Sickle cell disease MedDRA version: 9.1 Level: LLT Classification code 10040644 Term: Sickle cell disease
Conditions
Interventions
Trade Name: Pentosan Polysulfate SP 54
Pharmaceutical Form: Tablet
INN or Proposed INN: pentosan polysulfate sodium
CAS Number: 8000044895
Current Sponsor code: TRF - 1101
Concentration unit: mg milli
Sponsors
TRF Pharma, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Be 18 years of age or older at the time of informed consent; 2. Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta thalassemia; 3. Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life’s activities [i.e., school, work, planned leisure activity] because of pain); 4. If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of misepristone is not allowed); 5. Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (EC) approved Informed Consent Form prior to entering the clinical investigation. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation; 2. Be currently taking anticoagulant or thrombolytic medication; 3. Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®); 4. Have a known sensitivity or allergy to heparin or PPS; 5. Have a history of thrombocytopenia (platelet count 15% from prior transfusion; 10. Creatinine levels > 1.53 mg/dL (135 micromol/L); 11. ALT levels = 3 times upper limit of normal; 12. Platelet count 2.2; 14. Be unable to tolerate oral medications; 15. Have unreliable venous access; 16. Be noncompliant with regular care; 17. Have a positive pregnancy test, be currently lactating, or be trying to become pregnant; 18. Have participation in an investigational drug or medical device study within previous 30 days; 19. Have any other condition or circumstance that in the opinion of the Investigator makes the patient a poor candidate for participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of pentosan polysulfate sodium administered daily for three months to patients with sickle cell disease;Secondary Objective: 1) To assess the pharmacodynamic activity of pentosan polysulfate sodium 2) To assess activity of pentosan polysulfate sodium on vascular endothelial injury and vasoocclusive pain associated with sickle cell disease. ;Primary end point(s): There are five outcomes to be measured in this study. 1) Changes in blood flow as measured by laser Doppler technology. Blood flow in the microcirculation will be measured in three places on the subject’s forearm. It is anticipated that a combination of the four measurements will be used in the calculation of the statistics to be used for this outcome. The Statistical Analysis Plan will define this outcome in detail. It is also possible to analyse this outcome as a multi-level clustered model with clustering at the forearm level (four measurements at different locations) and over time (measurements at multiple visits) to control for the inherent correlation within the forearm and within the same subjects measured repeatedly over time. 2) Change in circulating markers of blood vessel injury and inflammation: The change (as defined for outcome above) for each marker will be determined for use in descriptive analysis and in comparison of changes between treatment groups. 3) Change in frequency of pain: The change in frequency of pain will be calculated as a median with a non-parametric comparison of frequency between treatment groups due to the inherently non-normal distribution of pain scores. 4) Physician’s global assessment of response to treatment: This assessment would occur at every visit and would consist of a single reading ranging from -3 to +3. The change in this assessment would be calculated as the change from baseline represented in a multi-dimensional contingency table. 5) Change in the level of P-selectin blocking activity: The change in | — |
Countries
France, United Kingdom
Outcome results
None listed