NCT03632213

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Hospital de Clinicas de Porto Alegre · Academic / Other
• Sex: All
• Age: 10 Years – 40 Years
• Healthy volunteers: Not accepted

Summary

Mucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. For some of the cardiovascular manifestations, such as aortic root dilation and valve diseases, there is no effective treatment currently available. Losartan, on the other hand, has been shown to be an effective drug for dilation of the aortic root, at least in animal models. This study aims to evaluate the safety and efficacy of losartan in patients with MPS VI and other mucopolysaccharidoses.

Detailed description

Mucopolysaccharidoses (MPS) are a group of lysosomal diseases characterized by deficiency of enzymes responsible for the degradation of glycosaminoglycans. MPS are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. Enzyme replacement therapy and bone marrow transplantation, despite being well established treatments, are not yet capable of reversing or preventing the progression of some of the cardiological manifestations of MPS. On the other hand, these patients may benefit from other conventional drug or surgical treatment, which can be instituted at an appropriate time if there is a better understanding of how these manifestations progress. In particular, the occurrence of aortic root dilation, although described in animal models, has only recently been evaluated in the studies on mucopolysaccharidoses. In addition, verifying the effectiveness of losartan in controlling these manifestations in the animal model opens the perspective of clinical use of this drug. Losartan is a low-cost drug and, if its efficacy is demonstrated, may represent an accessible therapy directed at the unmet needs of these patients.

Conditions

• Mucopolysaccharidosis IV A
• Mucopolysaccharidosis VI
• Mucopolysaccharidoses
• MPS IV A
• MPS VI
• MPS - Mucopolysaccharidosis
• Morquio A Syndrome
• Morquio Syndrome A
• Morquio Syndrome

Interventions

Type	Name	Description
DRUG	Losartan	Losartan group: 15 patients, both sexes, will receive Losartan 0.4 to 1.4 mg/kg/day orally for 12 months.
DRUG	Placebo	Placebo group: 15 patients, both sexes, will receive oral placebo for 12 months.

Timeline

• Start date: 2018-11-07
• Primary completion: 2023-05-04
• Completion: 2023-08-03
• First posted: 2018-08-15
• Last updated: 2022-12-19

Locations

1 site across 1 country: Brazil