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Main
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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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16 May 2022 |
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Main ID: |
NCT02012114 |
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Date of registration:
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11/12/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications
Crystobs |
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Scientific title:
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A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications |
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Date of first enrolment:
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December 2011 |
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Target sample size:
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65 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02012114 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).
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Phase:
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N/A
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Countries of recruitment
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France
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Contacts
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Name:
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Cochat Pierre, PUPH |
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Address:
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Telephone:
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Email:
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Affiliation:
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Hospices Civils de Lyon |
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Key inclusion & exclusion criteria
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- Male and female subjects with confirmed diagnosis of nephropathic cystinosis (defined
by clinical signs and WBC cystine level).
- Age > 4 years.
- Subjects receiving any oral cysteamine treatment: Cystagon or RP103.
- Sexually active female subjects of childbearing potential must agree to utilize the
same acceptable form of contraception from day 1 through completion of the study.
- Subjects or their parent or legal guardian must provide written informed consent prior
to participation in the study.
- Subjects covered by or having the right to social security.
Exclusion Criteria:
- Subjects with known hypersensitivity to cysteamine and penicillamine.
- Females who are nursing, planning a pregnancy, known or suspected to be pregnant, or
with a positive urinary pregnancy test.
- Subjects who, in the opinion of the Investigator, are not able or willing to comply
with the protocol.
- Contra-indication to MRI assessment
CONTROLS FOR METABONOMIC ASSESSMENTS
Inclusion Criteria:
- Age and sex matched to study population: Age range: patients aged 4 to 5 years, 6 to 11
years, patients aged 12 to 17 years, adult patients: 18 or above.
Sex: male or female
- Subjects or their parent or legal guardian must provide written informed consent prior
to participation in the study.
- Subjects covered by or having the right to social security.
Exclusion Criteria:
- Any uropathology or nephropathology.
CONTROLS FOR NMRS ASSESSMENTS
Inclusion Criteria:
- Age and sex matched to study population: Age range: patients aged 4 to 5 years, 6 to 11
years, patients aged 12 to 17 years, adult patients: 18 or above.
Sex: male or female
- Subjects or their parent or legal guardian must provide written informed consent prior
to participation in the study.
- Subjects covered by or having the right to social security.
Exclusion Criteria:
- Any uropathology or nephropathology.
- Any neurological and/or psychiatric disorder
- Contra-indication to MRI assessment.
Age minimum:
4 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystinosis
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Intervention(s)
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Other: Cysteamine bitartrate
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Primary Outcome(s)
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WBC cystine levels
[Time Frame: Day1and every three months : Month1, Month3, Month6, Month9, Month12, Month15, Month18, Month21 and Month24.]
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Secondary Outcome(s)
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Memory and visuoperceptual tests repeated during the study
[Time Frame: at Day 1 and two years]
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concentration of cysteamine in blood (measured by toxicological HPLC analysis with fluorescence detection)
[Time Frame: at Day1 and every 3 months : month1, month3, month6, month9, month12, month15, month18, month21, month24]
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Presence or absence and accountability of cystine crystals.
[Time Frame: Every 6 month : Month1, Month6, Month12, Month18, Month24]
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Concentration effect, dose effect model.
[Time Frame: at month 6]
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Concentration of cysteamine in the CSF, associated to a measurement of the CSF pressure
[Time Frame: at any visit]
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discrimination of urine and blood samples,from metabolic spectroscopic data obtained by nuclear magnetic resonance.
[Time Frame: Every 3 months during 6 months and every 6 months to month 6 until two years]
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presence or absence of cystine accumulation, determination of the sites of cystine accumulation in the CNS and relationship with the compliance to cysteamine treatment, relationship with neuropsychological status.
[Time Frame: at Month 1 and two years]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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