Clinical Trials Register

Summary
EudraCT Number:	2010-024328-53
Sponsor's Protocol Code Number:	HULPOFT-2010-01
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:	2011-12-12
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

2010-024328-53

A.3

Full title of the trial

Phase IIa clinical trial to determine the feasibility and safety of using autologous expanded stem cell (ASC) from fat in the treatment of keratopathy associated with bilateral limbic insufficiency

Ensayo clínico en fase IIa para conocer la factibilidad y seguridad del uso autólogo de células madre expandidas (ASC) derivadas de la grasa en el tratamiento de la queratopatía asociada a la insuficiencia límbica bilateral

A.3.1

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

The proposal is to join a clinical research study in Phase IIa to test the feasibility and safety of a new therapeutic application based on the use of stem cells derived from lipoaspirate (ASC).

Se le propone incorporarse a un estudio de investigación clínica en Fase IIa para comprobar la factibilidad y seguridad de una nueva aplicación terapéutica basada en el uso de células madre derivadas de lipoaspirado (ASC).

A.3.2

Name or abbreviated title of the trial where available

FLPURO-2010-01

A.4.1

Sponsor's protocol code number

HULPOFT-2010-01

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	Fundación para la Investigación Biomédica del Hospital Universitario La Paz
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	Ministerio de Sanidad, Política Social e
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	Fundación para la Investigación Biomédica del Hospital
B.5.2	Functional name of contact point	Unidad de Terapia Celular
B.5.3	Address:
B.5.3.1	Street Address	Paseo de la Castellana, 261
B.5.3.2	Town/ city	Madrid
B.5.3.3	Post code	28016
B.5.3.4	Country	Spain
B.5.4	Telephone number	0034912071022
B.5.5	Fax number	0034912071512
B.5.6	E-mail	mgarciaa.hulp@salud.madrid.org

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	Suspensión de células madre mesenquimales adultas derivadas de tejido adiposo
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	Intralesional use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Suspensión de células madre mesenquimales adultas derivadas de tejido adiposo
D.3.10	Strength
D.3.10.1	Concentration unit	Other
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	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)	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	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

Keratopathy associated with bilateral limbic insufficiency.

Queratopatía asociada a la insuficiencia límbica bilateral.

E.1.1.1

Medical condition in easily understood language

Keratopathy associated with bilateral limbic insufficiency.

Queratopatía asociada a la insuficiencia límbica bilateral.

E.1.1.2

Therapeutic area

Diseases [C] - Eye Diseases [C11]

MedDRA Classification

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 feasibility and safety of autologous ASC to treat keratopathy associated with bilateral limbic insufficiency.

Evaluar la factibilidad y seguridad de las ASC autólogas para tratar queratopatía asociada a insuficiencia límbica bilateral.

E.2.2

Secondary objectives of the trial

To evaluate changes in quality of life of treated patients.
To determine the direct evolution of patients after being treated by applying ASC in the ocular surface

Evaluar los cambios en la calidad de vida de los pacientes tratados.
Determinar la evolución directa de los pacientes tras ser tratados mediante la aplicación ASC en la superficie ocular

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1 .- Over 18 years old in good general health, according to data from the clinical history and physical examination.
2 .- Previous diagnosis of bilateral IL.
3 .- Have chronic keratopathy under the following conditions:
Confimación of IL through impression cytology
Repeated failure of standard treatments for this condition
4 .- Signing of informed consent

1.- Ser mayor de 18 años con buen estado general de salud, de acuerdo con los datos de la historia clínica y la exploración física.
2.- Diagnóstico previo de IL bilateral.
3.- Tener queratopatía crónica y que cumplan las siguientes condiciones:
Confimación de la IL por citología de impresión
Fracaso reiterado de los tratamientos habituales para esta patología
4.- Firma del consentimiento informado

E.4

Principal exclusion criteria

1 .- Do not meet any criteria for inclusion
2 .- History of malignancy within the last 5 years.
3 .- Allergy to local anesthetics
4 .- Patients who have participated in other studies during the 90 days prior to their inclusion.
5 .- Tacrolimus or cyclosporine administration in the 4 weeks prior to cell therapy.
6 .- Medical or psychiatric illness of any kind, according to the investigator, may be a reason for exclusion from the study.
7 .- Subjects with congenital or acquired immunodeficiencies. Syphilis, Hepatitis B and / or C or tuberculosis diagnosed at the time of inclusion.
8 .- Major surgery or major trauma of the subject in the previous semester.
9 .- Pregnant or lactating women.

