Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia Subtype A.

• Register: EUCTR
• Last refreshed on: 13 May 2013
• Main ID: EUCTR2011-006100-12-ES
• Date of registration: 27/01/2012
• Prospective Registration: Yes
• Primary sponsor: FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS
• Public title: Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia Subtype A.
• Scientific title: Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A. - Fancolen-1
• Date of first enrolment: 12/04/2013
• Target sample size: 5
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-006100-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Spain

Contacts

• Name: Servicio Oncohematologia
• Affiliation: Hospital Niño Jesus

• Name: Servicio Oncohematologia
• Affiliation: Hospital Niño Jesus

Key inclusion & exclusion criteria

Inclusion criteria:
- Patients diagnosed with the Fanconi anemia complementation group-A
- At least one of the following parameters must be less than the indicated values?: hemoglobin 8.0 g / dL, neutrophil count: 750/mm3, platelets: 30.000/mm3
- Age> 1 year
- Lansky Index> 60%
- Mild functional impairment of organs
- To provide informed consent in accordance with current legislation
- Number of transduced CD34+ cells: At least 3x100000 purified CD34 + / kg body weight
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
- Patients with HLA-identical family donor.
- Evidence of leukemia or myelodysplastic syndrome, or cytogenetic abnormalities in bone marrow aspirates predictive of those. In this case studies performed two months before the entry of the patient in the clinical trial will be considered as valid
- Evidence that the patient has signs of somatic mosaicism associated to hematologic improvement.
- Any concomitant disease or condition in the investigator's opinion incapacitate the subject for their participation in the study.
- Effect on pre-existing sensory or motor> = grade 2 according to the criteria of the National Cancer Institute (NCI).


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Fanconi anemia (Subtype A)
MedDRA version: 15.1 Level: LLT Classification code 10055206 Term: Fanconi's anemia System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: CD34+ Cells
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: CD34+ CELLS
Other descriptive name: CD34+ CELLS
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: not less then
Concentration number: 100.000-

Primary Outcome(s)

Primary end point(s): - Determine the toxicity associated with infusion of CD34 + cells transduced with lentiviral vector therapy in FA-A patients.
- Determining the degree of graft associated to infusion o fautologous CD34 + cells transduced with lentiviral vector therapy in FA-A patients.

Main Objective: The main objective of this Phase I/II clinical trial is to evaluate the safety and the therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene in patients with Fanconi Anemia Subtype A.

Timepoint(s) of evaluation of this end point: Up to 2 years after infusion

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): - To determine the clinical response associated to infusion pof autologous CD34 + cells transduced with therapeutic lentiviral vector in FA-A patients

Timepoint(s) of evaluation of this end point: Up to 2 years after infusion

Secondary ID(s)

FANCOLEN-1

Source(s) of Monetary Support

Fundacion para la investigacion Biomédica del Hospital Universitario Niño Jesus