Trial record 2 of 3 for:
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
This study has been completed.
Information provided by:
First received: May 20, 2010
Last updated: June 22, 2011
Last verified: June 2011
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.
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
||A Phase I/II Crossover Study To Evaluate and Compare the Pharmacokinetics of a Single IV Dose of D-Mannitol (Osmitrol®10%) to Single and Multiple, Escalating Doses of Liquid, Oral Prodarsan™ in Patients With Cockayne Syndrome
Primary Outcome Measures:
- 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 [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Evaluate the safety and tolerability of administering oral Prodarsan in CS patients over a six (6) to eight (8) day period, including dose escalation to reach a Target Dose [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||September 2010 (Final data collection date for primary outcome measure)
Experimental: oral, liquid solution
Prodarsan TID, oral solution, 6-8 days
|Ages Eligible for Study:
||2 Years to 10 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Any concurrent illness (other than related to CS), disability or clinically significant abnormality, including laboratory tests, that may affect the interpretation of the PK or safety data or prevent the patient from safely completing the assessments required by the protocol as judged by the investigator. Such conditions include, but are not limited to:
- Ascites or generalized edema.
- Nephrotic syndrome or history of abnormal kidney function.
- Clinically significant thyrotoxicosis.
- Known history of hyperprolinemia.
- Clinically significant dehydration as judged by the investigator
- Severely compromised venous access.
- Presence of an external ventricular, abdominal, or chest drain.
- Subjects due to receive radioiodine therapy, two (2) weeks before or two (2) weeks following the study period.
- Participation in another PK or treatment clinical study within thirty (30) days prior to signing and dating of Informed Consent/Assent Form for this study.
As judged by the investigator, clinical features present at the time of initial screening, that are associated with the terminal phases of the natural progression of CS, indicating that safe travel and completion of the study and its assessments are unlikely, including any of the following:
- Continuous or intermittent dependence on supplemental oxygen at home during the six (6) months prior to enrollment in this study; OR
- Two or more hospitalizations due to pneumonia, during the twelve (12) months prior to enrollment in this study; OR
- A documented, net weight loss of at least 10%, which has not been recovered, and which includes a significant net weight loss (beyond the estimated error of the measurement) over the most recent 6 months despite intensive nutritional support including the use of gastrostomy tube feedings.
- Known hypersensitivity to any of the components found in Prodarsan, D-mannitol, iohexol or iodine compounds.
- History of clinically significant drug sensitivity or allergic reaction such as anaphylaxis.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01142154
|Children's Hospital Boston
|Boston, Massachusetts, United States, 02115 |
||Edward Neilan, MD
||Children's Hospital Boston
No publications provided
||Lia Dam/Director Clinical Operations, DNage
History of Changes
|Other Study ID Numbers:
|Study First Received:
||May 20, 2010
||June 22, 2011
||United States: Food and Drug Administration
Keywords provided by DNage B.V.:
Genetic disease, inborn
DNA Repair-Deficiency Disorders
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on June 18, 2013
Bone Diseases, Developmental
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders