Clinical Trials Register

Summary
EudraCT Number:	2021-002331-34
Sponsor's Protocol Code Number:	ALOFEC-2019
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-03-10
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

Spain - AEMPS

A.2

EudraCT number

2021-002331-34

A.3

Full title of the trial

A multicenter, randomized, double-blind Phase IIb study to evaluate the safety and efficacy of the intralesional administration of two doses of expanded allogeneic adipose tissue adult stem mesenchymal cells (ADTCT), packed in hyaluronic acid hydrogel, as a treatment for patients with fecal incontinence.

Estudio Fase IIb multicéntrico, aleatorizado, doble ciego, para evaluar seguridad y eficacia de la administración intralesional de dos dosis de células mesenquimales troncales adultas alogénicas de tejido adiposo expandidas (CMTAd), acondicionadas en hidrogel de ácido hialurónico, como tratamiento para pacientes con incontinencia fecal.

A.3.1

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

Study to assess the safety and efficacy of the administration of multipotent mesenchymal stem cells as a treatment for patients with fecal incontinence.

Estudio para valorar la seguridad y eficacia de la administración de células madre mesenquimales multipotentes como tratamiento para pacientes con incontinencia fecal.

A.3.2

Name or abbreviated title of the trial where available

ALOFEC-2019

A.4.1

Sponsor's protocol code number

ALOFEC-2019

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	FIMABIS
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 Salud Carlos III
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	FIMABIS
B.5.2	Functional name of contact point	Gloria Luque Fernandez
B.5.3	Address:
B.5.3.1	Street Address	C/ Severo Ochoa 35
B.5.3.2	Town/ city	Málaga
B.5.3.3	Post code	29590
B.5.3.4	Country	Spain
B.5.4	Telephone number	34951291977
B.5.5	Fax number	34951440263
B.5.6	E-mail	gloria.luque@ibima.eu

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	Allogeneic stem mesenchymal cells of adipose tissue
D.3.2	Product code	CMTAd
D.3.4	Pharmaceutical form	Suspension 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	NA
D.3.9.1	CAS number	NA
D.3.9.2	Current sponsor code	CMTAd
D.3.9.3	Other descriptive name	EXPANDED HUMAN ALLOGENEIC MESENCHYMAL ADULT STEM CELLS EXTRACTED FROM ADIPOSE TISSUE
D.3.9.4	EV Substance Code	SUB30305
D.3.10	Strength
D.3.10.1	Concentration unit	DF dosage form
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	60000000 to 120000000
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	Yes
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	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	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	Cell suspension excipient. Hyaluronic acid
D.3.2	Product code	Comparator 2
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	NA
D.3.9.1	CAS number	9004-61-9
D.3.9.2	Current sponsor code	Hyaluronic acid
D.3.9.3	Other descriptive name	HYALURONIC ACID
D.3.9.4	EV Substance Code	SUB14126MIG
D.3.10	Strength
D.3.10.1	Concentration unit	DF dosage form
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	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: 3
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	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	Placebo. DMEM solution
D.3.2	Product code	Comparator 1
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	NA
D.3.9.1	CAS number	NA
D.3.9.2	Current sponsor code	DMEM
D.3.9.3	Other descriptive name	DULBECCO'S MODIFIED EAGLE MEDIUM
D.3.9.4	EV Substance Code	SUB21266
D.3.10	Strength
D.3.10.1	Concentration unit	DF dosage form
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	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	Solution for injection
D.8.4	Route of administration of the placebo	Intralesional use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Fecal incontinence

Incontinencia fecal

E.1.1.1

Medical condition in easily understood language

Fecal incontinence

Incontinencia fecal

E.1.1.2

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10016296
E.1.2	Term	Fecal incontinence
E.1.2	System Organ Class	100000004856

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

E.2.2

Secondary objectives of the trial

• Confirm the safety of the use of the two doses of allogeneic ADSCs (60 and 120 million ADSCs) at 18 and 24 months after administration
• Assess the efficacy of intralesional administration of the two different doses (60 million and 120 million) of allogeneic dSCTAs as therapy for FI, compared to placebo and hyaluronic acid hydrogel at 18 and 24 months after administration
• Establish the most efficient dose of ADSCs in the treatment of structural fecal incontinence.
• Assess the feasibility of the administered therapy. Determine if the administration of the cell/comparator product was carried out correctly, without incidents or complications.

