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Studying the effects of deferiprone in Pelizaeus-Merzbacher disease
EU CTIS ID: 2024-511968-81-00
What this study is testing
To evaluate the effect of deferiprone on gross motor function in children with PMD
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male whose age at screening is ≤ 8 years
- Genetically proven PMD with a hemizygous clinically relevant missense mutation in the PLP1 gene or a PLP1 triplication (or higher copy numbers) and a brain MRI compatible with the diagnosis.
- Lives within reasonable travel distance from Amsterdam.
- Possibility of weekly capillary blood sampling at or close to home.
- Connatal or classic form of the disease (defined as not being able to sit without support and/or a mutation predicting this form, e.g. PLP1 duplication or higher copy numbers; known missense mutations associated with severe forms).
You likely can't join if
- Patients with PLP1 duplications.
- Iron deficiency
- History of neutropenia in the last 12 months (absolute neutrophile count < 1.5 X 109/l)
- Clinically asymptomatic
- Comorbidity with another genetic defect, e.g. Down syndrome or other genetic disorders with impaired development.
- Presence of an unrelated serious condition (e.g. developmental anomaly, significant cardiac, liver, blood or kidney disease or malignancy).
See the full eligibility criteria
- Male whose age at screening is ≤ 8 years
- Genetically proven PMD with a hemizygous clinically relevant missense mutation in the PLP1 gene or a PLP1 triplication (or higher copy numbers) and a brain MRI compatible with the diagnosis.
- Lives within reasonable travel distance from Amsterdam.
- Possibility of weekly capillary blood sampling at or close to home.
- Connatal or classic form of the disease (defined as not being able to sit without support and/or a mutation predicting this form, e.g. PLP1 duplication or higher copy numbers; known missense mutations associated with severe forms).
- Patients with PLP1 duplications.
- Iron deficiency
- History of neutropenia in the last 12 months (absolute neutrophile count < 1.5 X 109/l)
- Clinically asymptomatic
- Comorbidity with another genetic defect, e.g. Down syndrome or other genetic disorders with impaired development.
- Presence of an unrelated serious condition (e.g. developmental anomaly, significant cardiac, liver, blood or kidney disease or malignancy).
- Participation in another clinical study with therapeutic intervention.
- Unable to undergo MRI due to metal-containing implants, such as cochlea implant, neurostimulator or pacemaker.
- Known allergy or hypersensitivity to deferiprone or to any of the other components of the formulation used in this study.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.