Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients

• Register: EUCTR
• Last refreshed on: 31 August 2015
• Main ID: EUCTR2013-003228-35-ES
• Date of registration: 12/11/2013
• Prospective Registration: Yes
• Primary sponsor: Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
• Public title: Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients
• Scientific title: Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients - Viscou solution cysteamina
• Date of first enrolment: 16/07/2014
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-003228-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Results of each patient will be compared with results with its previous treatment Number of treatment arms in the trial: 1

Countries of recruitment

Spain

Contacts

• Name: Study coordinator
• Affiliation: Dra. Nieves Martin

• Name: Study coordinator
• Affiliation: Dra. Nieves Martin

Key inclusion & exclusion criteria

Inclusion criteria:
Patients must meet each of the following criteria to be eligible for admission to the study:
-Cystinosis patients treated with cysteamine 0.55% eye drops (saline solution), made ??by the Pharmacy Department of Hospital Universitari Vall d'Hebron.
-Age ? 5 years
-Cooperative and compliant patient
-Parents who sign the consent or acquiescence in patients over 12 years old.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
-Using other eye topical treatments.
-Withdrawal of consent or acquiescence.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystinosis
MedDRA version: 16.1 Level: PT Classification code 10011777 Term: Cystinosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: cysteamine (mercaptamine) viscous solution
Pharmaceutical Form: Eye drops, solution
INN or Proposed INN: Mercaptamine
CAS Number: 156-57-0
Other descriptive name: MERCAPTAMINE HYDROCHLORIDE
Concentration unit: % percent
Concentration type: equal
Concentration number: 0.55-

Primary Outcome(s)

Main Objective: The main objective of the
study is to assess the efficacy of 0.55% cysteamine formulated in viscous solution compared with saline solution at the same dose, in corneal crystal deposition.

Primary end point(s): Number of corneal cystine crystals

Timepoint(s) of evaluation of this end point: 3 months

Secondary Objective: -The secondary objective of the study is to assess whether the efficacy of 0.55% cysteamine formulated viscous solution administered four times a day is comparable to more frequent administration.
-To evaluate the tolerance and compliance with the new formulation.

Secondary Outcome(s)

Secondary end point(s): Number of corneal cystine crystals (4 times a day)
Number of prescription instillations/ number of instillations performed

Timepoint(s) of evaluation of this end point: 3 months

Secondary ID(s)

18072013

Source(s) of Monetary Support

VHIR