• Confirmar la seguridad del uso de las dos dosis de CMTAd alogénicas (60 y 120 millones de CMTAd) a los 18 y 24 meses tras la administración
• Evaluar la eficacia de la administración intralesional de las dos dosis diferentes (60 millones y 120 millones) de CMTAd alogénicas como terapia para la IF, en comparación con placebo y con el hidrogel de ácido hialurónico a los 18 y 24 meses tras la administración
• Establecer la dosis más eficiente de CMTAd en el tratamiento de la Incontinencia Fecal estructural.
• Valorar la factibilidad de la terapia administrada. Determinar si la administración del producto celular/comparador, se realizó correctamente, sin incidencias o complicaciones.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Age 18-80 years (both inclusive), male or female.
2. A single internal and/or external sphincter defect, of no more than 140 degrees, at any level of the anal canal.
3. Fecal incontinence severity of 12 or more on the Wexner Test and/or at least 6 episodes of fecal incontinence over at least a 21-day period, recorded in the Patient Diary.
4. Duration of fecal incontinence for at least 1 year prior to inclusion.
5. Informed consent obtained in writing
6. Commitment to use an effective contraceptive method, both in men and women, during the entire follow-up period of the clinical trial**.

**Effective contraception for women is described as an oral contraceptive method (combining estrogens with gestagens) or another type of contraception, such as an injectable, implantable or patch hormonal contraceptive, IUD or surgical sterilization. The combined use of a condom plus a spermicidal cream is described as an effective contraceptive method for men.

1. Edad de 18-80 años (ambos inclusive), hombre o mujer.
2. Un único defecto esfínter interno y/o externo, de no más de 140 grados, a cualquier nivel del canal anal.
3. Gravedad de incontinencia fecal de 12 ó más en el Test de Wexner y/o como mínimo 6 episodios de incontinencia fecal durante al menos un período de 21 días, registrados en el Diario del Paciente.
4. Duración de la incontinencia fecal de cómo mínimo 1 año anterior a la inclusión.
5. Consentimiento informado obtenido por escrito
6. Compromiso de utilizar un método anticonceptivo efectivo, tanto en hombres como en mujeres, durante todo el periodo de seguimiento del ensayo clínico**.

**Se describe como método anticonceptivo efectivo para las mujeres, aquel método anticonceptivo oral (que combine estrógenos con gestágenos) o de otro tipo como, por ejemplo, un anticonceptivo hormonal inyectable, implantable o en parche, DIU o esterilización quirúrgica. Se describe como método anticonceptivo efectivo para los hombres, el uso combinado de preservativo más una crema espermicida.

E.4

Principal exclusion criteria

1. Have been previously treated with:
• Perianal bulking agents: dextranomer in stabilized hyaluronic acid (NASHA Dx), silicone material (PTQ™), autologous fat, Teflon, bovine glutaraldehyde cross-linked collagen, carbon-coated zirconium beads,

