Efficacy of Diroximel Fumarate in Reducing Perihaematomal Oedema in Patients with Spontaneous Intracerebral Haemorrhage: A Randomized, Double-Blind, Placebo-Controlled Trial

The primary objective is to evaluate the efficacy of diroximel fumarate in reducing perihaematoma oedema at day 8 compared to a placebo in patients with spontaneous intracerebral haemorrhage (Scope: 5). Secondary objectives include:

• Assessment of functional outcome at 6 months post-event (Scope: 5).
• Evaluation of the safety profile of diroximel fumarate (Scope: 4).

The sponsor did not provide information regarding the total number of participants. The study population consists of both male and female patients experiencing spontaneous intracerebral haemorrhage. Eligible participants are aged 18 years or older and must present with a first-ever or recurrent symptomatic supratentorial event confirmed via brain imaging. Inclusion requires the administration of the study treatment within 48 hours of symptom onset or the last time the individual was seen without a neurological deficit. The investigation aims to evaluate the efficacy of Diroximel Fumarate in reducing peri-haematoma oedema.

This phase 2b, multicenter, double-blind, placebo-controlled, parallel-group study is designed to evaluate the efficacy of diroximel fumarate in reducing perihaematomal oedema in patients with intracerebral haemorrhage. Participants will be assigned via stratified randomization to receive either the active treatment or a placebo. The clinical process begins with a screening visit to confirm eligibility based on criteria such as age and imaging-confirmed spontaneous haemorrhage. Following randomization, the primary efficacy endpoint is assessed via non-contrast CT scan at approximately day 8 to measure the absolute volume of oedema. The study includes follow-up procedures to evaluate functional outcome using the modified Rankin Scale and to monitor the rate of severe adverse events. The total duration of participant involvement extends to a six-month end-of-study visit.

The experimental treatment consists of diroximel fumarate administered as gastro-resistant hard capsules. The dosage is 924 mg provided via the oral route.

The control group receives a placebo designed to be identical to the active medication in visual appearance, taste, and form, but without the active substance.

The primary efficacy endpoint is the absolute volume of perihaematoma oedema (PHO), which is assessed at 8 ± 1 days using a non-contrast CT scan (NCCT). This assessment aims to evaluate the effect of diroximel fumarate in reducing edema in patients with intracerebral haemorrhage.

Secondary efficacy parameters include:

• Functional outcome, defined as global disability measured by the overall distribution of the modified Rankin Scale (mRS) score at 6 months, representing the end of follow-up.