Clinical trial NCT01273844: Study of Bortezomib +HSCT in Primary Systemic Amyloidosis (AL)
Official title: Study of the Combination of Bortezomib and Dexamethasone Followed by HSCT in AL
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Amyloidosis
Keywords: hematopoietic stem cell transplantation
amyloidosis
Bortezomib
Lead sponsor: Nanjing University School of Medicine
Sponsor class: OTHER
Collaborators: name: Soochow University
name: Nanjing Medical University
First posted: 2011-01-11
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the efficacy and safety of Vel-Dex therapy (bortezomib and dexamethasone) followed by autologous hematopoietic stem cell transplantation as an initial treatment in patients with newly diagnosed AL.

Detailed description: Patients will receive two 21-day cycles of induction therapy with vel / dex regimen. Bortezomib 1.3mg/m2 on days 1, 4, 8 and 11 will be given by intravenous bolus injection while Dexamethasone 40 mg/d will be taken orally on days 1-4. After two cycles of vel / dex therapy, the collection of peripheral blood stem cells (PBSC) should be completed within 4 weeks.Patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 100,140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients ) and Vel 1mg/m2 (days -6, -3, +1, +4) will be given. Four additional 21-day cycles of Vel treatment (with a dose of 1.6mg/m2 on day 1 and 8 of the cycle) will be conducted as consolidation therapy during the recommended 60-90 days after HSCT, or after resumption of hematopoietic function ( neutrophil count \> 1.5x109/L and platelet count\> 50x109/L). Later patients won't need maintenance therapy.

The efficacy criteria are the international standards set with consensus of experts in the Tenth International Symposium on Amyloid and Amyloidosis.

Eligibility criteria: Inclusion Criteria:

1. Male or female;
2. aged 18-65 years;
3. Patients with newly diagnosed AL;
4. Appropriate for autologous hematopoietic stem cell transplantation;
5. Abnormal M protein or free light chain detected in serum and / or urine
6. ECOG score 0-2 points;
7. Subjects (or their legal representatives) must signed an informed consent document indicating understanding the purpose of and procedures required for the study and willingness to participate in the study.

Exclusion Criteria:

1. Subjects received systematic treatment with steroids
2. Subjects received plasmapheresis to treat clinical significant hyperviscosity syndrome within 4 weeks before enrollment.
3. Pregnant and breastfeeding women;
4. Subjects suffering from multiple myeloma.
5. hypersensitivity to dexamethasone, bortezomib, mannitol, boron, or heparin (if use catheters);
6. Subjects have severe cardiovascular disease,
7. Subjects have serious physical disease and mental illnesses which may interfere the study

Primary outcomes:
measure: Complete response rate
timeFrame: 12 months
Secondary outcomes:
measure: overall response rate
timeFrame: 12 months
Other outcomes:
measure: progression-free survival
timeFrame: 2 years
Locations:
city: Suzhou
country: China
facility: Soochow University
state: Jiangsu
Study officials:
affiliation: Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China
name: zhihong Liu, Master
role: PRINCIPAL_INVESTIGATOR
References: