Ensayo clínico de fase III, doble ciego, aleatorizado, controlado con placebo, interventivo y multicéntrico para investigar la seguridad y eficacia de las células estromales mesenquimales positivas para ABCB5 (MSC ABCB5+) en la epidermólisis bullosa (EB)

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT05464381Tipo: INTERVENTIONALInicio: 13 de jun de 2023Fin estimado: 1 de ene de 2027

Resumen

El objetivo de este ensayo clínico es investigar la seguridad y eficacia de allo-APZ2-OTS administrado por vía intravenosa a sujetos con epidermólisis bullosa distrófica recesiva (EBDR) en comparación con placebo. Se incluirá un brazo adicional abierto controlado con línea de base para investigar la seguridad y eficacia de allo-APZ2-OTS administrado por vía intravenosa a sujetos con EBJ y a sujetos con EBDR menores de 1 año.

Elegibilidad

Sexo: ALL

Criterios de inclusión

Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM). A minimal body weight of at least 5 kg is required;
Subject is eligible to participate in this clinical trial based on general health condition;
Subject with a target wound meeting the following criteria: 5-50 cm2, and \< 9 months, no signs of acute infection;
Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
Women of childbearing potential must have a negative urine pregnancy test at Visit 1. Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial.

Criterios de exclusión

Signs and/or a history of skin precancerous and cancerous lesions at screening or any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
Known chronic lung disease;
Clinically significant laboratory values for coagulation and thrombocytes at screening;
Thromboembolic events of any grade in medical history;
Immunoreactions of any grade in medical history;
Any known allergies to components of the IP or premedication;
Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
Pregnant or lactating women;
Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
Previous participation in this clinical trial (except for screening failures);
Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits;
Employees of the sponsor, or employees or relatives of the investigator.

Intervenciones

drug

allo-APZ2-OTS

Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)

drug

Placebo

Ubicaciones

Sanatorio Mater Dei

Buenos Aires, Argentina

Patrocinadores

PrincipalRHEACELL GmbH & Co. KG

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

Ensayo clínico de fase III, doble ciego, aleatorizado, co... | EligiMed