Trials

Product	Study type	Status	Study title	clinicaltrials.gov identifier
Lomitapide	Phase 3 study	Planned	Phase III, Single-arm, Open-label, International, Multi-centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients with Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid-lowering Therapy
Lomitapide	Phase 3 study	Recruiting	Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)	NCT04681170
Metreleptin	Phase 3 study	Planned	A 12-Month Randomised, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Patients with Partial Lipodystrophy
Metreleptin	Phase 4 study	Recruiting	Immunogenicity of Metreleptin in Patients With Generalised Lipodystrophy	NCT04026178
Oleogel-S10	Phase 3 study	Active, not recruiting	Phase 3 Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa (EASE)	NCT03068780
Lomitapide	Registry	Recruiting	LOWER: Lomitapide Observational Worldwide Evaluation Registry	NCT02135705
Metreleptin	Registry	Recruiting	MEASuRE: Metreleptin Effectiveness And Safety Registry	NCT02325674

Registries

LOWER

The 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 Registry is currently enrolling in US, Europe, UK, Canada and Argentina. The Registry will enrol at least 300 adult HoFH patients who will be followed for a minimum of 10 years, in the EU and UK patients will be enrolled and followed indefinitely. The inclusion criteria include patients who commenced therapy with lomitapide. The exclusion criteria include those receiving lomitapide in a clinical trial or receiving an investigational agent.

Registry highlights

LOWER is a global observational registry that will enrol patients treated with lomitapide where LOWER is a regulatory requirement
All physicians in countries participating in LOWER who treat patients with lomitapide are eligible to participate.
LOWER is non-interventional with no study medication provided.
There are no protocol-mandated procedures or diagnostic tests.
All treatment decisions will be made at the discretion of the patient’s healthcare professional.
Electronic data collection for LOWER is designed to minimize the burden on participating physicians and site personnel.
Investigators will be reimbursed at fair market value for costs associated with completing registry requirements.

For more information on enrolling in the LOWER Registry, please contact medical information, [email protected]

MEASuRE

The global MEASuRE registry has been established to further evaluate the long-term safety and effectiveness of metreleptin in patients with generalised or partial lipodystrophy treated with metreleptin. 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.

The Registry will enrol at least 100 patients who will be followed for a minimum of 10 years, in the EU and UK patients will be enrolled and followed indefinitely.

Registry highlights

MEASuRE is an observational registry that will enrol patients treated with metreleptin
All physicians who treat patients with metreleptin where MEASuRE is a regulatory requirement are eligible to participate.
MEASuRE is non-interventional with no study medication provided for the purpose of the study.
There are no protocol-mandated procedures or diagnostic tests.
All treatment decisions will be made at the discretion of the patient’s healthcare professional.
Electronic data collection for MEASuRE is designed to minimise the burden on participating physicians and site personnel.

For more information on enrolling in the MEASuRE Registry, please contact medical information, [email protected]

The MACRO Registry

The MACRO Registry is the first ever acromegaly disease state registry in the United States.

For more information on enrolling in the MACRO registry please contact Medical Information at [email protected].