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Register:	ClinicalTrials.gov
Last refreshed on:	9 January 2023
Main ID:	NCT03632213
Date of registration:	03/08/2018
Prospective Registration:	Yes
Primary sponsor:	Hospital de Clinicas de Porto Alegre
Public title:	Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Scientific title:	A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Date of first enrolment:	November 7, 2018
Target sample size:	10
Recruitment status:	Active, not recruiting
URL:	https://clinicaltrials.gov/show/NCT03632213
Study type:	Interventional
Study design:	Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
Phase:	Phase 2

Countries of recruitment
Brazil

Contacts

Name:	Guilherme Baldo, PhD
Address:
Telephone:
Email:
Affiliation:	Hospital de Clinicas de Porto Algre

Name:	Roberto Giugliani, MD, PhD
Address:
Telephone:
Email:
Affiliation:	Hospital de Clinicas de Porto Alegre

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed biochemical or molecular diagnosis of MPS VI or MPS IVA.

- Age between 10 and 40 years.

- Presence of aortic root diameter greater than 1.0 standard deviation, as determined by
local measurement.

- Be in a stable treatment regime in the last 3 months (without performing Enzyme
replacement therapy (ERT), or performing ERT on a regular basis).

- Patient who agree to participate in the study protocol by signing a free informed
consent form.

Exclusion Criteria:

- Patient who underwent previous aortic surgery.

- Patient with aortic root diameter greater than 5 cm.

- Patient on angiotensin-converting-enzyme (ACE) inhibitor. In case of use of
beta-blocker, or calcium channel blocker, patient without adequate control of blood
pressure in the last 3 months.

- Patients with previous adverse events related to treatment with losartan or
contraindication to this treatment.

- Inability, in the opinion of the investigator, to complete the study procedures.

Age minimum: 10 Years
Age maximum: 40 Years
Gender: All

Health Condition(s) or Problem(s) studied

Morquio Syndrome A

MPS VI

Mucopolysaccharidoses

Mucopolysaccharidosis VI

Mucopolysaccharidosis IV A

Morquio A Syndrome

MPS - Mucopolysaccharidosis

Morquio Syndrome

MPS IV A

Intervention(s)

Drug: Placebo

Drug: Losartan

Primary Outcome(s)

Adverse events related to losartan use [Time Frame: 12 months]

Secondary Outcome(s)

Changes in left ventricular longitudinal strain [Time Frame: 12 months]

Changes of serum levels of Troponin I [Time Frame: 12 months]

Changes of ventricular-vascular coupling measures as assessed by echocardiography between the baseline and 12 months. [Time Frame: 12 months]

Changes in aortic valve regurgitation [Time Frame: 12 months]

Changes in E/e' ratio [Time Frame: 12 months]

Changes of serum levels of brain-type natriuretic peptide (BNP) [Time Frame: 12 months]

Changes of serum levels of Chemokine (C-X-C motif) ligand 16 (CXCL16) [Time Frame: 12 months]

Changes of serum levels of Endocan-1 (ESM-1) [Time Frame: 12 months]

Changes of serum levels of N-terminal pro b-type natriuretic peptide (NT-ProBNP) [Time Frame: 12 months]

Changes of serum levels of Fatty acid binding protein 3 (FAPB3) [Time Frame: 12 months]

Changes of serum levels of Placental growth factor (PLGF) [Time Frame: 12 months]

Changes in ejection fraction [Time Frame: 12 months]

Z score of maximal aortic root diameter measured by Valsalva sinus [Time Frame: 12 months]

Changes of serum levels of transforming growth factor (TGF-Beta-1) [Time Frame: 12 months]

Changes of serum levels of Fatty acid binding protein 4 (FAPB4) [Time Frame: 12 months]

Changes of serum levels of Oncostatin M [Time Frame: 12 months]

Changes of serum levels of creatine kinase-myocardial ban (ck-mb) [Time Frame: 12 months]

Changes in E/A ratio [Time Frame: 12 months]

Changes in mitral valve regurgitation [Time Frame: 12 months]

Changes of serum levels of Chemokine (C-X-C motif) ligand 6 (CXCL6) [Time Frame: 12 months]

Secondary ID(s)

17-0685

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Isaac Foundation

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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