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Effect of Low Dose Radiotherapy on ATTR Cardiac Amyloidosis : a Proof of Concept Study

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ClinicalTrials.gov Identifier: NCT03397810
Recruitment Status : Not yet recruiting
First Posted : January 12, 2018
Last Update Posted : January 12, 2018
Sponsor:
Information provided by (Responsible Party):
Doctor René Nkoulou, University Hospital, Geneva

Brief Summary:
Cardiac amyloidosis is responsible for significant morbidity associated with heart failure, and carries a poor prognosis. Currently there are very limited treatment options for this condition. Radiotherapy has been used successfully to treat amyloidosis elsewhere in the body, however has not been trialled in cardiac amyloidosis. Therefore this study aims to assess the effect of radiotherapy on cardiac amyloidosis, to evaluate whether it can successfully reduce the burden of amyloid deposits in the myocardium as assessed by 18F-Amyloid PET.

Condition or disease Intervention/treatment Phase
Amyloid Cardiomyopathy Radiation: low dose radiotherapy Not Applicable

Detailed Description:

The intervention will involve administration of external beam radiotherapy (5 fractions of 2Gy) focused to the heart.

Measurements of effect will be assessed at 12 weeks by:

Amyloid PET Cardiac MRI with administration of gadolinium and ultrasound A blood venous sample (cardiac biomarkers) Quality of life assessments


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effect of Low Dose Radiotherapy on ATTR Cardiac Amyloidosis : a Proof of Concept Study
Estimated Study Start Date : February 1, 2018
Estimated Primary Completion Date : January 4, 2020
Estimated Study Completion Date : December 30, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arm Intervention/treatment
Experimental: Singe arm
Subjects will receive a low dose radiotherapy focused to the heart
Radiation: low dose radiotherapy
10 Gy in 5 fractions of 2 Gy on 5 consecutive days




Primary Outcome Measures :
  1. Assessment of the degree of amyloid [ Time Frame: 12 weeks ]
    Assess intra-individual change in a quantitative measure of amyloid deposits on 18F-Amyloid (Standard Uptake Value ratio) SUVR between amyloid PET scans before and after low dose RT


Secondary Outcome Measures :
  1. Safety and adverse event associated with cardiac low dose RT [ Time Frame: 6 months ]
    Assess the number of patients who report adverse events

  2. Modification in echocardiographic global longitudinal strain [ Time Frame: 6 months ]
    Assess intra-individual change in a quantitative measures

  3. Modification in MRI extracellular volumes and T1 values [ Time Frame: 12 weeks ]
    Assess intra-individual change in a quantitative measures

  4. Modification in cardiac biomarker (ultrasensitive troponin T and BNP blood levels) features of cardiac amyloidosis [ Time Frame: 6 months ]
    Assess intra-individual change in a quantitative measures

  5. Modification of quality of life [ Time Frame: 6 months ]
    The quality of life will be assessed by the SF-36 short form health survey quality of life scale



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Ages Eligible for Study:   65 Years and older   (Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age >65 y.o.
  • Dyspnoea on exertion (NYHA II or more).
  • Stable elevated cardiac enzymes (ultra sensitive Troponin T > 14 ng/L on consecutive sampling or BNP > 100pg/mL)
  • A positive 99mTc-DPD cardiac scintigraphy (Grade 2 and 3) suggesting an ATTR or wt amyloidosis.
  • Additional imaging also compatible with cardiac amyloidosis (cardiac ultrasound showing basal to apical longitudinal strain gradient and magnetic resonance imaging with elevated T1 value or extracellular volume).
  • Compliance with the informed consent as attested by its signature.
  • Positive baseline 18F-Florbetapir imaging, as assessed visually and quantitatively by a Tissue to Background Ratio > 1.45

Exclusion Criteria:

  • Positive serum protein immunoelectrophoresis with monoclonal gammapathy.
  • Previous external beam radiotherapy including the chest.
  • Claustrophobia
  • Presence of internal non-MR compatible devices
  • Creatinine glomerular filtration rate < 30 ml/min
  • Oncologic disease (excluding skin cancer) active or in remission from less than 5 years

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03397810


Contacts
Contact: René Nkoulou, Dr. +41 22 37 27 196 rene.nkoulou@hcuge.ch
Contact: Philippe Meyer, Dr. +41 22 37 27 225 philippe.meyer@hcuge.ch

Sponsors and Collaborators
Doctor René Nkoulou

Responsible Party: Doctor René Nkoulou, Principal Investigator, University Hospital, Geneva
ClinicalTrials.gov Identifier: NCT03397810     History of Changes
Other Study ID Numbers: 2017-00640
First Posted: January 12, 2018    Key Record Dates
Last Update Posted: January 12, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Doctor René Nkoulou, University Hospital, Geneva:
cardiac amyloidosis
radiotherapy

Additional relevant MeSH terms:
Cardiomyopathies
Amyloidosis
Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases