A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to Investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects With Sickle-Cell Disease (SCD).
Overview
- Phase
- Phase 2
- Intervention
- Placebo
- Conditions
- Sickle-Cell Disease
- Sponsor
- Modus Therapeutics AB
- Enrollment
- 147
- Locations
- 20
- Primary Endpoint
- Time to resolution of VOC
- Status
- Completed
- Last Updated
- 6 years ago
Overview
Brief Summary
A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease (SCD).
Detailed Description
This will be a phase II, multi-centre, randomized, double-blind, placebo-controlled study designed to assess preliminary efficacy, safety and pharmacokinetics (PK) of 2-7 days continuous IV administration of sevuparin for the management of acute VOC in subjects with SCD. Adults and adolescents ≥ 12 years of age will be randomized to treatment with sevuparin or placebo (ratio 1:1).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Sign a written informed consent (adults, parents) and assent (adolescents)
- •Male or female, age 12-50 years.
- •Diagnosis of Sickle cell disease
- •Subjects admitted for an acute, painful VOC to be treated/or treated with parenteral opioid analgesia.
- •Expectancy of need for hospitalization during at least 48 hours.
- •Be at least 1 year postmenopausal, surgically sterile, or if Women of Child Bearing Potential (WOCBP), e.g. following menarche practicing an effective method of birth control
Exclusion Criteria
- •Severe hepatic failure/disease, abnormal liver enzyme tests or history of hepatitis B virus (HBV), hepatitis C virus (HCV)
- •Abnormal conjugated (direct) bilirubin 3 fold above ULN
- •History of clinically significant bleeding in vital organs
- •Current clinically significant bleeding, as judged by the investigator
- •Current use of acetylsalicylic acid (ASA), anti-platelet therapy, anticoagulant therapy
- •Abnormal coagulation laboratory values
- •A platelet count \<75,000/µL.
- •Subjects with more than 5 hospitalizations for VOC during the last 6 months
- •Evidence of acute SCD complications other than VOC at screening
- •The use of strong opioids for \> 3 consecutive days during the last 15 days before presenting to the hospital
Arms & Interventions
Placebo
Placebo infusion
Intervention: Placebo
Sevuparin
Sevuparin infusion
Intervention: Sevuparin
Outcomes
Primary Outcomes
Time to resolution of VOC
Time Frame: From hospitalisation until discharge, defined as freedom from parenteral opioid use and readiness for discharge i.e. from randomisation until day 7
Time from start of infusion until resolution of VOC crisis/episode
Secondary Outcomes
- Cumulative dose of parenteral opioids(From baseline (visit 1) until day 3-7)
- Duration of severest pain,(From baseline (visit 1) until day 3-7)
- Frequency and pattern of treatment-emergent adverse event (TEAEs)(Time from start randomsiation until end of study, approximately 1 month 1 week after randomisation)
- Pharmacokinetic (PK) characteristics of sevuparin(Pre dose, 1h, 2h, 24h, 1/day (day 3-8))
- Mean change in pain intensity(From baseline (visit 1) until day 3-7)