Trial · NCT00477971
Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
NCT00477971, Phase 3, Completed, Mayo Clinic; results first posted 2015-07-21
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis. Trials, record NCT00477971. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00477971
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
- NCT number
- NCT00477971
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 89 participants, actual
- Lead sponsor
- Mayo Clinic
- Conditions
- Multiple Myeloma and Plasma Cell Neoplasm
- Interventions
- filgrastim, dexamethasone, melphalan, autologous hematopoietic stem cell transplantation
- First posted
- 2007-05-24
- Last updated
- 2016-05-17
- Results first posted
- 2015-07-21
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Hematologic Response Rate | 10 years | Response that was confirmed on 2 consecutive evaluations during treatment. A hematologic response consisted of a Complete response, Very Good Partial Response or Partial Response. * Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and \<5% plasma cells in bone marrow. * Very Good Partial Response (VGPR): \>=90% reduction in serum M-component; Urine M-Component \<=100 mg per 24 hours. * Partial Response (PR): \>=50% reduction in serum M-component and/or Urine M-Component \>=90% reduction or \<200 mg per 24 hours; or \>=50% decrease in difference between involved and uninvolved FLC levels. |
| Secondary | 3 Year Overall Survival | 3 years | Percentage of patients who were alive at 3 years. The 3-year survival rate was estimated using the Kaplan Meier method. |
| Secondary | Organ Response to Treatment | 10 years | Organ response was evaluated on the basis of improvement of one or more affected organ; only one parameter was required to satisfy the criteria. Response needed to be maintained for a minimum of 3 months to be considered valid. Renal response required a 50% reduction in 24-hour urine protein excretion (at least 0.5 g/d) with stable creatinine. Cardiac response required one of \>= 2-mm reduction in the interventricular septal (IVS) thickness by echocardiogram, or improvement of ejection fraction by \>= 20%, or improvement by 2 NYHA classes without an increase in diuretic use. Hepatic response required either \>= 50% decrease in (or normalization of) an initially elevated alkaline phosphatase level or reduction in the size of the liver by at least 2 cm by radiographic determination. Gastrointestinal tract improvement was defined as normalization of a low serum carotene level, or reduction of diarrhea to \< 50% of previous movements/day, or decrease in fecal fat excretion by 50%. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Hematologic Response Rate | 10 years | percentage of participants | NUMBER | |
| SECONDARY | 3 Year Overall Survival | 3 years | percentage of participants | NUMBER | |
| SECONDARY | Organ Response to Treatment | 10 years | percentage of participants | NUMBER | |
| POST_HOC | 3-Year Progression Free Survival | 3 years | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Hematologic Response Rate | Low-Dose Melphalan | 55.9 | 95% Confidence Interval 37.9 to 72.8 | percentage of participants | |
| Hematologic Response Rate | High-Dose Melphalan + Autologous HSC | 69.1 | 95% Confidence Interval 55.2 to 80.9 | percentage of participants | |
| 3 Year Overall Survival | Low-Dose Melphalan | 58.8 | 95% Confidence Interval 44.4 to 77.9 | percentage of participants | |
| 3 Year Overall Survival | High-Dose Melphalan + Autologous HSC | 83.6 | 95% Confidence Interval 74.7 to 94 | percentage of participants | |
| Organ Response to Treatment | Low-Dose Melphalan | 26.5 | 95% Confidence Interval 12.8 to 44.4 | percentage of participants | |
| Organ Response to Treatment | High-Dose Melphalan + Autologous HSC | 29.1 | 95% Confidence Interval 17.6 to 42.9 | percentage of participants | |
| 3-Year Progression Free Survival | Low-Dose Melphalan | 29.1 | 95% Confidence Interval 17.2 to 49.4 | percentage of participants | |
| 3-Year Progression Free Survival | High-Dose Melphalan + Autologous HSC | 51.7 | 95% Confidence Interval 39.8 to 67 | percentage of participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Low-Dose Melphalan | 34 | |
| Overall Study | STARTED | High-Dose Melphalan + Autologous HSC | 55 | |
| Overall Study | COMPLETED | Low-Dose Melphalan | 17 | |
| Overall Study | COMPLETED | High-Dose Melphalan + Autologous HSC | 49 | |
| Overall Study | NOT COMPLETED | Low-Dose Melphalan | 17 | |
| Overall Study | NOT COMPLETED | High-Dose Melphalan + Autologous HSC | 6 |
Source ClinicalTrials.gov
Adverse events310
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Blood disorder | Blood and lymphatic system disorders | Low-Dose Melphalan | 0 | 34 | 0 |
| other | Blood disorder | Blood and lymphatic system disorders | High-Dose Melphalan + Autologous HSC | 1 | 55 | 1 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Low-Dose Melphalan | 0 | 34 | 0 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | High-Dose Melphalan + Autologous HSC | 32 | 55 | 33 |
| other | Hemoglobin decreased | Blood and lymphatic system disorders | Low-Dose Melphalan | 17 | 34 | 41 |
| other | Hemoglobin decreased | Blood and lymphatic system disorders | High-Dose Melphalan + Autologous HSC | 49 | 55 | 63 |
| other | Hemolysis | Blood and lymphatic system disorders | Low-Dose Melphalan | 0 | 34 | 0 |
| other | Hemolysis | Blood and lymphatic system disorders | High-Dose Melphalan + Autologous HSC | 1 | 55 | 1 |
| other | Arrhythmia | Cardiac disorders | Low-Dose Melphalan | 0 | 34 | 0 |
| other | Arrhythmia | Cardiac disorders | High-Dose Melphalan + Autologous HSC | 2 | 55 | 2 |
1 to 10 of 310Source ClinicalTrials.gov
See all 310 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Low-Dose Melphalan | ||
| Total, all-cause mortality | High-Dose Melphalan + Autologous HSC | ||
| Total, other adverse events | Low-Dose Melphalan | 34 | 34 |
| Total, other adverse events | High-Dose Melphalan + Autologous HSC | 55 | 55 |
| Total, serious adverse events | Low-Dose Melphalan | 6 | 34 |
| Total, serious adverse events | High-Dose Melphalan + Autologous HSC | 3 | 55 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| P30CA015083 | Mayo Clinic Comprehensive Cancer Center Support Grant | WILLMAN, CHERYL LYNN | MAYO CLINIC ROCHESTER | NATIONAL CANCER INSTITUTE | 1985 to 2025 |
Source NIH RePORTER
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Low-Dose Melphalan vs High-Dose Melphalan + Autologous HSC: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00477971
GET https://conductscience.org/api/trials/v1/NCT00477971/events?page=2