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Trial record 12 of 65 for:    "Toni-Debre-Fanconi syndrome"

Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

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ClinicalTrials.gov Identifier: NCT02931071
Recruitment Status : Recruiting
First Posted : October 12, 2016
Last Update Posted : October 12, 2016
Sponsor:
Collaborators:
CIEMAT
CIBERER
Information provided by (Responsible Party):
Hospital Universitari Vall d'Hebron Research Institute

Brief Summary:
Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hematopoietic progenitor cells for later use in another clinical trial entitled "Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells mobilized with mozobil and filgrastim, and transduced with a lentiviral vector carrying the FANCA gene (Orphan Drug) for patients with Fanconi Anemia Subtype A ". The objectives of this study are, therefore, to assess the safety and efficacy of CD34+ cells mobilization with mozobil and filgrastim, which is postulated the most efficient for the collection of CD34+ cells from FA patients.

Condition or disease Intervention/treatment Phase
Fanconi Anemia Drug: filgrastim Drug: plerixafor Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient
Study Start Date : September 2013
Estimated Primary Completion Date : September 2018
Estimated Study Completion Date : September 2018


Arm Intervention/treatment
Experimental: plerixafor and filgrastim treatment
to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim
Drug: filgrastim
G-CSF (12 μg/Kg/12 h) 8 days.

Drug: plerixafor
Plerixafor 0,24 mg/kg/day after the fourth day of G-CSF, and until 5 cells CD34+/μL, max 4 doses of plerixafor




Primary Outcome Measures :
  1. Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0) [ Time Frame: after 12 months ]
    At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.


Secondary Outcome Measures :
  1. Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor [ Time Frame: after 8 days ]
    mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter

  2. Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years [ Time Frame: after 8 days ]
    the CD34+ cell collection protocol will be determined by the percentage of patients who reach at least one million CD34 + cells per kilogram of body weight projected to 5 years after the mobilization process

  3. Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50% [ Time Frame: after 8 days ]
    the of CD34+ cell selection process will be determined by the proportion of immunoselected samples where the recovery of CD34 + cells is at least 50%, and where the final percentage of CD34+ cells is at least 50%

  4. Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years [ Time Frame: after 8 days ]
    will be determined by the percentage of patients who reach at least one million CD34 + cells per kilo of weight projected to 5 years after the immunomagnetic selection process of all the collected cells



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Ages Eligible for Study:   2 Years to 64 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female > 1 year
  • diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C
  • At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3
  • Lansky index> 60%.
  • Left ventricular ejection fraction >50%.
  • To grant informed consent in agreement with current law norms
  • Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last

Exclusion Criteria:

  • Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted
  • Patients with active infection process or any other underlaying severe medical process
  • Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria
  • Haematopoietic transplant
  • Any disease or concomitant process that is not compatible with the study as per investigator opinion
  • Patients not elegible because of an psico-social evaluation
  • Patients that received transfusional support during the last 3 months.
  • Pregnant or breastfeeding women

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02931071


Contacts
Contact: Cristina Díaz de Heredia, MD, PhD +34934893093 crdiaz@vhebron.net

Locations
Spain
Hospital Universitari Vall d'Hebron Recruiting
Barcelona, Spain, 08035
Contact: Cristina Díaz de Heredia, MD, PhD    +34934893093    crdiaz@vhebron.net   
Hospital Infantil Universitario Niño Jesus Recruiting
Madrid, Spain, 28009
Contact: Julian Sevilla Navarro, MD, PhD    0034 91 5035938    jsevilla.hnjs@salud.madrid.org   
Sponsors and Collaborators
Hospital Universitari Vall d'Hebron Research Institute
CIEMAT
CIBERER
Investigators
Principal Investigator: Cristina Díaz de Heredia, MD, PhD Hospital Vall d'Hebron

Responsible Party: Hospital Universitari Vall d'Hebron Research Institute
ClinicalTrials.gov Identifier: NCT02931071     History of Changes
Other Study ID Numbers: FANCOSTEM-1
2011-006197-88 ( EudraCT Number )
First Posted: October 12, 2016    Key Record Dates
Last Update Posted: October 12, 2016
Last Verified: October 2016

Additional relevant MeSH terms:
Fanconi Syndrome
Anemia
Fanconi Anemia
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Lenograstim
JM 3100
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents