A randomised, double-blind, placebo-controlled study to assess the safety and activity of pentosan polysulfate sodium on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain in patients with sickle cell disease - A Study of PPS in Sickle Cell Disease

Phase 1

• Conditions: Sickle cell disease; MedDRA version: 9.1Level: LLTClassification code 10040644Term: Sickle cell disease

• Registration Number: EUCTR2008-003573-41-FR
• Lead Sponsor: TRF Pharma, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2008-07-28
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 100

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

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 < 100 x 103/mm3) induced by heparin or PPS;
6. Have had fewer than 2 documented pain crises in the past year;
7. Have had a pain crisis within one month of screening or randomization;
8. If currently on or recently discontinued hydroxyurea treatment, have initiated or discontinued treatment or changed regimen within the past 6 months;
9. Have had a transfusion within last 120 days or have HbA% > 15% from prior transfusion;
10. Creatinine levels > 2 mg/dL (176.8 ?mol/L);
11. ALT levels = 5 times normal;
12. Platelet count < 100 x 103/mm3;
13. INR > 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified