NCT02678533

Brief Summary

The purpose of this study is to assess the feasibility of Plerixafor used in combination with G-CSF (Granulocyte Colony Stimulating Factor) in 5 Fanconi anemia patients to mobilize and collect a sufficient number of peripheral blood CD34+ cells for peripheral blood apheresis, for further gene therapy study.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
4

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2017

Typical duration for phase_1

Geographic Reach
1 country

1 active site

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

First Submitted

Initial submission to the registry

February 5, 2016

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 9, 2016

Completed
1 year until next milestone

Study Start

First participant enrolled

February 10, 2017

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 20, 2018

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 3, 2019

Completed
Last Updated

September 11, 2025

Status Verified

September 1, 2025

Enrollment Period

1.8 years

First QC Date

February 5, 2016

Last Update Submit

September 5, 2025

Conditions

Fanconi Anemia

Keywords

Fanconi AnemiaCD34+ cells mobilizationG-CSFPlerixaforgene therapy

Outcome Measures

Primary Outcomes (1)

  • level of CD34+ cells mobilization

    from day 5 to day 8 after the first injection of G-CSF

Secondary Outcomes (1)

  • number of treatment-related adverse events as a measure of tolerability

    30 days after cytapheresis

Study Arms (1)

Fanconi anemia

EXPERIMENTAL

G-CSF and Plerixafor

Drug: G-CSFDrug: Plerixafor

Interventions

G-CSFDRUG

D1 to D4 : Injection of 12 µg/kg of G-CSF twice a day . D5 : injection of 12 µg/kg of G-CSF (once/ twice a day according to cytapheresis's realization)

Fanconi anemia
PlerixaforDRUG

D5 : injection of 24mg/kg of plerixafor once a day until cytapheresis has be done (maximum of 4 days)

Fanconi anemia

Eligibility Criteria

Age2 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patient with Fanconi anemia
  • Patient from 2 to 17 years old
  • Potential indication for allogenic bone arrow graft without HLA-identical brotherhood available
  • Patient's weight \>10kg
  • Treated and followed for at least the previous two years in a specialized center where they got a full assessment of their disease
  • For women of childbearing age, not pregnant and use of an effective contraception during the entire participation in the research.
  • Affiliated or beneficiary of an health insurance regimen
  • Informed and signed consent

You may not qualify if:

  • Patient unable to follow the visits required by the protocol
  • Positive serology for HIV-1/2, HTLV-1/2, HCV and HbS
  • Bacterial, viral, fungal or parasitic active infection with clinical signs
  • Personal history of cancer, myeloproliferative hematopathy or immune deficiency
  • Heart failure and / or heart rhythm disorder
  • History of allogeneic graft of hematopoietic stem cells
  • Patient with an HLA-identical brotherhood donor available
  • Myelodysplasia diagnose on myelogram
  • Cytogenetic abnormality on karyotype
  • Malignant solid tumor
  • Documented spontaneous genetic reversion of medullary process
  • Diagnosis of a psychiatric disorder that could compromise his/her ability to participate in the study
  • Any disorder according to the investigator, that could compromise the ability of patient to give his writing consent and/or to comply with requiring study's procedures
  • Current Pregnancy
  • Heart, kidney or liver failure
  • +4 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hôpital necker-Enfants malades

Paris, PARIS, 75015, France

Location

Related Publications (1)

  • Diana JS, Manceau S, Leblanc T, Magnani A, Magrin E, Bendavid M, Couzin C, Joseph L, Soulier J, Cavazzana M, Lefrere F. A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy. Transfusion. 2022 Jan;62(1):165-172. doi: 10.1111/trf.16721. Epub 2021 Nov 9.

    PMID: 34751952BACKGROUND

MeSH Terms

Conditions

Fanconi Anemia

Interventions

Granulocyte Colony-Stimulating Factorplerixafor

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)

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

Study Officials

  • Marina CAVAZZANA, MD, PhD

    AP-HP, Necker hospital

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 5, 2016

First Posted

February 9, 2016

Study Start

February 10, 2017

Primary Completion

November 20, 2018

Study Completion

May 3, 2019

Last Updated

September 11, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)