Evaluation of the efficacy of iloprost in the management of vaso-occlusive crises in adult patients with sickle cell: a multicentre, randomised, double-blind, placebo-controlled study _ PROSTASICKLE

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire Rouen

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Decision date (initial)

2026-03-09

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

Evaluate the efficacy of a maximum 5-day infusion of iloprost compared to placebo on opioid consumption during hospitalization (with a maximum hospitalization duration of 21 days) for a vaso-occlusive crisis in adult patients with sickle cell disease.

Conditions and MedDRA coding

sickle cell anaemia with vaso-occlusive crisis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Patients with sickle cell disease (all genotypes)
2. Age ≥18 years.
3. Requiring hospitalisation and parenteral infusion of opioids for the treatment of a vaso-occlusive crisis
4. Admitted to the Adult Emergency Department or the Internal Medicine or Haematology Department for less than 36 hours.
5. 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
6. 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.
7. Affiliated to a social security scheme.

Exclusion criteria 13

1. 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
2. Active smoking
3. Patients with liver failure or kidney failure requiring dialysis
4. Patients who have had a cerebrovascular event in the last 3 months
5. Person deprived of liberty by an administrative or judicial decision or person placed under court protection / sub-guardianship or guardianship.
6. Patient already included in the PROSTASICKLE protocol within 90 days of randomisation
7. Patient participating in another therapeutic trial or having participated in another trial within 1 months of inclusion
8. 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
9. Hypersensitivity to 5% glucose
10. Severe malnutrition
11. Thiamine deficiency in chronic alcoholic patients
12. Patient presenting with arterial hypotension
13. History of documented opioid dependence

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean opioid consumption in morphine equivalent mg/day over the 28 days following randomization

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire Rouen

Sponsor organisation

Centre Hospitalier Universitaire Rouen

Address

1 Rue De Germont, Bp 96031 Bp 96031

City

Rouen Cedex

Postcode

76031

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire Rouen

Contact name

Delphine PICOCHE

Public contact point

Organisation

Centre Hospitalier Universitaire Rouen

Contact name

Delphine PICOCHE

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 15 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications