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Efficacy of Injectable Gentamicin in Hereditary Ichthyosis

ConditionIchthyosis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorUniversity Hospital, Toulouse

About this trial

This study will evaluate the efficacy and safety of intravenous gentamicin in congenital ichthyosis due to a non-sens mutation. The primary objective is the severity of scales and erythema at the third month, compared to baseline. Secondary objectives will include: the importance of itching, trans epidermal water loss, cutaneous expression of the targeted protein, the security of the drug and patients' satisfaction.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult patients affiliated to a social insurance protection regimen.

Hereditary ichthyosis caused by a homozygous non-sense mutation of a gene responsible for hereditary ichthyosis (TGM1, PNPLA1, ALOX12B, NIPAL4, ALOXE3, SDR9C7, ABCA12, CERS3, SPINK5 and CDSN)

Moderate to severe forms of ichthyosis defined as Validating an Ichthyosis Severity Index 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).

Disqualifiers

Cutaneous signs suggesting a surinfection

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Gentamicin Injectable Solution

    Drug

    Gentamicin (10 mg/kg) will be administrated once weekly for 3 months

Treatment groups

26 Participants
are divided into 1 treatment group
Group A: GentamicinExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Gentamicin efficacity

Proportion of patients with a reduction in Visual Index of Ichthyosis Severity score of at least 15%

Time frame
3 months

Secondary outcomes

1

Gentamicin efficacity

Proportion of patients with a reduction in Visual Index of Ichthyosis Severity score

Time frame
Month 1, Month 2, Month 4, Month 5, Month 6 and Month 9
2

Gentamicin efficacity on quality of life

Assessment of quality of life by IQoL-32 score (specific to ichthyosis)

Time frame
Month 3, Month 6 and Month 9

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University Hospital, Toulouse

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: