Clinical Trials Register

Summary
EudraCT Number:	2016-003952-63
Sponsor's Protocol Code Number:	LIQUENIA
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-05-08
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

Spain - AEMPS

A.2

EudraCT number

2016-003952-63

A.3

Full title of the trial

Pilot study of vulval lichen sclerosus treatment by adipose tissue associated with autologous platelet-rich plasma

Estudio piloto del tratamiento del liquen escleroso vulvar mediante tejido adiposo asociado a plasma rico en plaquetas autólogo

A.3.1

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

Study of vulval lichen sclerosus treatment by adipose tissue associated with autologous platelet-rich plasma

Estudio del tratamiento del liquen escleroso vulvar mediante tejido adiposo asociado a plasma rico en plaquetas autólogo

A.4.1

Sponsor's protocol code number

LIQUENIA

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	Instituto de Investigación Sanitaria La Fe
B.1.3.4	Country	Spain
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Instituto de Investigación Sanitaria La Fe
B.4.2	Country	Spain
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Instituto de Investigación Sanitaria La Fe
B.5.2	Functional name of contact point	Jose Maria Millan Salvador
B.5.3	Address:
B.5.3.1	Street Address	Avenida Fernando Abril Martorell 106
B.5.3.2	Town/ city	Valencia
B.5.3.3	Post code	46026
B.5.3.4	Country	Spain
B.5.6	E-mail	investigacion_clinica@iislafe.es

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 fat graft associatte with autologous platelet rich plasma (PRP)
D.3.4	Pharmaceutical form	Injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intradermal use Subdermal use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Platelet-rich plasma (PRP)
D.3.9.3	Other descriptive name	PLATELETS, HUMAN BLOOD
D.3.9.4	EV Substance Code	SUB127875
D.3.10	Strength
D.3.10.1	Concentration unit	ml millilitre(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	EASCS
D.3.9.3	Other descriptive name	EXPANDED HUMAN AUTOLOGOUS MESENCHYMAL ADULT STEM CELLS EXTRACTED FROM ADIPOSE TISSUE (EASCS)
D.3.9.4	EV Substance Code	SUB30158
D.3.10	Strength
D.3.10.1	Concentration unit	ml millilitre(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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)	Yes
	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	Yes
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	Comparator
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	Clobetasol Propionate 0,05%
D.2.1.2	Country which granted the Marketing Authorisation	Spain
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	Cream
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Topical use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	CLOBETASOL PROPIONATE
D.3.9.1	CAS number	25122-46-7
D.3.9.4	EV Substance Code	SUB01346MIG
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.05
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

Vulval lichen sclerosus

Liquen escleroso vulvar

E.1.1.1

Medical condition in easily understood language

Vulval lichen sclerosus

Liquen escleroso vulvar

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.1
E.1.2	Level	LLT
E.1.2	Classification code	10047761
E.1.2	Term	Vulval lichen sclerosus et atrophicus
E.1.2	System Organ Class	100000004858

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Demonstrate the improvement in the elasticity of the fibrosis plaques of patients affected by vulval lichen sclerosus after two infiltrations of fat tissue and autologous platelet-rich plasma.

Demostrar la mejoría en la elasticidad de las placas de fibrosis de las pacientes afectadas de liquen escleroso vulvar tras dos infiltraciones de tejido graso y plasma rico en plaquetas autólogo.

E.2.2

Secondary objectives of the trial

Study of vulvar subunits structural improvement; Evaluate structural improvement in the vulvular areas treated; Analysis of the fibrosis and inflammation improvement at 6 months of the first infiltration and 3 months of the second infiltration;To evaluate if there is symptoms improvement at the month, 3 months, 6 months and 12 months of the first infiltration and at 3 and 9 months of the second infiltration;Examine if there is an improvement in quality of life;Determine whether the beneficial effects at the level of symptom reduction following infiltration of the patient's own fat tissue associated with platelet-rich plasma are maintained in the long term (for at least one year from the first infiltration);Later use of the clinical and pain scale of this study in studies of vulvar lichen sclerosus with greater number of patients;Evaluate treatment-derived adverse events during the first year after first infiltration by CRD collection.

