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.

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

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-006100-12-ES

Enrollment

Registered

2012-01-27

Start date

2013-04-12

Completion date

Unknown

Last updated

2013-05-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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 Co

Eligibility

Sex/Gender

All

Inclusion 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

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).

Design outcomes

Primary

Measure	Time frame
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.;Secondary Objective: Not applicable;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.;Timepoint(s) of evaluation of this end point: Up to 2 years after infusion	—

Measure

Time frame

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.;Secondary Objective: Not applicable;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.;Timepoint(s) of evaluation of this end point: Up to 2 years after infusion

—

Secondary

Measure	Time frame
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	—

Countries

Spain

Contacts

Public ContactServicio Oncohematologia

Hospital Niño Jesus

jsevilla.hnjs@salud.madrid.org+34915035938

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026