None listed
Conditions
Brief summary
This study aims to investigate the safety and efficacy of autologous stem cell transplantation in AL amyloid patients with advanced cardiac disease. after a orthotopic heart transplantation. Who is it for? You may be eligible to join this study if you are aged between 18-65 years and have been diagnosed with cardiac AL amyloidosis. Study details All participants in this study are required to have previously received chemotherapy and a orthotopic heart transplantation before being enrolled in the study to received an autologous stem cell transplantation. Patients will undergo autologous stem cell transplantation (ASCT) within 3-6 months after OHT. patient will have an Autologous stem cell transplant using Melphalan 200mg/m2 on day -1 with stem cell collected given on day 0 previously from the patient before the study. All participants will be followed up every 3 to 6 months for a period of 5 years, in order to assess survival, and safety and efficacy of treatment. This pilot study will determine if treating patient with a stem cell transplant with cardiac amyloid after receiving a heart transplant will increase disease free survival
Interventions
Patients who have advanced cardiac amyloidosis who have received a heart transplant after completing Bortezomib-based or immunomodulatory (thalidomide, lenalidomide or pomalidomide) based chemotherapy, are eligible to enro in this study. The study involves eligible patients who are well enough as assessed by their treating physicians to undergo Autologous Stem Cell Transplant within 3-6 months after heart transplant.
Enrolled patients will receive melphalan 200mg/m2 for one dose intravenously t
Patients who have advanced cardiac amyloidosis who have received a heart transplant after completing Bortezomib-based or immunomodulatory (thalidomide, lenalidomide or pomalidomide) based chemotherapy, are eligible to enro in this study. The study involves eligible patients who are well enough as assessed by their treating physicians to undergo Autologous Stem Cell Transplant within 3-6 months after heart transplant. Enrolled patients will receive melphalan 200mg/m2 for one dose intravenously the day before stem cells are infused. Prophylactic medication to prevent infection will commence as determined by treating physician. - Subcutaneous Filgrastim 5 ug/kg given daily could be considered if transplant engraftment is delayed beyond 28 days. The ASCT will follow standard guidelines by the BMT Network . Patients will be followed up clinically weekly in the first month, then every 3 months for the first year, then 6 monthly for 5 years by their treating physician after stem cell transplant using physical assessments and pathology tests - After the stem cell transplant, medical reviews will also be customised be for each patient if patient need more regular follow up.
Sponsors
St Vincent's Hospital Sydney
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Cardiac AL Amyloidosis, Stage III (a) or (b) prior to heart transplantation 2. received orthotopic heart transplantation 3. adequate cardiac function: Ejection fraction > 50%, no restrictive cardiomyopathy in echocardiogram or cardiac MRI 4. absence of cardiac rejection 5. no evidence of amyloid infiltration to the cardiac allograft 6. Measurable light chains prior to induction chemotherapy (FLC > 1.5xULN with abnormal kappa:lambda ratio) 7. Measureable NT-ProBNP and Troponin-T prior to induction chemotherapy 8. ECOG status of less than 2 or Karnofsky score less than 60 (see appendix B) 9. Able to provide informed consent
Exclusion criteria
1. Amyloidosis other than AL Amyloidosis. This includes AA amyloidosis, senile amyloidosis, heritable amyloidosis (including but not limited to transerythin (ATTR) cardiac amyloidosis). Patients will require a negative genetic screen for heritable amyloidosis at Westmead Hospital Amyloid unit. 2. Diagnosis of multiple myeloma with more than 20% bone marrow plasma cells with end-organ involvement 3. Diagnosis of other haematological or solid organ malignancies 4. Other Amyloidosis-related end-organ diseases including renal disease (creatinine greater than 2x ULN), hepatic failure (AST, ALT greater than 3x ULN, Bilirubin > 2x ULN) 5. Significant cytopenias: Haemoglobin level <80g/L, neutrophil count <1x109/L, platelet count <75x109/L 6. Hepatitis B, C or HIV seropositivity 7. Pregnancy or breastfeeding 8. Patient with other serious medical or psychiatric illness likely to interfere with participation in this clinical study 9. Greater than grade 1 peripheral neuropathy 10. Smoking or intravenous drug use within 6 months of potential cardiac transplant
Outcome results
None listed