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WithdrawnNCT04656171MicroFancIIUpdated Feb 3, 2023

Microcephaly, Fanconi Anemia and Praxial Disorders

An interventional study of MRI of the hand and forearm, in Fanconi Anemia, sponsored by Assistance Publique - Hôpitaux de Paris. Withdrawn at 1 site in France. Open to participants aged 5 Years to 21 Years. Per ClinicalTrials.gov, last updated 2023-02-03.

Sponsored by Assistance Publique - Hôpitaux de Paris · Not applicable, Interventional, and Other

Why this study was withdrawn
No financement AFAM
Phase
Not applicable
Study type
Interventional
Enrollment
0
Allocation
Non-randomized
Ages
5 Years to 21 Years
Sex
All
01

Study summary

Fanconi Anemia (FA) is mentioned in children with congenital malformations including kidney, hart and skeletal malformations (absence or abnormal thumb or forearm), and bone marrow failure or myelodysplasia with a progressive onset in childhood or adulthood. No study has focused on microcephaly, a reduction in brain volume, which is present in 20% of children, and its consequences on cognitive and structural level of the brain. Since 2014, Robert-Debré's team has been interested in this functional cognitive and neuroanatomical approach trough a National PHRC. Preliminary results carried out on 12 children show that their intellectual efficiency was in the normal range for age. However, we noticed a significant difference between abilities in comprehension and verbal reasoning corresponding to what is expected for age, and the sensorimotor skills or fine motor praxia significantly reduced. These difficulties, graphically penalizing for these children, are not always explained by a skeletal malformation of the upper limb, suggesting that musculo-tendinous anomalies may be associated. The objectives of our project are: 1) to identify upper limb musculo-tendinous abnormalities and their functional consequences, 2) to determine if these abnormalities could influence the somatosensory representation of the upper limb at the cerebral cortical level. This project should help us to better understand the fine motor disabilities or developmental coordination disorder of these children, which penalize their learning, and provide them with adapted solutions.

Read the detailed description

Our hypothesis is that children with FA present a developmental dyspraxia. This condition is very penalizing for children especially regarding graphic tasks, handwriting, whether or not they have skeletal malformations of the upper limbs. Consequences are fatigue because of energy expended trying to execute fine motor movements correctly.

Main objective:

To identify gesture dyspraxia in order to propose a targeted rehabilitation leading to national recommendations.

Main Evaluation Criteria :

  1. measurement of fine motor praxia
  2. quantification of dyspraxia

Secondary Objectives :

To identify the musculoskeletal or tendinous anomalies in the upper limbs of AF children and to assess their functional consequences.

To determine if these upper limbs abnormalities could influence the somatosensory map of this part of the body in the cerebral cortex.

Secondary Evaluation Criteria :

  1. MRI of the hand and forearm, orthopedic examination and functional assessment
  2. Previously obtained brain MRI data
02

Conditions studied

03

Who can participate

Ages eligible
5 Years to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Patients with Fanconi Anemia defined according to two of the following diagnostic criteria already included in the MicroFanc study:

    • Chromosome breakage test after exposure to an alkylating agent (mitomycin) on peripheral blood lymphocytes.
    • FancD2 test on lymphocytes or fibroblasts
    • sensitivity of fibroblasts to mitomycin
    • mutation in one of the FANC complementation genes (A, B, C, D1, D2, E, F, G, I, J, L, M, N)
  2. Non-transplanted patients or patients at a distance from CSH transplant (>3 years)
  3. Age ≥5 years of age at inclusion (minimum age of accessibility for neuropsychological tests and no need for sedation for MRI)

Exclusion criteria

Exclusion Criteria:

Subjects for whom both parents have not agreed to participate in the research, or for whom MRI is contraindicated.

04

Study design

Phase
Not applicable
Primary purpose
Other
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    Minor patients with Fanconi anemia

    MRI of hands and forearm, neuropsychological and neuromotor tests

    Radiation: MRI of the hand and forearm,

  • Active comparator
    Minor controls

    MRI of the hand and forearm, orthopedic evaluation, neuromotor tests of the upper limbs, praxies evaluation, neurocognitive evaluation

    Radiation: MRI of the hand and forearm,

Interventions

  • RadiationMRI of the hand and forearm,

    MRI of the hand and forearm,

05

What researchers measure

Primary outcomes

  1. measurement of fine motor praxia

    Time frame: 24 months

06

Study locations

1 site
  • Robert Drbré Hospital
    Paris, 75019, France
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT04656171
Lead sponsor
Assistance Publique - Hôpitaux de Paris
Responsible party
Sponsor
First posted
Dec 7, 2020
Start date
Jan 30, 2023
Primary completion
Jan 30, 2023
Completion
Jan 30, 2023
Last update
Feb 3, 2023

Study contacts

Sandrine Passemard, MD
principal investigator · Assistance Publique - Hôpitaux de Paris

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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