Amryt Pharma is currently recruiting patients into its Phase III global clinical trial, EASE, to assess the efficacy and safety of Oleogel-S10 (AP101) as a topical treatment for Epidermolysis Bullosa (EB).The study will enrol 230 patients with either recessive or dominant dystrophic EB (RDEB, DDEB), Junctional EB or Kindler syndrome. The study is not enrolling patients with EB Simplex (EBS). This is the largest global phase III study in EB to date.
Patients apply Oleogel-S10 or placebo to an eligible target wound and all other EB wounds according to preferred schedule of dressing changes (every 1-4 days with own choice of non-adhesive dressing) to achieve target wound closure.
After 3 months of double-blind treatment period, all patients can receive Oleogel-S10 in an open label, 24-month safety extension.
A pre-specified interim efficacy analysis of the EASE study occurred in January 2019 after 50% of study patients were enrolled and reached the primary endpoint of wound healing assessment at Day 45 of the study. The purpose of this analysis was to determine that a sufficient number of subjects were enrolled in the study to adequately detect a difference in the efficacy on wound healing of Oleogel-S10 compared with placebo with a power of 80%. The recommendation of an independent data monitoring committee (IDMC) was to increase in the number of evaluable subjects in the study from 182 to a total of 230.
At the end of February 2019, a prespecified interim safety analysis was conducted. The Independent Data Monitoring Committee’s (IDMC) analysis was conducted using pharmacokinetic (“PK”) data received from patients already enrolled in the trial (aged four years and older). The IDMC interim safety analysis has recommended that sites can now include enrolment of infants and children with EB between the ages of 21 days to 4 years of age into the trial.
If you would like more information regarding the EASE study or wish to consider participating in it, please contact us at: EASEstudy@amrytpharma.com. You can also find details on the EASE study at the following website: clinicaltrials.gov.
In addition to the real-world evidence, there is data from the global Lomitapide Observational Worldwide Evaluation Registry (LOWER), a post-marketing requirement to study the long-term safety, effectiveness and patterns of use in patients treated with lomitapide globally. The occurrence and outcomes of pregnancies is evaluated through a separate pregnancy-exposure registry [PER].
The registry is non-interventional, with no provided study medications and no required procedures. The Registry will enrol at least 300 adult HoFH patients who will be followed for a minimum of 10 years, in the EU patients will be followed indefinitely. The inclusion criteria include patients at least 18 years old and who commenced therapy with lomitapide. The exclusion criteria include those receiving lomitapide in a clinical trial or receiving an investigational agent. The Registry is currently enrolling in US, Europe and Canada.
In order to further evaluate the long-term safety and effectiveness of metreleptin under normal conditions of clinical practice, the MEASURE registry has been established to enrol all patients with generalised or partial lipodystrophy treated with metreleptin according to an agreed protocol. The registry was initially established following approval of metreleptin in the United States to treat patients with generalised lipodystrophy and has now been expanded following approval in the EU to include metreleptin treated patients with both generalised and partial lipodystrophy.
Metreleptin pre-clinical and clinical development programs revealed known and potential serious risks associated with its use including serious adverse sequelae due to the development of neutralizing antibodies that could result in the loss of endogenous leptin activity causing severe infections or loss of efficacy, autoimmune disorders, hypersensitivity reactions, pancreatitis, hepatic adverse events, and hypoglycemia. The goal of the registry study is to gather additional data to better assess risks related to the long-term use of the drug by collection of clinical and laboratory data from additional person-years of exposure.