Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-01-07

Study Completion Date

2023-09-08

Brief Summary

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This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .

CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.

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Detailed Description

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The main objective of this open-label Phase I / II clinical trial is to evaluate the safety and therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi's Anemia Subtype A.

The drug to be administered to the patients consists of the cellular product resulting from the transduction of autologous CD34 + cells with the therapeutic lentiviral vector PGK-FANCA.Wpre \*.

The dose of cells to infuse in the patients will be that obtained from the transduction process of between 3x10\^5 and 4x10\^6 CD34 + cells / kg of patient body weight.

The cells will be infused intravenously in a single dose, after complete the transduction process.

Follow-up period: 3 years after infusion of transduced cells. However, patients will be monitored outside the clinical trial over a 10-year period.

Follow-up of the grafted transduced cells will be performed on peripheral blood and bone marrow samples.

Conditions

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Fanconi Anemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Autologous CD34+ cells transducted with PGK-FANCA-Wpre *

CD34 + cells from patients with Fanconi subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the gene FANCA, PGK-FANCA-Wpre\*The product to be infused consist of a suspension of transduced CD34\^+ cells.

Group Type EXPERIMENTAL

IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)

Intervention Type PROCEDURE

Genetically Engineered Hematopoietic Stem/Progenitor Cells

Intervention Type BIOLOGICAL

Undergo infusion of genetically modified hematopoietic progenitor cell therapy

Laboratory Biomarker Analysis

Intervention Type OTHER

Correlative studies

Filgrastim

Intervention Type BIOLOGICAL

Given subcutaneously (SC)

Plerixafor

Intervention Type DRUG

Given SC

Bone Marrow Aspiration

Intervention Type PROCEDURE

Interventions

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IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)

Intervention Type PROCEDURE

Genetically Engineered Hematopoietic Stem/Progenitor Cells

Undergo infusion of genetically modified hematopoietic progenitor cell therapy

Intervention Type BIOLOGICAL

Laboratory Biomarker Analysis

Correlative studies

Intervention Type OTHER

Filgrastim

Given subcutaneously (SC)

Intervention Type BIOLOGICAL

Plerixafor

Given SC

Intervention Type DRUG

Bone Marrow Aspiration

Intervention Type PROCEDURE

Other Intervention Names

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Genetically Engineered HSPCs Filgrastim XM02, Filgrastim-sndz, G-CSF (Colony Stimulating Factor), Neupogen, r-metHug-CSF, Recombinant Methionyl Human Granulocyte CSF, rG-CSF, Tbo-filgrastim, Zarxio AMD 3100, JM-3100, Mozobil, SDZ SID 791

Eligibility Criteria

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Inclusion Criteria

* Patients diagnosed with Fanconi Anemia complementation group A (FA-A)
* Minimum age 1 year
* Maximum age 21 years
* Lansky Index\> 60%.
* Informed consent in accordance with current legal regulations.
* Number of cells to be transduced: At least 3x10\^5 purified CD34+ / kg body weight.
* Negative result in the urine pregnancy test at the baseline visit for women of childbearing age, who should be committed to using an effective contraceptive method during the period of study participation.

Exclusion Criteria

* Patients with an human leukocyte antigen (HLA) identical family donor.
* Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predicting the same in bone marrow aspirates. In this case, the studies carried out two months in advance of the patient's entry into the clinical trial will be considered valid.
* Evidence that the patient to be infused has signs of somatic mosaicism, with hematologic improvement.
* Any illness or concomitant process that in the opinion of the investigator incapacitates the subject for their participation in the study.
* Pre-existing sensory or motor impairment\> = grade 2 according to the National Cancer Institute (NCl) criteria.
* Pregnant or lactating women.

Minimum Eligible Age

1 Year

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Julian Sevilla

M.D.PhD Specialist in Hematology Hemotherapy, Responsible for the Transfusion Service and Unit for the Obtention and Processing of Hematopoietic Progenitors and other cellular therapies at the Hospital Infantil Universitario Niño Jesús de Madrid.

Responsibility Role PRINCIPAL_INVESTIGATOR