ICTRP Search Portal

Main

Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	EUCTR
Last refreshed on:	23 December 2013
Main ID:	EUCTR2007-006024-35-FR
Date of registration:	12/12/2007
Prospective Registration:	Yes
Primary sponsor:	Orphan Europe SARL
Public title:	Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot study
Scientific title:	Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study - CYSTADROPS OCT-1 pilot study
Date of first enrolment:	04/02/2008
Target sample size:
Recruitment status:	Not Recruiting
URL:	https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-006024-35
Study type:	Interventional clinical trial of medicinal product
Study design:	Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
Phase:

Countries of recruitment
France

Contacts

Name:
Address:
Telephone:
Email:
Affiliation:

Name:
Address:
Telephone:
Email:
Affiliation:

Key inclusion & exclusion criteria

Inclusion criteria:
Treated with reference Cysteamine eye drops at least for 1 month at an usual regimen dose of at least 3 instillations per day
Able to comply with a regular dose regimen for 6 months

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
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:
- Patients with uncontrolled hepatic disorder, cardiovascular disease, neurologic disease, or cancer,
- Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if the investigator considers that they are not clinically significant for the conduct of study,
- Patients with history or presence of alcohol abuse or drug addiction,
- Pregnant or breast-feeding women,
- Women of child-bearing potential without effective contraception (oral pill or IUCD),
- Patients likely to be non-compliant to the study procedure or for whom a long-term follow-up seems to be difficult to achieve.

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

Health Condition(s) or Problem(s) studied

Nephropathic cystinotic patients with cystine crystals corneal deposits
MedDRA version: 9.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders

Intervention(s)

Product Name: CYSTADROPS
Pharmaceutical Form: Eye drops*
INN or Proposed INN: cysteamine hydrochloride
CAS Number: 156-57-0
Other descriptive name: mercaptamine, mercamine, 2-aminoethanethiol
Concentration unit: % percent
Concentration type: equal
Concentration number: 0.55-

Primary Outcome(s)

Secondary Objective: - to compare efficacy of Cystadrops versus a recommended dose regimen reference treatment
- to find the lowest efficient dose according to an empiric adaptive dose regimen algorithm
- to evaluate the latest developments of measurement methods for corneal topography and cystine crystals deposit assessment.

Primary end point(s): 1-Safety
The safety will be based on the assessment and the recording of the adverse events, physical examinations, laboratory parameters and ocular findings including:
oTransitional ocular symptoms evaluated by patient and/or parents:
- Redness
- Blurring
- Irritation
- Itching
- Burning
- Discomfort
- Pain
o Ocular tonometry
o Eye fundus examination
o Anterior segment topography (Optical Coherence Tomography, slit-scanning)

2- Efficacy
o Clinical
o Photophobia: 0-5 points scale
o Pain: Visual Analogic Scale
o Visual acuity (ETDRS chart, contrast scale)
o Imaging: corneal crystals deposits measurement
o HRT confocal biomicroscopy (Heidelberg Retina Tomograph)
o Anterior segment topography (Optical Coherence Tomography)
o Slit-lamp CCCS assessment (Cystinosis Corneal Crystal Score)

3- Compliance
o Vials weight
o Diary card

Main Objective: To compare safety of Cystadrops versus a recommended dose regimen reference treatment

Secondary Outcome(s)

Secondary ID(s)

cysteamine gel formulation/07/cysta

Source(s) of Monetary Support

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.