Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02931071
Other study ID # FANCOSTEM-1
Secondary ID 2011-006197-88
Status Completed
Phase Phase 2
First received
Last updated
Start date September 2013
Est. completion date October 2018

Study information

Verified date May 2020
Source Hospital Universitari Vall d'Hebron Research Institute
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 13
Est. completion date October 2018
Est. primary completion date October 2018
Accepts healthy volunteers No
Gender All
Age group 2 Years to 64 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
filgrastim
G-CSF (12 µg/Kg/12 h) 8 days.
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

Locations

Country Name City State
Spain Hospital Universitari Vall d'Hebron Barcelona
Spain Hospital Infantil Universitario Niño Jesus Madrid

Sponsors (3)

Lead Sponsor Collaborator
Hospital Universitari Vall d'Hebron Research Institute CIBERER, CIEMAT

Country where clinical trial is conducted

Spain, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter after 8 days
Secondary Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years 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 after 8 days
Secondary Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50% 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% after 8 days
Secondary Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years 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 after 8 days
See also
  Status Clinical Trial Phase
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Recruiting NCT00084695 - Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases Phase 2
Completed NCT00000603 - Cord Blood Stem Cell Transplantation Study (COBLT) Phase 2
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT01146210 - Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia N/A
Completed NCT01082133 - Multicenter Transplant Study for Fanconi Anemia Phase 2
Completed NCT00965666 - Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia Early Phase 1
Terminated NCT00290628 - Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer N/A
Recruiting NCT00027274 - Cancer in Inherited Bone Marrow Failure Syndromes
Terminated NCT01001598 - Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita Phase 1/Phase 2
Recruiting NCT03206086 - Eltrombopag for People With Fanconi Anemia Phase 2
Recruiting NCT03579875 - Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Phase 2
Recruiting NCT05598515 - Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia N/A
Active, not recruiting NCT03476330 - Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia Phase 2
Terminated NCT05910853 - Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
Completed NCT03609840 - Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Completed NCT00479115 - Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 Phase 1/Phase 2
Completed NCT00352976 - TBI Dose De-escalation for Fanconi Anemia Phase 2/Phase 3
Terminated NCT03600909 - A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia Phase 2
Completed NCT03398824 - Pilot Study of Metformin for Patients With Fanconi Anemia Phase 2