Efficacy of Iloprost for the Management of Vaso-Occlusive Crises in Adults with Sickle Cell Disease: A Randomized, Double-Blind, Placebo-Controlled Study
- Trial ID
- 2025-522005-38-00
- Protocol
- 2020/0421/HP
Trial statistics
Diseases & Conditions
Objectives
The primary objective is to evaluate the efficacy of a maximum 5-day infusion of iloprost compared to a placebo regarding opioid consumption during hospitalization in adult patients experiencing a vaso-occlusive crisis due to sickle cell disease. This investigation is clinically relevant for managing acute pain and minimizing the requirement for analgesic medication during inpatient care.
Participants
The sponsor did not provide information regarding the total number of participants. The study population consists of adult patients, aged 18 years or older, diagnosed with sickle cell disease. Participants include both males and females who are experiencing a vaso-occlusive crisis requiring hospitalization and parenteral infusion of opioids. Eligible individuals are those admitted to an emergency, internal medicine, or hematology department for less than 36 hours. For women of childbearing age, specific requirements regarding contraception and negative pregnancy tests are mandatory. The population is selected based on the clinical necessity for inpatient management of acute pain crises.
Plans and Procedures
This multicentre, randomised, double-blind, placebo-controlled study is designed to evaluate the efficacy of iloprost compared to a placebo in the management of vaso-occlusive crisis in adult patients with sickle cell disease. Following a screening process to confirm eligibility, participants are assigned to receive either a five-day infusion of the test product or a glucose solution. The primary objective is to assess the mean opioid consumption in morphine equivalent mg/day over the 28 days following randomisation. Hospitalization for the crisis is limited to a maximum of 21 days. The overall duration of participant involvement is determined by the clinical course and the required follow-up period to monitor outcomes. Early termination from the study may occur based on clinical conditions or protocol deviations.
Treatment
The experimental treatment consists of iloprost, administered as a solution for infusion. The prescribed dosage is 50 µg via infusion.
The control group receives a placebo consisting of glucose monohydrate, provided as a 250 ml solution for infusion.
The study evaluates the efficacy of a maximum 5-day infusion for the management of vaso-occlusive crisis in adult patients with sickle cell disease. The primary endpoint is measured by opioid consumption during hospitalization.
Efficacy
The efficacy of iloprost is evaluated by comparing its impact against a placebo in the management of vaso-occlusive crisis in adult patients with sickle cell disease. The primary endpoint is the mean opioid consumption, expressed in morphine equivalent mg/day, measured over the 28 days following randomization.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Patients with sickle cell disease (all genotypes)
- Age ≥18 years.
- Requiring hospitalisation and parenteral infusion of opioids for the treatment of a vaso-occlusive crisis
- Admitted to the Adult Emergency Department or the Internal Medicine or Haematology Department for less than 36 hours.
- Patient having read and understood the information letter and signed the consent form or oral consent obtained and consent retrieved within 48 hours of randomisation
- For women: o Of childbearing age (defined by the CTCG as a fertile woman, after menarche and until menopause, except in cases of permanent infertility (including hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) o Using a highly effective method of contraception according to CTCG recommendations (combined hormonal contraception [containing estrogens and progestogens] associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing Synopsis PROSTASICKLE – Protocol Summary Version 1.1 – 02/02/2026 system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence) for at least 4 weeks prior to inclusion and throughout the entire duration of systemic exposure to the study treatment. AND;. And, o Presenting a negative urinary pregnancy test at inclusion; o menopausal:Menopause, according to CTCG recommendations, is defined as the absence of menstruation for 12 months without any other medical cause. Elevated follicle-stimulating hormone (FSH) levels in the postmenopausal interval can be used to confirm postmenopausal status in women who are not using hormonal contraception or hormone replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
- Affiliated to a social security scheme.
Exclusion Criteria
- Patients with a contraindication to iloprost (ILOPROST ZENTIVA 100 micrograms/mL, solution for dilution for infusion) o Pregnancy, breast-feeding. o Hypersensitivity to the active substance or to one of the excipients. o Conditions where the risk of bleeding may be increased due to the effects of iloprost on platelets. o Severe coronary heart disease or unstable angina. o Myocardial infarction within the previous six months. o Acute or chronic heart failure (NYHA classification II to IV). o Severe arrhythmias. o Suspected pulmonary congestion
- Active smoking
- Patients with liver failure or kidney failure requiring dialysis
- Patients who have had a cerebrovascular event in the last 3 months
- Person deprived of liberty by an administrative or judicial decision or person placed under court protection / sub-guardianship or guardianship.
- Patient already included in the PROSTASICKLE protocol within 90 days of randomisation
- Patient participating in another therapeutic trial or having participated in another trial within 1 months of inclusion
- Patient presenting with a contraindication to 5% glucose (Glucose 5% FRESENIUS KABI France solution for infusion): a. Hypersensitivity to corn b. Uncontrolled hyperglycemia, c. Decompensated diabetes, d. Other known glucose intolerances (e.g. situations of metabolic stress, acute shock states, collapse), e. Hyperosmolar coma, f. Hyperlactatemia, g. Metabolic acidosis, h. Fluid overload. The administration of high volumes may in particular result, due to fluid overload, in the following contraindications: i. Hyperhydration j. Acute heart failure k. Pulmonary edema
- Hypersensitivity to 5% glucose
- Severe malnutrition
- Thiamine deficiency in chronic alcoholic patients
- Patient presenting with arterial hypotension
- History of documented opioid dependence
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 France | Not Yet Recruiting | 01 Sept 2026 | 144 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
ILOPROST ZENTIVA 100 microgrammes/mL, solution à diluer pour perfusion | Test | SOLUTION À DILUER POUR PERFUSION | INFUSION | 50 | 5 | PRD9525474 |
Glucose 5 % Fresenius Kabi, solution pour perfusion | Placebo | SOLUTION POUR PERFUSION | INFUSION | 250 | 5 | PRD2016937 |