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Institutional Registry of Amyloidosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Hospital Italiano de Buenos Aires
Sponsor:
Information provided by (Responsible Party):
Diego Hernan Giunta, MD, Hospital Italiano de Buenos Aires
ClinicalTrials.gov Identifier:
NCT01347047
First received: May 2, 2011
Last updated: May 28, 2014
Last verified: May 2014
  Purpose
  1. Creating a population-based registry system Amyloidosis prospective epidemiological survey

    • risk factors
    • diagnosis
    • prognosis
    • treatment
    • monitoring
    • survival
  2. Describe the occurrence of amyloidosis in the population of HIBA, Hospital Italiano de Buenos Aires.
  3. Describe the characteristics of clinical presentation, evolution and predisposing factors of amyloidosis.

Condition
Amyloidosis

Study Type: Observational
Study Design: Observational Model: Ecologic or Community
Time Perspective: Prospective
Official Title: Institutional Registry of Amyloidosis

Resource links provided by NLM:


Further study details as provided by Hospital Italiano de Buenos Aires:

Primary Outcome Measures:
  • Epidemiologic characteristics [ Time Frame: From inclusion date to 1year ] [ Designated as safety issue: No ]

Estimated Enrollment: 120
Study Start Date: April 2011
Estimated Study Completion Date: January 2020
Estimated Primary Completion Date: January 2020 (Final data collection date for primary outcome measure)
Detailed Description:

Amyloidosis is a systemic disease that is usually a result of misfolded proteins in the form of amorphous fibrillar material in various tissues and can cause progressive dysfunction of the same. The prevalence of amyloidosis varies depending on the population concerned and the type of amyloid. While prevalence in the general population is unknown, according to estimates by the Mayo Clinic this prevalence is 1 in 90 666% in the U.S. In England this disease generated about 0.0084% (1367 / 16232579) of all hospital visits between April 2008 and April 2009.

The most common clinical manifestations include cardiac disease, renal and liver function, but it may vary widely depending on the type of amyloidosis, the organ infected and extent of the deposits. Amyloid infiltration can produce signs and symptoms that may be very similar to other rheumatic diseases. This may suggest potential clinical polymorphic underdiagnosis due to low clinical suspicion.

The registries are organized systems of systematic data collection of a large number of patients quickly and efficiently on a particular disease at a given time.

The main difficulty of the registries is the guarantee of the quality of their data.

The main objectives of the registry are:

  1. Understand risk factors and prognosis.
  2. Evaluate the diagnostic and therapeutic comparison with current standards.
  3. Advance knowledge of the disease to optimize the assessment, treatment and monitoring of patients.
  4. Analyze the effectiveness of new therapies.
  5. Studying differences between populations.
  6. Quickly estimate the morbidity, mortality and resource utilization associated with a disease entity.
  7. Examine the course of a disease
  8. Formulate novel hypotheses for further prospective studies. Currently, there are registries for patients with transthyretin amyloidosis (TAHOS), global registry of transplant patients with familial amyloid poly neuropathy. Also, there are indirect registries such as the kidney transplant, heart transplant, among others.

The investigators found no data on the prevalence or incidence, evolution and prognosis of amyloidosis in our country. There are no existing records of national Amyloidosis in Latin America that could describe the behavior of this disease in our environment. Because it is a chronic disease with amyloid infiltration and can produce signs and symptoms that may be very similar to other rheumatic diseases, this clinic potentially poliform, may suggest underestimation of low clinical suspicion. As there is no cure, some patients may persist symptomatic despite adequate therapy, that is why it is important the creation of a monitoring system to generate data on the evolution and prognosis. The data registries can be used to develop new treatment guidelines and recommendations, also to inform and educate physicians on the management of this disease.

The Hospital Italiano de Buenos Aires is a center of high complexity of derivation of this type of pathology and due to the fact that the hospital, that has a private health insurance system (HIBA's health plan [plan de salud, PS], gives the unique opportunity denominators for the generation of a population on their affiliates, therefore the investigators propose to make a Institutional Registry of Amyloidosis.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Adults over 18 years old with diagnosis or suspected amyloidosis of Italiano Hospital of Buenos Aires

Criteria

Inclusion Criteria:

Patients over 18 years:

Confirmed amyloidosis:

• Proof of deposit of amyloid pathology by tissue biopsy in abdominal fat, bone marrow, rectum or organ involved (eg, kidney, liver, sural nerve)

Clinically compatible but unconfirmed case of Amyloidosis :

Cardiology: The presence of 2 or more of these points:

  • Right heart failure and/or left
  • ECG: QRS voltage decreased
  • Echocardiogram: thickness increased with hyper-refraction of the ventricular walls, increased inter-atrial septum thickness. With no other clear cause of increased ventricular thickness (hypertension, valvular heart disease)
  • Cardiac MRI: diffuse subendocardial increased gadolinium uptake on delayed images.

Renal:

• Renal involvement but serum free lambda chains

Other:

  • Evidence of clonal proliferation of plasma cells. This includes monoclonal protein serum/urinary, plasma cells in bone marrow, abnormal relationship of serum light chains without evidence of amyloid deposit composed of fragments of light chain by immunofluorescence or immunohistochemistry, both lambda and kappa
  • blood relatives of patients with confirmed familial amyloidosis presenting clinical features of poly neuropathy without alternative cause gene mutation confirmed amyloidogenic protein

Exclusion Criteria:

Refusal to participate in the study or the informed consent process by the patient or legal representative or refusal to consent to participate in the study in the case of minors.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01347047

Contacts
Contact: DIego H GIUNTA, MD +5411 49590200 ext 4419 diego.giunta@hospitalitaliano.org.ar
Contact: Lourdes Posadas-MArtinez, MD +5411 49590200 ext 4419 maria.posadas@hospitalitaliano.org.ar

Locations
Argentina
Hospital Italiano de Buenos Aires Recruiting
Buenos Aires, Argentina
Contact: Diego H. Giunta, MD    5411 9590200 ext 4419    diego.giunta@hospitalitaliano.org.ar   
Sponsors and Collaborators
Hospital Italiano de Buenos Aires
Investigators
Study Chair: María Lourdes Posadas-Martínez, MD Clinical Research Area Staff
Study Chair: Elsa Nucifora, MD Hematology Service Chief
Study Chair: Diego Hernan Giunta, MD Clinical Research Area Coordinator
Study Chair: Dorotea Fantl, MD Hematology
Study Chair: Gustavo Greloni, MD Nephrology
Study Chair: Maria Adela Aguirre, MD Internal Medicine
Study Chair: Cesar Antonio Belziti, MD Cardiology
Study Chair: Judith Nuñez, Nurse Hematology Nurse
Study Chair: Melisa M Blomberg, MD Clinical Research Area Staff
  More Information

No publications provided

Responsible Party: Diego Hernan Giunta, MD, MD, Hospital Italiano de Buenos Aires
ClinicalTrials.gov Identifier: NCT01347047     History of Changes
Other Study ID Numbers: 1675
Study First Received: May 2, 2011
Last Updated: May 28, 2014
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica

Keywords provided by Hospital Italiano de Buenos Aires:
Amyloidosis
Known or Suspected

Additional relevant MeSH terms:
Amyloidosis
Metabolic Diseases
Proteostasis Deficiencies

ClinicalTrials.gov processed this record on November 24, 2014