Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
ID
Source
Brief title
Condition
- White blood cell disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
- To demonstrate superiority of RP103 versus Cystagon® in controlling white
blood cell cystine (WBC) levels over 24 hours in patients with
cystinosis.
- To assess long-term safety and tolerability of RP103.
Secondary outcome
- To compare WBC cystine measurements provided by a central laboratory versus
sites* local (regional, not associated with the clinical trial)
laboratories.
- To assess the long term quality of life and sleep using instruments
appropriate to the subjects* age and region (US or Europe).
- To assess dosing compliance with two different cysteamine bitartrate
formulations, Q6H immediate-release Cystagon® vs. Q12H delayedrelease
RP103.
- To assess halitosis during Cystagon® and RP103 treatment in a subgroup of
subjects.
Background summary
Cystinosis is an autosomal recessive inborn error of metabolism in which the
transport of cystine out of lysosomes is reduced or absent. Cystinosis is a
rare disease characterized by the accumulation of cystine and formation of
crystals damaging various organs, especially the kidney, leading to renal
tubular Fanconi Syndrome and progressive glomerular failure, with end stage
renal failure by the end of the first decade of life.
Study objective
Comparison of steady-state cysteamine-trough WBC cystine levels between
Cystagon® and RP103 over 3 months for each treatment period.
Study design
This is a long-term, open-label study of the safety, tolerability and
effectiveness of Cystagon® and RP103 in pediatric and adult subjects with
cystinosis. Subjects 12 years of age or older, with an average of WBC cystine
level > 1 nmol * cystine/mg protein over at least 2 measurements during the
previous 2 years, will be treated with their usual dose of Q6H Cystagon® for 3
months (with no dose adjustments) followed by Q12H RP103 started at a total
daily dose of 70% of their Cystagon® dose for at least 4 months (with dose
increase permitted only during the first month on RP103).
Intervention
Use of RP103 medication.
Study burden and risks
-nr of Blood samples: Op iedere visite wordt bloed afgenomen. Hoeveelheid is
afhankelijk van de visite, tussen de 10 en 20 ml per keer.
-nr of Visits to the investigator ;Minimaal 8 bezoeken tijdens de studie, en
daarna 3-maandelijkse visites met een totaal maximum van 24 maanden.
-Physical Exam: standaard lichamelijk onderzoek op iedere visite, inclusief een
ECG.
-Questionnaires and diaries: Op iedere visite wordt een dagboekje bekeken.
vragenlijsten mbt Kwaliteit van Leven, Vermoeidheid en een VAS score voor
slikklachten.
-physical or psychological discomfort: De bloedafnames zijn wellicht vervelend.
De PK/PD afnames op de vroege ochtend en/of late avond kunnen ook ongemak
veroorzaken.
- Er worden geen andere bijwerkingen verwacht dan de bekende bijwerkingen die
al bekend zijn bij het gebruik van Cystagon.
-De overgang van Cystagon naar RP103 zal naar verwachting een betere kwaliteit
van leven geven, aangezien er door de gunstigere inname tijden een betere
nachtrust verwacht wordt. Ook de reductie van het aantal pillen per inname is
waarschijnlijk een verbetering voor de patient.
Hamilton Landing, Suite 100 7
Novato CA 94949
US
Hamilton Landing, Suite 100 7
Novato CA 94949
US
Listed location countries
Age
Inclusion criteria
1. Male or female with a documented diagnosis of cystinosis.
2. On a stable dose of Cystagon® for at least 21 days prior to Screening.
3. WBC cystine level > 1 nmol * cystine/mg of protein on average over at least 2 measurements
collected during the 2 years prior to Screening.
4. No clinically significant change in liver function tests, i.e. 1.5 times ULN for ALT and AST, and/or
1.5 times ULN for total bilirubin, within 6 months prior to Screening.
Exclusion criteria
1. Younger than 12 years of age.
2. Current history of the following conditions or any other health issues that make it, in the opinion
of the Investigator, unsafe for study participation:
- Inflammatory bowel disease if currently active, or prior resection of small intestine;
- Heart disease (e.g. myocardial infarction, heart failure, unstable arrhythmias, or poorly controlled
hypertension) within 90 days prior to Screening;
- Active bleeding disorder within 90 days prior to Screening;
- History of malignant disease within 2 years prior to Screening.
3. Hemoglobin level of < 9 g/dL at Screening or, in the opinion of the Investigator, a hemoglobin
level that would make it unsafe for study participation.
4. Known hypersensitivity to cysteamine and penicillamine.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | NCT01733316 |
| EudraCT | EUCTR2012-002773-64-NL |
| CCMO | NL41935.091.13 |