Efficacy of Injectable Gentamicin in Hereditary Ichthyosis

NCT06362447 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: RC31/22/0320
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Ichthyosis

Keywords

Congenital ichthyoses; injectable gentamicin

Outcome Measures

Primary Outcomes (1)

• Gentamicin efficacity

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

  3 months

Secondary Outcomes (2)

• Gentamicin efficacity

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

• Gentamicin efficacity on quality of life

  Month 3, Month 6 and Month 9

Study Arms (1)

Gentamicin

EXPERIMENTAL

Gentamicin injection

Drug: Gentamicin Injectable Solution

Interventions

Gentamicin Injectable Solution DRUG

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

Gentamicin

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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
• 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 contraception measures in accordance with Clinical Trials Facilitation and Coordination Group recommendations
• 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
• Grade B or C cirrhosis according to Child-Pugh classification
• Nephropathy or other situation at risk of renal dysfunction
• Renal insufficiency with glomerular filtration rate \< 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 ribonucleic acid (mitochondrial deoxyribonucleic acid) gene
• Patient under guardianship, curatorship or deprived of their liberty

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (2)

Hôpital Saint-Louis APHP

Paris, France, 75010, France

Location

CHU de Toulouse

Toulouse, France, 31059, France

Location

MeSH Terms

Conditions

Ichthyosis

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• SEVERINO-FREIRE Maella, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

Central Study Contacts

SEVERINO-FREIRE Maella, MD

CONTACT

05 67 77 81 41; severino-freire.m@chu-toulouse.fr

TEXIER Hélène

CONTACT

05 67 77 81 80; texier.h@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2024
• First Posted: April 12, 2024
• Study Start: September 1, 2024
• Primary Completion: April 1, 2026
• Study Completion (Estimated): April 1, 2027
• Last Updated: September 5, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)