BridgeMD
Phase 2 study evaluating the efficacy of injectable gentamicin in hereditary ichthyosis - GENTIC
EU CTIS ID: 2023-505570-15-00
What this study is testing
To evaluate the efficacy of gentamicin on the severity of scales and erythema, at M3 versus baseline in moderate to severe congenital ichthyosis caused by a nonsense mutation
- 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
- Adult patients (≥18 years) affiliated to a social insurance protection regimen
- Hereditiary ichthyosis caused by a homozygous non-sense mutation of a gene responsible for hereditiary ichthyosis (TGM1, PNPLA1, ALOX12B, NIPAL4, ALOXE3, SDR9C7, ABCA12, CERS3, SPINK5 and CDSN)
- Moderate to severe forms of ichthyosis defined as VIIS score at 2-3 on at least 2 out of 4 areas evaluated (back, upper limbs, lower limbs, back of the foot)
- Free, informed consent, written and signed by the participant and the investigator (at the latest on the day of inclusion and before any examination required by the research).
You likely can't join if
- Cutaneous signs suggesting a surinfection
- Patient under guardianship, curatorship or deprived of their liberty
- Variation greater than 15% in the VIIS score between two baseline measurements at the end of the "run-in"period
- Hypersensibility of active substance or one of the gentamicin excipients
- Administration of an aminoside in the previous 3 months
- Treatment with nephrotoxic or ototoxic medication in the previous 6 weeks
See the full eligibility criteria
- Adult patients (≥18 years) affiliated to a social insurance protection regimen
- Hereditiary ichthyosis caused by a homozygous non-sense mutation of a gene responsible for hereditiary ichthyosis (TGM1, PNPLA1, ALOX12B, NIPAL4, ALOXE3, SDR9C7, ABCA12, CERS3, SPINK5 and CDSN)
- Moderate to severe forms of ichthyosis defined as VIIS score at 2-3 on at least 2 out of 4 areas evaluated (back, upper limbs, lower limbs, back of the foot)
- Free, informed consent, written and signed by the participant and the investigator (at the latest on the day of inclusion and before any examination required by the research).
- Cutaneous signs suggesting a surinfection
- Patient under guardianship, curatorship or deprived of their liberty
- Variation greater than 15% in the VIIS score between two baseline measurements at the end of the "run-in"period
- Hypersensibility of active substance or one of the gentamicin excipients
- Administration of an aminoside in the previous 3 months
- Treatment with nephrotoxic or ototoxic medication in the previous 6 weeks
- Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study. Women of childbearing age, potentially sexually active, and unwilling to use acceptable contraceptive measures in accordance with CTFG recommandations
- Subjects >75 years (physiological impairment of kidney function)
- Left ventricular insufficiency
- Hypoalbuminemia
- Myasthenia
- History of necrosis at the injection site during previous treatment with aminosid
- Patient with pre-existing neuromuscular disease
- Patient participating in another clinical study with an investigational treatment
- Grade B or C cirrhosis according to Child-Pugh classification
- Nephropathy or other situation at risk of renal dysfunction
- Renal insufficiency with GFR < 60mL/min
- Surdity which is not caused by plug scales in the external ear canals or other situation at risk of surdity including the presence of the A1555G mutation in the 12S rRNA (mitochondrial DNA) gene
- Patient who modify his keratolytic or emollient treatment in the last two weeks previous the inclusion visit
- Patient who modify his retinoid topic treatment in the month previous the inclusion visit
- Patient who modify his systemic retinoid treatment in the 3 months previous the inclusion visit
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 all sexes, 18-64 years, 65+ 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.