Trial record 5 of 130 for:    "AL amyloidosis"

High Cut-off Hemodialysis in Patients With Advanced Cardiac AL Amyloidosis and End Stage Renal Disease (DIACAL)

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2012 by IRCCS Policlinico S. Matteo
Sponsor:
Information provided by (Responsible Party):
Giampaolo Merlini, IRCCS Policlinico S. Matteo
ClinicalTrials.gov Identifier:
NCT01531751
First received: January 11, 2012
Last updated: November 30, 2012
Last verified: November 2012
  Purpose

The aim of the study is to assess survival of patients with advanced cardiac AL amyloidosis treated with high cut-off hemodialysis (HCO-HD) combined with chemotherapy.


Condition Intervention Phase
Primary Amyloidosis of Light Chain Type
Device: High Cut-off Hemodialysis
Drug: Chemotherapy
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Phase II Trial of Free Light Chain Removal by Extended High Cut-Off Hemodialysis in Patients With Advanced Cardiac AL Amyloidosis and End-Stage Renal Disease.

Resource links provided by NLM:


Further study details as provided by IRCCS Policlinico S. Matteo:

Primary Outcome Measures:
  • Survival [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    The primary objective will be to assess survival of patients with advanced cardiac AL amyloidosis treated with HCO-HD combined with chemotherapy.


Secondary Outcome Measures:
  • tolerability of the experimental device [ Time Frame: 1 month ] [ Designated as safety issue: Yes ]
    Secondary objectives will be to assess the feasibility and tolerability of HCO-HD in patients with advanced cardiac AL amyloidosis, the efficiency of HCO-HD plus chemotherapy in reducing amyloidogenic FLC in this setting, and the ability of this approach to promote improvement of cardiac dysfunction as assessed by biomarkers.


Estimated Enrollment: 16
Study Start Date: February 2012
Estimated Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: High Cut-off Hemodialysis Device: High Cut-off Hemodialysis
During dialysis the patients will undergo the first cycle of chemotherapy according to the current standard of care for subjects with advanced cardiac AL amyloidosis. Measurements of FLC and cardiac biomarkers will be done before and after each dialysis session. High cutoff HD will be continued during the first chemotherapy cycle. After the first cycle the end-of-treatment evaluation will take place.
Other Name: Theralite
Drug: Chemotherapy
Chemotherapy will be based on alchilators, proteasome inhibitors, steroids, IMiDs.

Detailed Description:

This will be a single-center phase II open label trial. Subjects with advanced cardiac AL amyloidosis and end-stage renal disease will undergo HCO-HD while receiving chemotherapy for their plasma cell dyscrasia according to the current standards of care.

After giving written informed consent, the patients will be evaluated for eligibility. Briefly, the subjects with a biopsy-proven diagnosis of AL amyloidosis who are cardiac stage 3 based on NT-proBNP (> 332 ng/L) and cTnI (> 100 ng/L) and whose estimated glomerular filtration rate (eGFR) is < 15 mL/min per 1.73 m2 will be eligible for the study. Patients with non-AL (e.g. familial and senile) amyloidosis will be excluded, as well as subjects who have less than 100 mg/L circulating amyloidogenic FLC. Sixteen patients will be enrolled.

The study includes 3 periods: screening, treatment followed by the end-of-treatment evaluation and follow-up. During dialysis the patients will undergo the first cycle of chemotherapy according to the current standard of care for subjects with advanced cardiac AL amyloidosis. Measurements of FLC and cardiac biomarkers will be done before and after each dialysis session. High cutoff HD will be continued during the first chemotherapy cycle. After the first cycle the end-of-treatment evaluation will take place. Follow-up evaluations will be performed after the end of each subsequent chemotherapy cycle and every 2 months after chemotherapy discontinuation for 1 year or until disease progression occurs.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of AL amyloidosis.
  • Age ≥ 18 years.
  • Evidence of a monoclonal light chain in serum and/or urine by positive immunofixation and abnormal FLC κ/λ ratio.
  • Evidence of cardiac involvement at echocardiography (mean left ventricular wall thickness > 12 mm in the absence of other causes).
  • Cardiac stage III (both NT-proBNP > 332 ng/L and cTnI > 100 ng/L).
  • Estimated GFR < 15 mL/min per 1.73 m2 or requiring dialysis.
  • NYHA class III or IV.
  • Initiation of chemotherapy for AL amyloidosis scheduled between day 3 and day 8 after first HCO-HD session.

Exclusion Criteria:

  • Non-AL (e.g. familial, senile) amyloidosis.
  • Concomitant non-amyloid related clinically significant cardiac diseases.
  • Involved (amyloidogenic) FLC < 100 mg/L.
  • Inability to undergo chemotherapy for AL amyloidosis.
  • Uncontrolled infection.
  • Inability to give informed consent.
  • Previous or ongoing psychiatric illness (excluding reactive depression).
  • Pregnant or nursing women.
  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: NCT01531751

Contacts
Contact: Giampaolo Merlini, Prof. +39(0)382502994 gmerlini@unipv.it
Contact: Giovanni Palladini, Dr. +39(0)382502994 g.palladini@smatteo.pv.it

Locations
Italy
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico S.Matteo Recruiting
Pavia, Italy, 27100
Contact: Giovanni Palladini, Dr.    +39(0)382502994    g.palladini@smatteo.pv.it   
Contact: Leda Roggeri, Dr.    +39(0)382502994    leda78@gmail.com   
Principal Investigator: Giampaolo Merlini, Prof.         
Sub-Investigator: Giovanni Palladini, Dr.         
Sponsors and Collaborators
IRCCS Policlinico S. Matteo
  More Information

Additional Information:
Publications:
Responsible Party: Giampaolo Merlini, Director, Amyloidosis Research and Treatment Center, IRCCS Policlinico S. Matteo
ClinicalTrials.gov Identifier: NCT01531751     History of Changes
Other Study ID Numbers: AC-005-IT
Study First Received: January 11, 2012
Last Updated: November 30, 2012
Health Authority: Italy: Ethics Committee

Keywords provided by IRCCS Policlinico S. Matteo:
HCO-HD
survival
cardiac biomarkers
AL amyloidosis
Light Chain Amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Kidney Diseases
Kidney Failure, Chronic
Proteostasis Deficiencies
Metabolic Diseases
Urologic Diseases
Renal Insufficiency, Chronic
Renal Insufficiency

ClinicalTrials.gov processed this record on July 24, 2014