Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With C… (NCT01142154) | Clinical Trial Compass
CompletedPhase 1/2
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
United States5 participantsStarted 2010-06
Plain-language summary
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients. Also the pharmacokinetics of single and multiple orally dosed Prodarsan will be evaluated and compared to intravenous dose of Osmitrol in CS patients.
Who can participate
Age range2 Years – 10 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Parents or legal guardian(s) of the pediatric patient with CS must be willing and able to give written Informed Consent. Informed Assent will be offered to children who can understand and participate in the Informed Assent process.
* Diagnosis of CS confirmed by one of the following laboratory diagnostic test results:
* Demonstration by molecular diagnostic analyses of two mutations in either the ERCC6 gene or the ERCC8 gene, wherein both mutations are either known to be pathogenic or are obviously detrimental (including nonsense or frameshift mutations, mutations of "invariant" splice site consensus signals, or large deletions/rearrangements); OR
* A pattern of DNA repair responses in patient's cultured skin fibroblast cells indicative of a specific deficiency of transcription-coupled DNA nucleotide excision repair after irradiation with ultraviolet light, namely a significant deficiency of cellular survival (and/or "recovery of ribonucleic acid \[RNA\] synthesis," if that has been specifically measured) coupled with a normal test for "unscheduled DNA synthesis" OR
* Decreased cell survival and/or "recovery of RNA synthesis" in UV-irradiated patient's skin fibroblast cultures and rescue of these parameters by fusion to reference cell lines with known NER defects (functional complementation analysis) OR
* Quantitative RT-PCR to quantify mRNA levels of CS-A and CS-B transcripts.
* Weight inclusive of 10 kg to 25 kg.
* Male or female, inclusive o…
What they're measuring
1
Evaluate and compare the pharmacokinetics of D-mannitol following a single IV dose of Osmitrol to single and multiple oral doses of Prodarsan in pediatric patients with Cockayne Syndrome