polydimethylsiloxane elastomer, polyacrylamide cross-linked hydrogel, porcine dermal collagen, synthetic calcium hydroxyapatite ceramic microspheres or polyacrylonitrile cylinders.
• Sphincteroplasty (whenever overlapping of the plasty is evidenced by anal ultrasound or magnetic resonance imaging).
• Artificial anal sphincter not explanted.
• Passive or dynamic graciloplasty.
• Gluteoplasty.
• Cerclage of any kind.
• Active sacral neuromodulation.
2. Anorectal tumors at the time of inclusion.
3. Patients with active perianal diseases at the time of inclusion: fissure, fistula, venereal disease and abscess.
4. Anorectal stenosis, which prevents the performance of ultrasound.
5. Significant chronic anorectal or pelvic pain (moderate to severe pain according to VAS scale)
6. Pregnant women or in the 6 months postpartum.
7. Medical history of Human Immunodeficiency Virus (HIV) infection or any severe immunocompromised state or administration of immunosuppressive therapy.
8. Malignancies in remission for less than one year prior to study. An exception to the requirement is basal cell carcinoma (BCC). BCC in “remission” for less than one year is not an exclusion criterion, provided the BCC has been treated and there is no evidence of active disease.
9. Patients with a pathology or clinical condition that prevents the administration of anesthesia and the performance of the intervention in the opinion of the anesthesiologist (eg hemorrhagic diathesis, thrombocytopenia or anticoagulant therapy).
10. Chemotherapy during the 6 months prior to the time of inclusion.
11. Prior radiation with evidence of radiation injury to the area to be treated.
12. Participation in any other clinical study during the 3 months prior to the pre-selection visit, as well as during the execution of the trial.
13. Patients with other serious conditions, anticipated to be unreliable, or otherwise not suitable for participation in the investigator's judgment (eg, severe psychiatric illness or other illness that precludes or does not ensure adequate patient follow-up).
14. Patients with inflammatory or autoimmune disease, uncontrolled or controlled with immunosuppressants or immunomodulators.

15. Patients with active infection by virus B, virus C or syphilis.
16. Patients who require a surgical procedure related to the sphincter apparatus at the time of inclusion.
17. Patients with uncontrolled diarrhea or secondary to a process diagnosed as collagenous colitis, inflammatory disease, etc., at the time of inclusion.
Patients with chronic constipation who require taking laxatives at the time of inclusion.

1. Haber sido objeto de tratamiento previo con:
• Agentes aumentadores de volumen perianal: dextranómero en ácido hialurónico estabilizado (NASHA Dx), material de silicona (PTQ™), grasa autóloga, teflón, colágeno reticulado con glutaraldehído bovino, perlas de circonio recubiertas de carbono,

elastómero de polidimetilsiloxano, hidrogel reticulado con poliacrilamida, colágeno dérmico porcino, microesferas cerámicas sintéticas de hidroxiapatita de calcio o cilindros de poliacrilonitrilo.
• Esfinteroplastia (siempre que se evidencia solapamiento de la plastia por ecografía anal o resonancia magnética).
• Esfínter anal artificial no explantado.
• Graciloplastia pasiva o dinámica.
• Gluteoplastia.
• Cerclaje de cualquier tipo.
• Neuromodulación sacra activa.
2. Tumores anorrectales en el momento de la inclusión.
3. Pacientes con enfermedades perianales activas al momento de la inclusión: fisura, fístula, enfermedad venérea y absceso.
4. Estenosis anorrectal, que impida la realización de ecografía.
5. Dolor anorrectal o pélvico crónico significativo (dolor moderado a severo según escala EVA)
6. Mujeres embarazadas o en los 6 meses postparto.
7. Antecedentes médicos de infección por el Virus de la Inmunodeficiencia Humana (VIH) o cualquier estado inmunocomprometido grave o administración de terapia inmunosupresora.
8. Malignidades en remisión durante menos de un año antes del estudio. Una excepción al requisito es el carcinoma basocelular (CBC). El CBC en “remisión” durante menos de un año no es un criterio de exclusión, a condición de que se haya tratado el CBC y no haya evidencia de enfermedad activa.
9. Pacientes con una patología o condición clínica que impida la administración de anestesia y la realización de la intervención a juicio de anestesiólogo (p.ej. Diátesis hemorrágica, trombocitopenia o terapia anticoagulante).
10. Quimioterapia durante los 6 meses anteriores al momento de la inclusión.
11. Radiación previa con evidencia de lesión por radiación en el área a tratar.
12. Participación en cualquier otro estudio clínico durante los 3 meses anteriores a la visita pre- selección, así como durante la ejecución del ensayo.
13. Pacientes con otros trastornos graves, anticipados como poco fiables o los que de otro modo no son adecuados para la participación a juicio del investigador (por ejemplo, enfermedad psiquiátrica grave u otra enfermedad que imposibilite o no asegure un adecuado seguimiento del paciente).
14. Enfermos con enfermedad inflamatoria o autoinmune, no controlada o controlada con inmunosupresores o inmunomoduladores.

