Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD

NCT02515838 | Completed Phase 2 DMC

• 1 other identifier: TVOC01
• Study Type: interventional
• Target: 147
• Locations: 7 countries
• Sites: 20

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).

Conditions

Sickle-Cell Disease

Outcome Measures

Primary Outcomes (1)

• Time to resolution of VOC

  Time from start of infusion until resolution of VOC crisis/episode

  From hospitalisation until discharge, defined as freedom from parenteral opioid use and readiness for discharge i.e. from randomisation until day 7

Secondary Outcomes (5)

• 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

• Duration of severest pain,

  From baseline (visit 1) until day 3-7

• Cumulative dose of parenteral opioids

  From baseline (visit 1) until day 3-7

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo infusion

Other: Placebo

Sevuparin

EXPERIMENTAL

Sevuparin infusion

Drug: Sevuparin

Interventions

Sevuparin DRUG

The Drug Product sevuparin solution for IV infusion

Sevuparin

Placebo OTHER

Placebo for IV infusion

Placebo

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.
• BMI \>35
• 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
• History of chronic drug abuse.
• Renal dysfunction
• Known infection (positivity) with human immunodeficiency virus (HIV), HBV or HCV.
• Significant ECG abnormality

Sponsors & Collaborators

• Modus Therapeutics AB: lead
• Ergomed: collaborator

Study Sites (20)

Salmaniya Hospital, Kingdom of Bahrain

Manama, Bahrain

Location

Salmaniya Medical Complex, Bahrain

Manama, Bahrain

Location

Annotto Bay Hospital

Annotto Bay, Jamaica

Location

Kingston Public Hospital

Kingston, Jamaica

Location

University Hospital of the West Indies

Kingston, Jamaica

Location

Winchester Surgical and Medical Institute

Kingston, Jamaica

Location

Mandeville Regional Hospital

Mandeville, Jamaica

Location

May Pen Public Hospital Clarendon

May Pen, Jamaica

Location

Cornwall Regional Hospital, Jamaica

Montego Bay, Jamaica

Location

American University of Beirut Medical Center, Beirut, Cairo street, Beirut, Lebanon

Beirut, Lebanon

Location

Nini Hospital

Tripoli, Lebanon

Location

Dept of Haematology

Amsterdam, Netherlands

Location

Erasmus MC

Rotterdam, Netherlands

Location

Sultan Qaboos University Hospital Alkhodh, Oman

Muscat, Oman

Location

Sultan Qaboos University

Muscat, Oman

Location

King Fahd Medical City, As Sulimaniyah, Riyadh Saudiarabien

Riyadh, Saudi Arabia

Location

King Saud University, Riyadh, Saudiarabien

Riyadh, Saudi Arabia

Location

Mersin University Faculty of Medicine

Adana, Mersin, Turkey (Türkiye)

Location

Cukurova University Faculty Of Medicine Tıp Fakültesi

Adana, Turkey (Türkiye)

Location

Dr Antmen

Adana, Turkey (Türkiye)

Location

Related Publications (1)

• Biemond BJ, Tombak A, Kilinc Y, Al-Khabori M, Abboud M, Nafea M, Inati A, Wali Y, Kristensen J, Kowalski J, Donnelly E, Ohd J; TVOC01 Investigators Group. Sevuparin for the treatment of acute pain crisis in patients with sickle cell disease: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2021 May;8(5):e334-e343. doi: 10.1016/S2352-3026(21)00053-3.

  PMID: 33894169 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

sevuparin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Dr Bart J Biemond, MD, PhD

  Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2015
• First Posted: August 5, 2015
• Study Start: July 1, 2015
• Primary Completion: May 1, 2019
• Study Completion: May 1, 2019
• Last Updated: July 16, 2019

Record last verified: 2019-02

Locations

OM (2); SA (2); LE (2); JA (7); NL (2); TU (3); BA (2)