Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study

• Register: EUCTR
• Last refreshed on: 23 December 2013
• Main ID: EUCTR2009-012564-13-FR
• Date of registration: 19/06/2013
• Prospective Registration: No
• Primary sponsor: Orphan Europe SARL
• Public title: Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study
• Scientific title: Cysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study - CYSTADROPS CHOC study
• Date of first enrolment: 03/01/2013
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-012564-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: yes Other trial design description: blinded ophtalmologist who perform an independant and masked reading of the results If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Pharmacy hospital formulation Number of treatment arms in the trial: 2

Countries of recruitment

France

Contacts

• Name: Céline Plisson
• Affiliation: Orphan Europe SARL

• Name: Céline Plisson
• Affiliation: Orphan Europe SARL

Key inclusion & exclusion criteria

Inclusion criteria:
- Signed and dated written inform consent form in accordance with local regulations: Having freely given their written informed consent to participate in the study. For patients aged less than 18 years consent will be obtained from the two parents (or legal representatives),
- Diagnosis of cystinosis based on a previous white blood cells cystine concentration > 1.5 nmoles half-cystine per mg protein,
- Presence of corneal crystal deposits attested during a slit-lamp examination within 3 months prior inclusion,
- Ability to comply with their usual eye drops treatment in order to comply with the eyewash regimen of 4 instillations,
- Agreement to move to Ophthalmic Core Laboratory for the assessment visits,
- Likely to be able to participate in all scheduled evaluation and complete all required study procedures,
- In the opinion of the investigator, the patient will be compliant and have a high probability of completing the study.
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 16
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 procedures or for whom a long-term follow-up seems to be difficult to achieve (In the younger children, the confocal microscopy may not be feasible and therefore will not be excluded from the study but only those patients able to undergo the IVCM procedure should be included in the primary analysis).



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

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Nephropatic cystinosis patients with cystine corneal deposits
MedDRA version: 16.0 Level: LLT Classification code 10071112 Term: Nephropathic cystinosis System Organ Class: 100000004850

Intervention(s)

Product Name: CYSTADROPS 0.55% eye drops, solution
Pharmaceutical Form: Eye drops, solution
INN or Proposed INN: CYSTEAMINE HYDROCHLORIDE
CAS Number: 156-57-0
Other descriptive name: Mercaptamine hydrochloride
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 0.55-

Primary Outcome(s)

Primary end point(s): Corneal cystine crystal density measured by IVCM (In Vivo Confocal Microscopy), composite score assessed by independent masked reader.

Timepoint(s) of evaluation of this end point: D1-D30-D90

Main Objective: To compare efficacy of Cystadrops® versus 0.10% cysteamine hydrochloride eye drops solution in terms of superiority in patients with nephropathic cystinosis.

Secondary Objective: To evaluate safety profile of Cystadrops® in patients with nephropathic cystinosis.

Secondary Outcome(s)

Secondary end point(s): - photophobia (rated by investigator and patient)
- CCCS (corneal cystine crystal score by slit-lamp)

Timepoint(s) of evaluation of this end point: D1-D30-D90

Secondary ID(s)

Cystadrops®/09/choc-study

Source(s) of Monetary Support

Orphan Europe SARL