Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: Trade Data, Destination Markets, and Economic Impact
Clinical Trials in France / NCT07868120
Starting soon Not applicable

Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN)

NCT07868120 · tracked via the Priya Life Science France tracker
Sponsor
University Hospital, Bordeaux
Phase
Not applicable
Started
2026-12
Last updated
2026-10-09

Condition(s) studied

Neurodegeneration With Brain Iron Accumulation (NBIA)Iron Metabolism DisordersBrain Disease

Investigational drug(s) / intervention(s)

Standardized brain MRI including specific sequences usable for Quantitative Susceptibility Mapping (QSM)

Standardized brain MRI including specific sequences usable for Quantitative Susceptibility Mapping (QSM): All participants will undergo standardized study procedures at two time points (baseline and 18 months), including brain MRI acquisition and clinical assessments. Brain MRI will be performed using a standardized protocol, including conventional sequences and additional sequences required for Quantitative Susceptibility Mapping (QSM), such as multi-echo gradient-echo acquisitions sensitive to magnetic susceptibility variations. No contrast agent will be administered. QSM maps will be reconstructed using validated post-processing methods, and quantitative analyses will be conducted in predefined regions of interest (e.g., substantia nigra, globus pallidus) to estimate intracerebral iron load. At each visit, participants will undergo clinical evaluations, including neurological examination and assessment of motor and adaptive functions using validated scales. All procedures will be repeated at 18 months under similar conditions to enable longitudinal intra-individual comparisons.

Study summary

This study aims to quantify intracerebral iron load in patients with Beta-Propeller Protein-Associated Neurodegeneration (BPAN) using Quantitative Susceptibility Mapping (QSM) technique. The primary objective is to assess the longitudinal robustness of QSM measurements over an 18-month follow-up. This pilot study also explores the relationship between iron accumulation and clinical severity. Ultimately, it seeks to validate QSM as a non-invasive biomarker for disease monitoring and future therapeutic trials

Eligibility

Sex
ALL
Min age
5 Years
Max age
18 Years
Healthy volunteers
No
Inclusion Criteria: * Affiliation to a social security system * French-speaking participants * Age between 5 and 18 years * Genetically confirmed BPAN (WDR45 mutation) * Written and free informed consent from at least one parent (e.g., in single-parent families) or the holder(s) of parental responsibility. Exclusion Criteria: * Patient-specific exclusion criteria : o Participation in an interventional clinical trial that could interfere with our study or that includes an ongoing exclusion period * Contraindications to brain MRI : * Presence of a pacemaker, neurostimulator, or implantable defibrillator (risk of temporary or permanent damage to the device, deprogramming, or heating of metal components) * Presence of cochlear implants (risk of demagnetization, electrode overheating, and artifacts) * Presence of ferromagnetic foreign bodies in the eye or brain near nerve structures (risk of displacement and complications such as brain injury or blindness) * Presence of metal prostheses, particularly orthodontic braces * Presence of a ventriculoperitoneal shunt * Contraindications related to anesthesia: * Known allergy to one or more products used for anesthesia

Primary outcome measure(s)

  • Brain Iron — Baseline (T0), month 18 (T18).
    Quantitative change on intracerebral iron levels using QSM technology in BPAN patients between baseline (Day 0) and 18 months

Trial sites (1)

FacilityCityRegionStatus
CHU de Bordeaux Bordeaux France

More University Hospital, Bordeaux trials in France

Labor Induction in Low-risk Nulliparous Women at 39 Weeks of Gestation to Reduce Cesarean: A Randomized Trial of Induction Versus Expectant Management in France (FRENCH-ARRIVE) Recruiting
SAGE-Heart : Senior Assessment fraGility and Post-opErative QALYs 1 Year After Emergency HEART Surgery for Patients Aged 75 and Older Recruiting
Venetoclax in Association With 3+7 and Midostaurin in FLT3-mutated Acute Myeloid Leukemia Starting soon
Modeling the Early Stages of Autoimmunity Through the Study of Immunological Thrombocytopenic Purpura and Juvenile Lupus Starting soon
Outpatient Holmium LASER Enucleation of the Prostate: Benefit of MOSES(TM) 2.0 Technology Active, not recruiting

Other trials for the same condition

Study of Markers of Iron Metabolism and Their Relationship With Phosphocalcic and Hepatic Metabolism and Inflammation in Hemodialysis Patients Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07868120 on ClinicalTrials.gov ↗ ← All trials in France