15. Pacientes con infección activa por virus B, virus C o sífilis.
16. Pacientes que requieran un procedimiento quirúrgico relacionado con el aparato esfinteriano al momento de la inclusión.
17. Pacientes con diarrea no controlada o secundaria a un proceso diagnosticado como colitis colágena, enfermedad inflamatoria, etc, en el momento de la inclusión.
Pacientes con estreñimiento crónico que precise toma de laxantes al momento de la inclusión.

E.5 End points

E.5.1

Primary end point(s)

. To confirm the safety of the use of allogeneic adipose-derived mesenchymal stem cells (ADSCs) combined with a hyaluronic acid matrix in the treatment of structural fecal incontinence, through the incidence of adverse events related to the investigational product in the first 12 months post-infusion after intralesional administration.
• Assess the efficacy of intralesional administration of allogeneic dSTSCs as a therapy for FI, comparing two different doses (60 million cells and 120 million dSTSCs resuspended in hyaluronic acid matrix) compared to placebo (Solution A: DMEM supplemented with 2 % of human serum albumin and 2% of L-glutamine) and with the hyaluronic acid hydrogel without diluted ADSCs (1:1, v/v) in Solution A. For this, the changes at 3, 6 and 12 months after the administration of the investigational drug with respect to the baseline determination (prior to the administration of the MEI) of the following variables:
Number of episodes of fecal incontinence of loose or solid stools (according to data from the patient's stool diary),
· Jorge-Wexner scale score
· Fecal incontinence quality of life scale score (FIQL).
Anal manometry: maximum resting pressure, maximum voluntary contraction pressure
Imaging variables: endorectal ultrasound and flexible sigmoidoscopy/rigid rectoscopy

• Confirmar la seguridad del uso de células troncales mesenquimales derivadas de tejido adiposo (CMTAd) alogénicas combinadas con una matriz de ácido hialurónico en el tratamiento de la incontinencia fecal estructural, mediante la incidencia de eventos adversos relacionados con el producto en investigación en los primeros 12 meses postinfusión tras la administración intralesional.
• Evaluar la eficacia de la administración intralesional de CMTAd alogénicas como terapia para la IF, comparando dos dosis diferentes (60 millones de células y 120 millones de CMTAd resuspendidas en matriz de ácido hialurónico) en comparación con placebo (Solución A: DMEM suplementado con 2% de albùmina sérica humana y 2% de L-glutamina) y con el hidrogel de ácido hialurónico sin CMTAd diluido (1:1, v/v) en Solución A. Para ello, se evaluarán los cambios a los 3, 6 y 12 meses tras la administración del medicamento en investigación respecto a la determinación basal (previa a la administración del MEI) de las siguientes variables:
· Número de episodios de incontinencia fecal de heces sueltas o sólidas (según los datos del diario de heces del paciente),
· Puntuación en escala de Jorge-Wexner
· Puntuación en escala de calidad de vida de incontinencia fecal (FIQL).
· Manometría anal: presión máxima en reposo, presión de contracción máxima voluntaria
· Variables de imagen: Ecografía endorrectal y Sigmoidoscopia flexible/rectoscopia rígida

E.5.1.1

Timepoint(s) of evaluation of this end point

Preselection visit.
Selection visit ( +25 days)
Treatment visit (+13 days)
Weeks: 4, 12, 24, 48, 72 y 96

Visita de preselección
Visita selección ( +25 días)
Visita tratamiento (+13 días)
Semanas: 4, 12, 24, 48, 72 y 96

E.5.2

Secondary end point(s)

E.5.2.1

Timepoint(s) of evaluation of this end point

Preselection visit.
Selection visit ( +25 days)
Treatment visit (+13 days)
Weeks: 4, 12, 24, 48, 72 y 96

Visita de preselección
Visita selección ( +25 días)
Visita tratamiento (+13 días)
Semanas: 4, 12, 24, 48, 72 y 96

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

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

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

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

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

LVLP

Última vista del último paciente

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

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

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)

Best treatment available

Mejor tratamiento disponible

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

2022-11-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-04-28

P. End of Trial
P.	End of Trial Status	Ongoing

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Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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