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Cystagon Medical Research Studies

Up-to-date List of Cystagon Medical Research Studies

What Research is Being Done?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies. Following list includes both interventional and observational studies.

Latest Cystagon Medical Research Studies

Rank Status Study
1 Recruiting Open-Label Safety & Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cystinosis
Condition: Cystinosis
Interventions: Drug: RP103 Q12H;   Drug: Cystagon Q6H
Outcome Measures: White Blood Cell (WBC) Cystine Levels;   Long-Term Safety and Tolerability;   Quality of Life - General;   Quality of Life - Fatigue/Sleep
2 Recruiting Safety/Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Condition: Cystinosis
Intervention: Drug: RP103 Q12H
Outcome Measures: White Blood Cell (WBC) Cystine Levels;   Long-Term Safety and Tolerability
3 Recruiting Open-Label, Dose-Escalating Study to Assess Safety, Tolerability, Efficacy, PK and PD of RP103 in Children With Inherited Mitochondrial Disease
Condition: Inherited Mitochondrial Disease, Including Leigh Syndrome
Intervention: Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
Outcome Measures: Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score;   Change over time in Pharmacodynamic Biomarkers
4 Recruiting A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications
Condition: Cystinosis
Intervention: Other: Cysteamine bitartrate
Outcome Measures: WBC cystine levels;   Presence or absence and accountability of cystine crystals.;   Memory and visuoperceptual tests repeated during the study;   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.;   Concentration of cysteamine in the CSF, associated to a measurement of the CSF pressure;   concentration of cysteamine in blood (measured by toxicological HPLC analysis with fluorescence detection);   discrimination of urine and blood samples,from metabolic spectroscopic data obtained by nuclear magnetic resonance.;   Concentration effect, dose effect model.
5 Recruiting Use of Cysteamine in the Treatment of Cystinosis
Condition: Cystinosis
Intervention: Drug: Cysteamine
Outcome Measures: Survival;   Renal function, secondary complications of disease

These studies may lead to new treatments and are adding insight into Cystagon etiology and treatment.

A major focus of Cystagon research is the development of new drugs and other treatment options. Studies seek to identify new drugs to treat various related disorders and to find safer, more effective doses for medications already being used. Other research is aimed at identifying receptors or drug targets.


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