of urine due to physical activity such as coughing, sneezing, exercise or laughing.

E.1.1.2

Therapeutic area

Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13]

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	10066218
E.1.2	Term	Stress urinary incontinence
E.1.2	System Organ Class	10038359 - Renal and urinary 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 efficacy of a single dose of 150 x 10(6) AMDC-USR in the reduction of stress incontinence episode frequency (EIF) in adult female subjects at 12 months post-treatment.

E.2.2

Secondary objectives of the trial

To determine the safety of a single dose of 150 x 10(6) AMDC-USR at 12 months and 2 years post-treatment.
and
To evaluate improvement in quality of life (QOL) and the extent of reduction of stress IEF after a single dose of 150 x 10(6) AMDC-USR at 12 months and 2 years post-treatment.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Adult female 50 to 75 years of age who has primary and moderate-to-severe symptoms of SUI (9-45 stress incontinence episodes over 3 days, as recorded in the 3-Day Diary at screening) for a duration of at least 6 months, as confirmed by subject medical history and clinical symptoms, including a focused incontinence evaluation.
- Must have low hypermobility of the urethra with Valsalva Q-tip ≤ 30º.
- Must be willing and able to comply with the study procedures, be
mentally competent and able to understand all study requirements, and
must agree to read and sign the informed consent form prior to any
study-related procedures.
- Must have completed 100% of their screening 3-Day Diary Evening
Reports.
- Must be willing to use acceptable methods of contraception if of childbearing potential.

E.4

Principal exclusion criteria

- Symptoms of only urge incontinence as confirmed by basic evaluation of etiology from a subject medical history, including a focused incontinence history.
- Symptoms of mixed urinary incontinence where urge incontinence is the predominant factor, defined by the 3-Day Diary as having more than 2x the number of urge leaks than stress leaks (Assessment of predominant urge incontinence is based on the medical record, physician's assessment, and a one-time 3-Day Diary).
- Has not previously attempted conservative treatment prior to signing the informed consent (Examples of conservative treatment include behavior modifications, bladder exercises, biofeedback, PFMT).
- Morbidly obese (BMI ≥ 35).
- Have medically diagnosed autoimmune disease(s) or other types of immune system compromise or disorder that has the potential for an exacerbation of the symptoms that may cause the subject to become susceptible to infections, or may require systemic corticosteroid/immunosuppressive treatment during the study.
- Has known allergy or hypersensitivity to bovine proteins or allergens, gentamicin sulfate, DMSO, or ampicillin that medically warrants exclusion as determined by the physician.
- History of cancer in pelvic organs, ureters, or kidneys.
- Any cancer that has undergone treatment within the past 12 months.
- Actively undergoing treatment with stimulation neuromodulation system (sacral, tibial, or percutaneous tibial nerve stimulation) within the last 6 months or planned during the course of the study.
- Pregnant, lactating, or plans to become pregnant during the course of the study.

E.5 End points

E.5.1

Primary end point(s)

A comparison between the AMDC-USR and placebo groups in the percentage of subjects with ≥ 75% reduction in stress IEF as recorded in the 3-Day Diary at 12 months compared with baseline.

E.5.1.1

Timepoint(s) of evaluation of this end point

12 months

E.5.2

Secondary end point(s)

A comparison between the AMDC-USR and placebo groups for:
- Change in mean I-QOL score as determined by I-QOL score and subscores at 12 months compared with baseline;
- Percentage of subjects with ≥ 50% reduction in stress IEF as recorded in the 3-Day Diary at 12 months compared with baseline;
- Percentage of subjects with continence restored defined as 0 or 1 stress incontinence episode as recorded in the 3-Day Diary at 12 months;
and
- Improvement (reduction) in stress IEF as recorded in the 3-Day Diary at 12 months compared with baseline.

E.5.2.1

Timepoint(s) of evaluation of this end point

12 months

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

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Placebo treated patients will be given option to receive AMDC-USR after their 12 months follow up.

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

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 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

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

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:

230

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

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

144

F.4.2

For a multinational trial

F.4.2.1

In the EEA

192

F.4.2.2

In the whole clinical trial

320

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)

Patients are under physician care prior to enrollment in the study and are expected to remain under the supervision of the responsible physician. No specific sponsor related treatment or care is anticipated after end of participation and no limits are expected on the provision of standard care following participation.

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

2018-08-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-11-09

P. End of Trial
P.	End of Trial Status	GB - no longer in EU/EEA

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