AmyloidosisPhase 2NCT00017680
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
A Phase 2 study testing Melphalan and Autologous Stem Cell Transplantation in people with Amyloidosis, run by Herbert Irving Comprehensive Cancer Center at 1 site in United States.
Trial summary
OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. II. Determine the toxicity of this regimen in these patients. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Amyloidosis
- What is being tested?
- Melphalan and Autologous Stem Cell Transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 1999to Apr 2004
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
Disease Characteristics
- Histologically confirmed primary amyloidosis
- Ineligible for other high priority national or international study
Prior/Concurrent Therapy
- Biologic therapy: Concurrent participation in gene therapy trials allowed
- Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
- Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes)
- Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed
Patient Characteristics
- Performance status: ECOG 0-3
- Hepatic: Bilirubin less than 2 times normal
- Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
- Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
- Other: HIV negative
Trial locations and contacts
1 site in 1 country.
United States1 site
Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
About this study
Official title: Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.
II. Determine the toxicity of this regimen in these patients.
Read the detailed description
PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.
Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.
Patients are followed every 3 months for 1 year and then annually for 5 years.
- Conditions
- Amyloidosis
- Being tested
- Melphalan, Autologous Stem Cell Transplantation
- Keywords
- amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease
- Medical subject headings
- Amyloidosis, Arthritis, Connective Tissue Diseases, Genetic Diseases, Inborn, Hematologic Neoplasms, Neoplasms, Plasma Cell, Immunoglobulin Light-chain Amyloidosis, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MelphalanDrug
100 mg/m2 twice a day from Days -2 and -1 as induction therapy
Autologous Stem Cell TransplantationProcedure or surgery
Bone marrow and peripheral blood stem cells harvested
What is being measured
The primary outcome is the main question the study is designed to answer.
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
Secondary outcomes (1)
Toxicity of high dose chemotherapy regimen
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2001 (estimated)
- Study startJul 1999
- Primary completionApr 2004
- Study completionApr 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants
Sponsor and investigators
- Lead sponsor
- Herbert Irving Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Investigators
- Charles S. Hesdorffer, Study chair, Herbert Irving Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00017680
- Sponsor's protocol ID
- 199/15927
- Other IDs
- CPMC-IRB-9041, CPMC-CAMP-009A
- Status last verified
- Jun 2008
- First submitted
- 6 Jun 2001
- Last updated
- 9 Jun 2008
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
- ClinicalTrials.gov ID
- NCT00017680
- Acronym / sponsor ID
- 199/15927
- Conditions
- Amyloidosis
- Keywords
- amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants
- Lead sponsor
- Herbert Irving Comprehensive Cancer CenterUniversity, hospital or other
- Study start
- Jul 1999
- Primary completion
- Apr 2004 (actual)
- Study completion
- Apr 2004 (actual)
- First posted
- 6 Jun 2001 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.
II. Determine the toxicity of this regimen in these patients.
Detailed description
PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.
Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.
Patients are followed every 3 months for 1 year and then annually for 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Melphalan100 mg/m2 twice a day from Days -2 and -1 as induction therapy | Drug | – |
| Autologous Stem Cell TransplantationBone marrow and peripheral blood stem cells harvested | Procedure or surgery | – |
Primary outcome
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
1 secondary outcome
Toxicity of high dose chemotherapy regimen
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2008.
Related trials for Amyloidosis
RecruitingObservational study
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Relapsed/Refractory AL Amyloidosis
Active, not recruitingPhase 2
Siltuximab to Decrease Symptom Burden After Autologous Stem Cell Transplantation for Patients With Multiple Myeloma and AL Amyloidosis
Multiple Myeloma, AL Amyloidosis
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
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MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charles S. Hesdorffer study chair
Herbert Irving Comprehensive Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Melphalan and Autologous Stem Cell Transplantation in people with Amyloidosis, run by Herbert Irving Comprehensive Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jun 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jun 2008; AzTrials last refreshed its copy on 7 Nov 2026.