A Study to Explore Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease (SCD)
- Conditions
- Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell DiseaseMedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2014-004416-11-BE
- Lead Sponsor
- Modus Therapeutics AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 133
1. Sign a written informed consent (adults, parents) and assent (adolescents).
2. Male or female, age 12-50 years.
3. Diagnosis of Sickle cell disease, types HbSS, HbSC, Hb O Arab, HbSß0-thalassemia or HbSß+-thalassemia (SCD type to be confirmed by liquid chromatography or other method of comparable reliability during the study, if confirmation is not available at time of inclusion)
4. Subjects admitted for an acute, painful VOC to be treated/or treated with parenteral opioid analgesia at the time of admission. VOC is defined as an episode of pain that led to a clinic or emergency department visit, and cannot be explained except by SCD. Please note: Study treatment should start as soon as possible and at latest within 24 hours from the time of the decision to hospitalize the subject.
5. Expectancy of need for hospitalization during at least 48 hours.
6. Be at least 1 year postmenopausal, surgically sterile, or if WOCBP, e.g. following menarche practicing an effective method of birth control (e.g. oral contraception, intrauterine device, or diaphragm with spermicide; or double barrier method) during study drug administration and one month following treatment completion.
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 93
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Severe hepatic failure/disease or liver enzyme tests (AST and ALT) above 2 times the upper limit of normal (ULN) range, or clinically significant impairment of liver function due to HBV, HCV or other liver diseases.
2. Conjugated (direct) bilirubin 3 fold above ULN.
3. History of clinically significant bleeding in vital organs (not due to relevant trauma), or pathological bleeding.
4. Current clinically significant bleeding, as judged by the investigator.
5. Current use of ASA, anti-platelet therapy, anticoagulant therapy and prophylactic and therapeutic LMWH or un-fractioned heparin.
6. APTT above normal range , and INR above 1.4.
7. A platelet count < 75,000/µL.
8. BMI >35
9. Subjects with more than 5 hospitalizations for VOC during the last 6 months (to exclude subjects with exacerbations of chronic pain rather than true vaso-occlusion).
10. Evidence of acute SCD complications other than VOC at screening (CVA, ACS, multi-organ failure).
11. The use of strong opioids for > 3 consecutive days during the last 15 days before presenting to hospital.
12. History of chronic drug abuse.
13. Renal dysfunction (GFR< 60 ml/min), calculated per Cockcroft-Gault formula
14. Known infection with HIV, and active infection with HBV or HCV.
15. Significant ECG abnormality including QTcf > 450 msec
16. History of a clinically significant drug allergy to heparin, LMWH’s or sevuparin.
17. Use of any investigational agent during the 30 days prior to the first dose.
18. For females: pregnancy, lactating or intention of becoming pregnant within the next 40 days.
19. Evidence of clinically significant disorders that might interfere with the study aim or safety of the subject, as judged by the Investigator: e.g. neurological, psychiatric (depression, psychosis or schizophrenia), cardiovascular (including arrhythmia), pulmonary, metabolic, gastrointestinal, endocrine diseases, coagulation or malignancies.
20. Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method