Estudio de la mejoría estructural de las subunidades vulvares afectadas;Evaluar si se produce una mejoría estructural en las zonas vulvares tratadas;Análisis de la mejoría de la fibrosis y de la inflamación;Estudiar si se produce una mejoría de los síntomas al mes, 3 meses, 6 meses y 12 meses de la primera infiltración y a los 3 y 9 meses de la segunda infiltración;Examinar si se produce una mejoría en la calidad de vida;Determinar si los efectos beneficiosos a nivel de reducción de síntomas tras la infiltración del tejido graso de la propia paciente asociado a plasma rico en plaquetas se mantienen a largo plazo (durante al menos un año desde la primera infiltración);Utilización posterior de la escala de afectación clínica y de dolor de este estudio en estudios de liquen escleroso vulvar con mayor número de pacientes;Evaluar los eventos adversos derivados del tratamiento durante el primer año tras la primera infiltración mediante recogida en el CRD.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Adult women between 18 and 70 years old
Patients with clear clinical and / or histological diagnosis of lichen sclerosus
Vulvar biopsy existence at some point in patient’s medical history to exclude malignant or pre-malignant disease
Moderate or severe disease at genital level
Patients who have had topical treatment for at least three months with 0.05% clobetasol propionate.
Informed consent previously signed.

Mujeres adultas entre 18 y 70 años de edad.
Pacientes con diagnóstico claro clínico y/o histológico de liquen escleroso (LE).
Existencia de biopsia vulvar en algún momento de su historia clínica a fin de excluir enfermedad maligna o pre-maligna.
Afectación moderada o severa de la enfermedad a nivel genital.
Las pacientes que hayan llevado tratamiento tópico durante al menos tres meses con propionato de clobetasol al 0,05%.
Consentimiento informado firmado previo.

E.4

Principal exclusion criteria

Pregnant or lactating women
Alcoholic patients
Patients with malignant disease diagnosed in the last 5 years
Patients infected with HIV, HBV and HCV virus
Injecting drug users
Patients with serious active infectious diseases.
Patients with allergy or intolerance recognized for any of the above treatments
Patients with inflammatory diseases that may affect the vulvar area (Crohn's disease, ulcerative colitis, Psoriasis, Eczema).
Patients with unrealistic expectations regarding the ultimate benefits of treatment

Mujeres embarazadas o en periodo de lactancia.
Pacientes alcohólicas.
Pacientes con enfermedad maligna diagnosticada en los últimos 5 años.
Pacientes infectados por los virus VIH, VHB y VHC.
Usuarios de drogas por vía parenteral.
Pacientes con enfermedades infecciosas activas graves.
Pacientes con alergia o intolerancia reconocida a cualquiera de los tratamientos mencionados.
Pacientes con enfermedades inflamatorias que puedan afectar a la zona vulvar (enfermedad de Crohn, Colitis ulcerosa, Psoriasis, Eczema).
Pacientes con expectativas poco realistas respecto a los beneficios finales del tratamiento.

E.5 End points

E.5.1

Primary end point(s)

Elasticity of fibrosis plaques in the affected vulvar area

Elasticidad de las placas de fibrosis de la zona vulvar afectada

E.5.1.1

Timepoint(s) of evaluation of this end point

1 month, 3 months, 6 months and 12 months after treatment.

Al mes, 3 meses, 6 y 12 meses de la primera infiltración, y a los 3 y 9 meses de la segunda infiltración.

E.5.2

Secondary end point(s)

Structural improvement of vulvar subunits, histological improvement, improvement in quality of life, improvement of clinical and symptoms, and improvement in scores on clinical scale, pain scale, pruritus scale, and IFS ( Index of sexual function) of Rosen.

Mejoría estructural de las subunidades vulvares, mejoría histológica, mejoría en la calidad de vida, mejoría de la clínica y los síntomas y mejora en las puntuaciones de la escala de afectación clínica, de la escala de dolor, escala de prurito, y del IFS (índice de función sexual) de Rosen.

E.5.2.1

Timepoint(s) of evaluation of this end point

1 month, 3 months, 6 months and 12 months after treatment.

Al mes, 3 meses, 6 y 12 meses de la primera infiltración, y a los 3 y 9 meses de la segunda infiltración.

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)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

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

E.8.1.5

Parallel group

Yes

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)

Yes

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

Yes

E.8.4

The trial involves multiple sites in the 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

E.8.6.2

Trial being conducted completely outside of the EEA

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

Last visit of the last subject undergoing the trial

Última visita del último paciente participando en el ensayo

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

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:

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

Yes

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.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)

None

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

2017-07-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-06-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2019-12-12

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