Ongoing Clinical Trials for Sickle Cell Anaemia With Crisis
There are currently 4 clinical trials underway studying treatments for sickle cell disease during painful crises. These studies are investigating new medications and approaches to manage vaso-occlusive episodes, which are sudden, painful events that occur when sickle-shaped red blood cells block blood flow in small vessels. The trials are taking place across several European countries and involve both children and adults with this genetic blood disorder.
Clinical trial locations
- Belgium
- France
- Study of Intranasal Sufentanil and Intravenous Morphine for Severe Sickle Cell Pain Crisis in Children
- Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
- Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
- Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients
- Germany
- Greece
- Italy
- Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
- Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
- Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients
- Netherlands
- Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
- Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
- Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients
- Spain
- Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
- Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
- Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients
Study of Intranasal Sufentanil and Intravenous Morphine for Severe Sickle Cell Pain Crisis in Children
This trial focuses on managing severe pain crises in children with sickle cell disease. The study is testing whether sufentanil, a pain medication given through a nasal spray, combined with the standard treatment of intravenous morphine, can provide better and faster pain relief compared to morphine alone.
Main inclusion criteria: Children under 18 years old who weigh more than 10 kg and have a confirmed diagnosis of sickle cell disease, including types HbSS, HbSC, or Sβ-thalassemia. Participants must be experiencing a vaso-occlusive crisis with severe pain, defined as a score of 10 or more out of 15 on the EVENDOL scale for younger children, or 7 or more out of 10 on the pain rating scale for children aged 8 to 17 years. Parental consent is required.
Main exclusion criteria: Children who are not experiencing a vaso-occlusive crisis, who fall outside the specified age range, or who cannot safely receive the study medications. Children with medical conditions that would make participation unsafe are also excluded.
Trial focus: The study evaluates how quickly and effectively the combination of intranasal sufentanil and intravenous morphine relieves severe pain compared to a placebo spray followed by morphine. Pain levels are measured at multiple time points starting from 30 minutes after the nasal spray is given. The trial also monitors side effects such as low blood pressure, breathing difficulties, and nausea.
Investigational drugs: Sufentanil is administered through the nose to provide rapid pain relief. Morphine is given intravenously as the standard pain management treatment. Both medications belong to the opioid class of pain relievers.
Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises
This international trial is evaluating CSL889, a form of human hemopexin protein, for treating vaso-occlusive crises in adolescents and adults with sickle cell disease. The medication is given through an intravenous infusion directly into the bloodstream.
Main inclusion criteria: Participants must be at least 12 years old, with approval required for adolescents aged 12 to 17. They must have a confirmed diagnosis of sickle cell disease and be experiencing a new acute vaso-occlusive crisis requiring treatment with strong pain medications given by injection.
Main exclusion criteria: People without sickle cell disease, those not experiencing a vaso-occlusive crisis, participants outside the specified age range, and those unable or unwilling to receive intravenous medication are excluded. Pregnant or breastfeeding women and those with interfering medical conditions or recent participation in other clinical trials may also be excluded.
Trial focus: The study assesses both the safety and effectiveness of CSL889 in helping vaso-occlusive crises resolve more quickly. Participants receive either CSL889 or a placebo, and researchers monitor how long it takes for painful episodes to resolve, track any side effects, and measure drug levels in the blood over time.
Investigational drugs: CSL889 is a human hemopexin protein that may help manage painful episodes by controlling levels of free heme in the blood, which can become elevated during a crisis.
Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients
This trial examines whether crovalimab can prevent painful vaso-occlusive episodes in people with sickle cell disease. Unlike the other studies that treat crises when they occur, this one focuses on prevention.
Main inclusion criteria: Participants must weigh at least 40 kg and have a confirmed diagnosis of HbSS or HbSβ0 sickle cell disease. They must have experienced between 2 to 10 documented vaso-occlusive episodes in the past year. If taking other treatments for sickle cell disease, the dose must have been stable for at least 3 months. Required vaccinations against specific infections must be up to date, and adequate liver and kidney function is necessary.
Main exclusion criteria: People without sickle cell disease, those outside the specified age range, and those not meeting other specific study requirements are excluded.
Trial focus: The study compares crovalimab to a placebo over time, monitoring how often participants experience vaso-occlusive episodes both at home and in medical facilities. Researchers will track changes in blood tests, the time until the first episode occurs after starting treatment, and overall health indicators through week 49 of the study.
Investigational drugs: Crovalimab is a monoclonal antibody that targets a specific protein in the immune system believed to contribute to blood vessel blockages. It can be given either intravenously or under the skin.
Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients
This companion study to the prevention trial focuses on using crovalimab to treat acute vaso-occlusive episodes when they occur, rather than preventing them.
Main inclusion criteria: Participants must weigh at least 40 kg and have confirmed HbSS or HbSβ0 sickle cell disease. They must be admitted to a hospital due to an acute uncomplicated vaso-occlusive episode requiring treatment with injected opioid pain relievers. Required vaccinations must be current, and adequate liver and kidney function is necessary. If receiving other sickle cell therapies, the dose must have been stable for at least 28 days.
Main exclusion criteria: People without sickle cell disease, those not experiencing a vaso-occlusive episode requiring hospitalization, and those with conditions that would make participation unsafe are excluded.
Trial focus: The study evaluates the safety of crovalimab compared to a placebo during acute episodes. Researchers monitor side effects, vital signs, infusion reactions, and measure drug levels in the blood over time. They also assess how quickly patients experience improvement from their episodes and check for any antibodies that might develop against the medication.
Investigational drugs: Crovalimab is a monoclonal antibody administered by injection, either intravenously or under the skin. It works by blocking a specific immune system protein that may contribute to vaso-occlusive episodes.
Summary
The four ongoing clinical trials for sickle cell anaemia with crisis represent different approaches to managing this painful condition. One trial focuses specifically on children and tests rapid pain relief methods using nasal spray medication. The other three studies involve adolescents and adults, with one testing a novel protein therapy and two examining a monoclonal antibody for both prevention and acute treatment of crises.
France participates in all four trials, making it the most active country in this research area. Italy, Spain, and the Netherlands are involved in three trials each, while Belgium, Germany, and Greece participate in one trial each. This broad geographic distribution across Europe suggests a coordinated effort to develop better treatments for this genetic blood disorder.
Two different therapeutic approaches are being tested: CSL889, a human hemopexin protein therapy, and crovalimab, a monoclonal antibody. The trials examining crovalimab are notable for investigating both prevention of episodes and treatment of acute crises, which could provide comprehensive management options if successful. All trials emphasize safety monitoring alongside effectiveness, reflecting the careful approach needed when testing new treatments for a vulnerable patient population.
