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Clinical Trial Details

Orthotopic heart transplantation followed by autologous stem cell transplantation in patients with cardiac AL amyloidosis - a Phase II study

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Trial Information

Broad Health Condition Cancer
Cardiovascular

Specific Health ConditionMyeloma
Other cardiovascular diseases

Trial FocusTreatment

Recruitment statusRecruiting

Recruitment Details
Recruitment State
NSW

Hospital
St Vincent's Hospital (Darlinghurst) - Darlinghurst


Postcode
2010 - Darlinghurst

Phase of TrialNot Applicable

Has the study received ethics approval?Further information iconApproved

Trial 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 
Eligibility

Key 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

Minimum age18 Years

Maximum age65 Years

GenderBoth males and females

Can Healthy volunteers participate?No

Key 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
Contact details and further information

Sponsor Primary Sponsor Type: Hospital
Primary Sponsor Name: St Vincent's Hospital Sydney
Primary Sponsor Address: 390 Victoria St, Darlinghurst NSW 2010
Primary Sponsor Country: Australia

Trial IDACTRN12617000215325

Contact person for information and recruitmentDr
Kris Ma
St Vincent's Hospital, Sydney 390 Victoria St Darlinghurst NSW 2010
+61 2 9355 5656
+61 2 9355 5735
Further information iconSVHS.CancerResearch@svh.org.au
Australia