In short
Clinical trials are investigating ILOPROST in severe frostbite and in sickle cell disease with vaso-occlusive crisis. These studies are looking at how well it works, how safe it is in these settings, and whether it can improve outcomes such as amputation rates or opioid use.
Key points
- Current clinical trials are studying ILOPROST in two serious conditions: severe frostbite and vaso-occlusive crisis in adult patients with sickle cell disease. Both studies are Phase 3 and are authorised, which means they are advanced trials that are ready to test outcomes in patients. The frostbite trial compares ILOPROST with tPA and looks mainly at amputation rates and longer-term function. The sickle cell trial compares ILOPROST with placebo and measures opioid use over 28 days after randomization. Together, these trials focus on whether ILOPROST can improve important patient outcomes in different blood-flow related emergencies.
Clinical trial overview
These studies are looking at ILOPROST in two different emergency conditions: severe frostbite and sickle cell disease with vaso-occlusive crisis. Both are Phase 3 interventional trials, which means the treatment is being tested in people to see how well it works in real clinical care settings.
Both studies are authorised and use a comparison group, so the results can help show whether ILOPROST changes important patient outcomes compared with another treatment or with placebo.
Frostbite treatment study
The first trial is titled Frostbite Treatment Study comparing tPA and iloprost therapy and includes severe frostbite injury to the extremities. It plans to enroll 100 participants and is designed as a randomized study, meaning patients are assigned by chance to one of the study groups.
This study compares ILOPROST with ALTEPLASE, also called tPA, and it uses a non-inferiority design. Non-inferiority means the study is trying to show that ILOPROST is not worse than the comparison treatment by more than a small, pre-set amount.
The main outcome is the level of distal extremity or digital amputation, which means whether fingers or toes, or the farthest parts of the arms or legs, need to be removed because of damage from frostbite. The study also plans to collect long-term functional outcome data and blood-related laboratory values before and after treatment.
Sickle cell vaso-occlusive crisis study
The second trial is called Evaluation of the efficacy of iloprost in the management of vaso-occlusive crises in adult patients with sickle cell, and it is also known as PROSTASICKLE. It includes adults with sickle cell anaemia who are having a vaso-occlusive crisis, which is a painful episode caused by blocked blood flow in small vessels.
This study is multicentre, randomized, double-blind, and placebo-controlled. Multicentre means it is being done in more than one hospital or study site, and placebo-controlled means one group receives a comparison infusion without the active treatment.
The trial plans to enroll 144 participants and will test a maximum 5-day infusion of ILOPROST against placebo during hospitalization, with a maximum hospital stay of 21 days. The main question is whether ILOPROST can reduce opioid consumption, measured as morphine equivalent milligrams per day over the 28 days after randomization.
Main outcomes being measured
In clinical trials, an endpoint is the main result the researchers want to measure. Endpoints help show whether the treatment makes a real difference for patients.
- Frostbite trial endpoint: the level of distal extremity or digital amputation, with extra follow-up on long-term function.
- Sickle cell trial endpoint: mean opioid use in morphine equivalent mg/day during the 28 days after randomization.
These outcomes focus on what matters most to patients: avoiding tissue loss in frostbite and reducing pain medicine needs during sickle cell crisis.
What patients should know about participation
These studies do not include all people with frostbite or sickle cell disease; they focus on specific groups that match the trial rules. The frostbite study is for severe cases, while the sickle cell study is for adults with sickle cell anaemia and a vaso-occlusive crisis.
Because the studies are Phase 3, they are meant to provide stronger evidence about how ILOPROST performs in real patients. The trial data also show that the researchers are collecting clinical outcomes and, in the frostbite study, blood test values before and after treatment.