Conditions: Amyloidosis
Keywords: Amyloid Amyloidosis AL Amyloidosis Primary Amyloidosis Stem Cell Transplant
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 35
Healthy volunteers: no

Brief summary:
The drugs dexamethasone and bortezomib are both FDA-approved for the treatment of multiple myeloma, a disease very similar to amyloidosis. However, they are currently investigational for the treatment of amyloidosis.

We want to find out if the addition of dexamethasone and bortezomib to standard high dose chemotherapy and stem cell transplant can help improve response.

Standard treatment includes four steps: 1) Stem Cell Mobilization (standard) 2) Stem Cell Collection (standard) 3) Conditioning Regimen (Melphalan chemotherapy). The conditioning regimen helps to kill the abnormal cells in the body and makes room in the bone marrow for new blood stem cells to grow. 4) Stem Cell Infusion

Participants in this study will have an additional treatment step called "induction therapy", designed as the first step towards reducing the number of abnormal cells in the body. Two cycles of the investigational drugs bortezomib and dexamethasone will be given during induction therapy. In addition, bortezomib will given as part of the conditioning regimen, in addition to the standard melphalan chemotherapy.

Detailed description:
The drugs dexamethasone and bortezomib are both FDA-approved drugs for the treatment of multiple myeloma, a disease very similar to amyloidosis. However, they are currently investigational for the treatment of amyloidosis.

The investigators want to find out if the addition of dexamethasone and bortezomib to standard treatment of high dose chemotherapy and stem cell transplant can help improve response to treatment.

Arms:
arm 1: Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Interventions: intervention 1: Bortezomib intervention 2: Dexamethasone intervention 3: Melphalan
Intervention descriptions: intervention 1: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 intervention 2: Induction:

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days intervention 3: Conditioning:

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Protocol document: