Fanconi Anaemia
Conditions
Interventions
Trade Name: Mozobil
Product Name: Mozobil
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Mozobil
Current Sponsor code:
Sponsors
Great Ormond Street Hospital NHS foundation Trust
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion criteria • Patients diagnosed with FA confirmed by chromosomal instability test with diepoxybutane or mitomycin C. • Age 1-15 years • At least one of the following parameters must exceed the values indicated: haemoglobin: 8.0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3. • Lansky index > 60%. • Left ventricular ejection fraction > 50%. • Provide informed consent in accordance with current legislation. Parent/Guardian consent and assent where appropriate • Women of childbearing potential must have a negative pregnancy test in serum or urine at the screening visit, and accept the use of adequate contraception method from, at least, the 14 days prior to the first dose of mobilisation treatment until 14 days after the last.(N/A for children) Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Exclusion criteria • Evidence of myelodysplastic syndrome or leukaemia, or cytogenetic abnormalities predictive of these conditions in bone marrow aspirate analysis. This assessment should be made by valid studies two months before the initial assessment. • Patients with active infectious process or other serious underlying medical condition including malignancy. • Severe (= grade 3) functional organ impairment (liver, kidney, respiratory) according to the criteria of the National Cancer Institute (NCI CTCAE v4.03). • Previously received haematopoietic transplantation. • Any concomitant disease or condition that, in the opinion of the investigator, deems the subject unfit to participate in the study. • Patients who, after a psychosocial assessment, are censored as unfit for the procedure. • Patients who have received blood transfusions in the previous three months. • Pregnant or breastfeeding women (N/A for children)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective is to determine the safety of mobilising CD34+ cells after treatment with filgrastim and plerixafor for subsequent use in a gene therapy trial in patients diagnosed with Fanconi anaemia.; Secondary Objective: The secondary objectives: •To determine the efficacy of mobilising CD34+ cells into peripheral blood after treatment with filgrastim and plerixafor in patients diagnosed with Fanconi anaemia. • To determine the efficacy of CD34+ cell collection after treatment with filgrastim and plerixafor in patients diagnosed with Fanconi anaemia. • To determine the efficacy of selecting haematopoietic progenitors (CD34+ cells) from the product of apheresis using an immunomagnetic procedure. ;Primary end point(s): The primary outcome measure is to determine the safety of mobilising CD34+ cells after treatment with filgrastim and plerixafor for subsequent use in a gene therapy trial in patients diagnosed with Fanconi anaemia.;Timepoint(s) of evaluation of this end point: Will be discussed with Collaborators | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - To determine the efficacy of mobilising CD34+ cells into peripheral blood after treatment with filgrastim and plerixafor in patients diagnosed with Fanconi anaemia. - To determine the efficacy of CD34+ cell collection after treatment with filgrastim and plerixafor in patients diagnosed with Fanconi anaemia. - To determine the efficacy of selecting haematopoietic progenitors (CD34+ cells) from the product of apheresis using an immunomagnetic procedure. ;Timepoint(s) of evaluation of this end point: Will be discussed with Collaborators | — |
Countries
France, Spain, United Kingdom
Contacts
Public ContactHavinder Hara
University College London-Institute of Child Health
Outcome results
None listed