Completed

A Phase II Trial of Tandem Transplantation in AL Amyloidosis

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What is being tested

filgrastim

+ melphalan
+ autologous peripheral blood stem cell transplantation
Drug
Procedure
Who is being recruted

Amyloidosis
+14

+ Blood Protein Disorders
+ Cardiovascular Diseases
From 18 to 65 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: August 2000
See protocol details

Summary

Principal SponsorBoston Medical Center
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: August 1, 2000Actual date on which the first participant was enrolled.

OBJECTIVES: * Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis. * Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients. * Determine the overall survival of patients treated with this regimen. OUTLINE: * First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected. Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0. * Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0. Treatment continues in the absence of unacceptable toxicity. Patients are followed at 3 and 6 months, 1 year, and then annually thereafter. PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.

Official TitleA Phase II Trial of Tandem Transplantation in AL Amyloidosis 
NCT00075621
Principal SponsorBoston Medical Center
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
62 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

How participants are assigned to different groups/arms
In this clinical study, all participants receive the same treatment. Since there is only one group, there is no need for randomization or assignment to different arms. This type of study is often used to test a new treatment without comparing it to another.

Other Ways to Assign Participants
Randomized allocation: Participants are assigned randomly, like flipping a coin, to ensure fairness and reduce bias.
Non-randomized allocation: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

How treatments are given to participants
In this study, all participants receive the same treatment. This approach is often used to evaluate the effects of a single intervention without comparing it to another.

Other Ways to Assign Treatments
Parallel assignment: Participants are split into separate groups, each receiving a different treatment.
Cross-over assignment: Participants switch between treatments during the study.
Factorial assignment: Participants receive different combinations of treatments.
Sequential assignment: Participants receive treatments one after another in a specific order, possibly based on individual responses.
Other assignment: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a non placebo-controlled study, no participants receive an inert substance (placebo) to compare outcomes. Instead, all participants receive either the experimental treatment or an alternative treatment (often the Standard of Care). This method allows researchers to compare the effects of the experimental treatment with those of a different active intervention, rather than a placebo.

Other Options
Placebo-Controlled: A placebo is used to compare the effects of the experimental treatment with those of an inert substance, isolating the true treatment effect.

How the interventions assigned to participants is kept confidential
Everyone involved in the study knows which treatment is being given. This is typically used when it's not possible or necessary to hide the treatment details from participants or researchers.

Other Ways to Mask Information
Single-blind: Participants do not know which treatment they are receiving, but researchers do.
Double-blind: Neither participants nor researchers know which treatment is given.
Triple-blind: Participants, researchers, and outcome assessors do not know which treatment is given.
Quadruple-blind: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
From 18 to 65 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Amyloidosis
Blood Protein Disorders
Cardiovascular Diseases
Hematologic Diseases
Hemorrhagic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphoproliferative Disorders
Metabolic Diseases
Multiple Myeloma
Neoplasms
Neoplasms by Histologic Type
Paraproteinemias
Vascular Diseases
Hemostatic Disorders
Neoplasms, Plasma Cell
Proteostasis Deficiencies
Criteria

Inclusion Criteria: DISEASE CHARACTERISTICS: * Histologically confirmed AL amyloidosis, meeting 1 of the following criteria: * Plasma cell dyscrasia, evidenced by 1 of the following: * Monoclonal protein in the serum or urine by immunofixation electrophoresis * Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype * Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out PATIENT CHARACTERISTICS: Age * 18 to 65 Performance status * SWOG 0-2 Life expectancy * At least 1 year Hematopoietic * Not specified Hepatic * Not specified Renal * Not specified Cardiovascular * LVEF ≥ 45% by MUGA or echocardiogram Pulmonary * DLCO ≥ 50% Other * Not pregnant or nursing * Fertile patients must use effective contraception * Able to tolerate 2 courses of high-dose therapy * HIV negative PRIOR CONCURRENT THERAPY: Biologic therapy * Not specified Chemotherapy * Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes * Prior total cumulative oral melphalan dose \< 300 mg Endocrine therapy * Not specified Radiotherapy * Not specified Surgery * Not specified Other * At least 4 weeks since prior cytotoxic therapy and recovered Exclusion Criteria: * No senile, secondary, localized, dialysis-related, or familial amyloidosis * No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive \[more than 2\] lytic lesions, hypercalcemia) Cardiovascular * No myocardial infarction within the past 6 months * No congestive heart failure * No arrhythmia refractory to therapy * No evidence of symptomatic transient ischemic attacks or strokes * No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
One single intervention group 

is designated in this study

This study does not include a placebo group 

Treatment Groups
Group I
Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection Drug: melphalan 200 mg/kg over 2 days Procedure/Surgery: autologous peripheral blood stem cell transplantation autologous peripheral blood stem cell transplantation

16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection

200 mg/kg over 2 days

autologous peripheral blood stem cell transplantation
Study Objectives
Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 1 location
Suspended
Cancer Research Center at Boston Medical CenterBoston, United StatesSee the location
CompletedOne Study Center
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