NCT02931071

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2013

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2013

Completed
3 years until next milestone

First Submitted

Initial submission to the registry

September 6, 2016

Completed
1 month until next milestone

First Posted

Study publicly available on registry

October 12, 2016

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2018

Completed
Last Updated

May 27, 2020

Status Verified

May 1, 2020

Enrollment Period

5.1 years

First QC Date

September 6, 2016

Last Update Submit

May 25, 2020

Conditions

Fanconi Anemia

Outcome Measures

Primary Outcomes (1)

  • Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)

    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.

    after 12 months

Secondary Outcomes (4)

  • Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor

    after 8 days

  • Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years

    after 8 days

  • Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%

    after 8 days

  • Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years

    after 8 days

Study Arms (1)

plerixafor and filgrastim treatment

EXPERIMENTAL

to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim

Drug: filgrastimDrug: plerixafor

Interventions

filgrastimDRUG

G-CSF (12 μg/Kg/12 h) 8 days.

plerixafor and filgrastim treatment
plerixaforDRUG

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

plerixafor and filgrastim treatment

Eligibility Criteria

Age2 Years - 64 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Infantil Universitario Niño Jesus

Madrid, 28009, Spain

Location

Related Publications (1)

  • Sevilla J, Navarro S, Rio P, Sanchez-Dominguez R, Zubicaray J, Galvez E, Merino E, Sebastian E, Azqueta C, Casado JA, Segovia JC, Alberquilla O, Bogliolo M, Roman-Rodriguez FJ, Gimenez Y, Larcher L, Salgado R, Pujol RM, Hladun R, Castillo A, Soulier J, Querol S, Fernandez J, Schwartz J, Garcia de Andoin N, Lopez R, Catala A, Surralles J, Diaz-de-Heredia C, Bueren JA. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes. Mol Ther Methods Clin Dev. 2021 Jun 12;22:66-75. doi: 10.1016/j.omtm.2021.06.001. eCollection 2021 Sep 10.

    PMID: 34485595DERIVED

MeSH Terms

Conditions

Fanconi Anemia

Interventions

Filgrastimplerixafor

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • Cristina Díaz de Heredia, MD, PhD

    Hospital Vall d'Hebron

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 6, 2016

First Posted

October 12, 2016

Study Start

September 1, 2013

Primary Completion

October 1, 2018

Study Completion

October 1, 2018

Last Updated

May 27, 2020

Record last verified: 2020-05

Locations

ES(2)