1.- No cumplir algún criterio de inclusión
2.- Historial de neoplasias en los últimos 5 años.
3.- Alergia a anestésicos locales
4.- Pacientes que hayan participado en otros estudios durante los 90 días previos a su inclusión.
5.- Administración de tacrolimus o ciclosporina en las 4 semanas anteriores a la terapia celular.
6.- Enfermedad médica o psiquiátrica de cualquier tipo que, en opinión del investigador, pueda suponer un motivo de exclusión del estudio.
7.- Sujetos con inmunodeficiencias congénitas o adquiridas. Sífilis, Hepatitis B y/o C o tuberculosis diagnosticada en el momento de la inclusión.
8.- Cirugía mayor o traumatismo grave del sujeto en el semestre anterior.
9.- Mujeres embarazadas o lactantes.

E.5 End points

E.5.1

Primary end point(s)

The evaluation of feasibility and safety of the study will be made once the patient has received treatment at 3-month implant and follow-up year. We define that the process is safe when development and monitoring of the trial has not produced any adverse event that may be related to the proposed therapy in the trial. All clinical adverse events during follow-up will be collected at the same time that the clinical evaluation. The main objective will be assessed the cumulative incidence of adverse effects attributed to therapy in the study.

De factibilidad y seguridad del estudio se realizará una vez el paciente haya recibido el tratamiento, al 3º mes del implante y al año de seguimiento. Se define que el proceso es seguro cuando durante el desarrollo y seguimiento del ensayo no se ha producido ningún acontecimiento adverso que se pueda relacionar con la terapia propuesta en el ensayo. Todos los acontecimientos adversos clínicos serán recogidos durante el seguimiento en los mismos tiempos que la evaluación clínica. Se evaluará como objetivo principal la incidencia acumulada de efectos adversos atribuidos a la terapia en el estudio.

E.5.1.1

Timepoint(s) of evaluation of this end point

at the 3-month implant and follow-up year

al 3º mes del implante y al año de seguimiento

E.5.2

Secondary end point(s)

As part of assessing the status of patients quality of life a SF-36 test will be filled out and the nonexistence of long-term adverse effects will be recorded as measured by the non-recurrence of pain, conjunctivalization, ulcers and corneal epithelial neovascularization, after 1 year from the implant. Both studies will be conducted by clinical investigators of the trial, if there is no agreement in the diagnosis an expert ophthalmologist from outside of the study will be consulted.

Como parte de la evaluación del estado de la calidad de vida de los pacientes se rellenará un test SF-36 y se registrará la no existencia de efectos adversos a largo plazo medida por la no recurrencia de dolor, conjuntivalización, úlceras epiteliales corneales y neovascularización, pasado 1 año desde el implante. Ambos estudios serán realizados por los investigadores clínicos del ensayo, en caso de no existir acuerdo en el diagnóstico se recurrirá a un oftalmólogo experto ajeno al estudio.

E.5.2.1

Timepoint(s) of evaluation of this end point

after 1 year from the implant

pasado 1 año desde el implante

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

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.8.3

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

When a patient is removed from the study for any reason, must be notified and the investigetor will carry out every end of the study procedures. Nevertheless the patient will be asked to come to the consultation at 12 weeks and one year after cell implantation to check for any adverse event has occurred.

Cuando un paciente sea retirado del estudio, por cualquier causa, debe ser notificado y el investigador cuplimentará todos los procedimientos de fin del estudio. No obstante se solicitará al paciente que acuda a la consulta a la semana 12 y al año posterior al implante celular para comprobar si ha ocurrido algún acontecimiento adverso.

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)

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.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)

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

2012-05-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-10-27

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
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