AL AmyloidosisPhase 3VelRandNCT02489500
Trial of High Dose Melphalan/Stem Cell Transplant With or Without Bortezomib
A Phase 3 study testing Bortezomib, Melphalan and Neupogen in people with AL Amyloidosis, run by Boston Medical Center at 1 site in United States.
Trial summary
Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant. This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AL Amyloidosis
- What is being tested?
- Bortezomib, Melphalan and Neupogen
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2015to 28 Apr 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histological diagnosis of primary systemic (AL) amyloidosis based on:
- Deposition of amyloid material by Congo red stain showing characteristic apple green birefringence,AND…
- evidence of a clonal plasma cell dyscrasia with monoclonal protein in the serum or urine by immunofixation electrophoresis studies AND/OR abnormal serum free light chain assay AND/OR clonal plasma cells in the bone marrow exam demonstrated by immunohistochemistry, flow cytometry or in situ hybridization AND…
- evidence of organ involvement other than carpal tunnel syndrome. Patients with senile, secondary, localized, dialysis-related or familial amyloidosis are not eligible. Confirmation of tissue diagnosis at all sites of organ dysfunction is encouraged, but not required.
- Patients must be > 18 years of age.
- Patients must have a performance status of 0-2 by Eastern Cooperative Oncology Group (ECOG) criteria
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- Patients must have left ventricular ejection fraction (LVEF) > 45% by echocardiogram within 60 days of enrollment
- Pulmonary Function Tests must show diffusing capacity of lung for carbon monoxide (DLCO) > 50%.
- All patients must be informed of the investigational nature of this study and must sign and give written informed consent in accordance with institutional and federal guidelines.
Who cannot join
- Patients with recent (< 6 months) myocardial infarction, congestive heart failure, New York Heart Association (NYHA) class III/IV or arrhythmia which are refractory to medical therapy are ineligible.
- Prior chemotherapy with alkylating agent allowed only if no evidence of Myelodysplastic Dysplastic Syndrome (MDS) morphologically or cytogenetically. Total cumulative dose of oral melphalan must be < 300 mg. Patients should not have received any cytotoxic therapy < 4 weeks prior to registration and should have fully recovered from the effects of such therapy.
- Patients must not have overt multiple myeloma (>30% bone marrow plasmacytosis and, extensive (>2) lytic lesions and hypercalcemia).
- No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years.
- Patients must not be HIV positive.
- Pregnant or nursing women may not participate. Women and men of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant.
This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.
Read the detailed description
This study seeks to enroll patients with AL amyloidosis who have been recommended for standard treatment with high-dose melphalan and stem cell transplant.
Standard treatment for this disease is high-dose melphalan and stem cell transplant.
The purpose of this study is to compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.
Patients enrolled in this study will receive either standard treatment with high-dose melphalan and stem cell transplant, or investigational bortezomib used in combination with standard treatment with high-dose melphalan and stem cell transplant.
- Conditions
- AL Amyloidosis
- Being tested
- Bortezomib, Melphalan, Neupogen, Stem Cell Collection, Stem cell infusion
- Keywords
- AL Amyloidosis, Stem Cell Transplantation
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
Also called VELCADE
Conditioning Regimen:
Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1
MelphalanDrug
Also called Alkeran
Conditioning Regimen:
Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0
NeupogenDrug
Also called granulocyte colony-stimulating factor (G-CSF)
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Stem Cell CollectionProcedure or surgery
Also called apheresis
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
Stem cell infusionProcedure or surgery
Also called infusion
infusion of previously collected autologous stem cells
| Study group | Role | What they receive |
|---|---|---|
| melphalan | Active comparator | Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Receives: Drug: Melphalan; Drug: Neupogen; Procedure: Stem Cell Collection; Procedure: Stem cell infusion |
| melphalan + Bortezomib | Experimental | Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Receives: Drug: Bortezomib; Drug: Melphalan; Drug: Neupogen; Procedure: Stem Cell Collection; Procedure: Stem cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Hematologic Response
Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains
Measured: 6 months
Secondary outcomes (3)
Toxicities
Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Measured: 100 days
Overall Survival
duration of overall survival measured in days
Measured: 5 years
Number of Participants With Organ Response
analysis of number of patients with organ response, as defined on page 13 of the detailed protocol for kidney, heart and liver.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2015 (estimated)
- Study startJun 2015
- Primary completion28 Apr 2017
- Study completion28 Apr 2017
Why it stopped: Enrollment held for toxicity evaluation; then closed due to competing trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AL AmyloidosisStudies of the same condition, open and under way.
- More trials run by Boston Medical CenterThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vaishali Sanchorawala, Boston Medical Center
- Investigators
- Vaishali Sanchorawala, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02489500
- Sponsor's protocol ID
- H-33808
- Other IDs
- VelRand
- Acronym
- VelRand
- Status last verified
- Aug 2018
- First submitted
- 9 Mar 2015
- Last updated
- 10 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
- ClinicalTrials.gov ID
- NCT02489500
- Acronym / sponsor ID
- VelRand / H-33808
- Conditions
- AL Amyloidosis
- Keywords
- AL Amyloidosis, Stem Cell Transplantation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment held for toxicity evaluation; then closed due to competing trial
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Boston Medical CenterUniversity, hospital or other
- Study start
- Jun 2015
- Primary completion
- 28 Apr 2017 (actual)
- Study completion
- 28 Apr 2017 (actual)
- First posted
- 3 Jul 2015 (estimated)
- Results
- Posted 6 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant.
This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.
Detailed description
This study seeks to enroll patients with AL amyloidosis who have been recommended for standard treatment with high-dose melphalan and stem cell transplant.
Standard treatment for this disease is high-dose melphalan and stem cell transplant.
The purpose of this study is to compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.
Patients enrolled in this study will receive either standard treatment with high-dose melphalan and stem cell transplant, or investigational bortezomib used in combination with standard treatment with high-dose melphalan and stem cell transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| melphalan | Active comparator | Drug: Melphalan, Drug: Neupogen, Procedure: Stem Cell Collection, Procedure: Stem cell infusionNeupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion |
| melphalan + Bortezomib | Experimental | Drug: Bortezomib, Drug: Melphalan, Drug: Neupogen, Procedure: Stem Cell Collection, Procedure: Stem cell infusionNeupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Histological diagnosis of primary systemic (AL) amyloidosis based on:
- Patients must be > 18 years of age.
- Patients must have a performance status of 0-2 by Eastern Cooperative Oncology Group (ECOG) criteria
- Patients must have left ventricular ejection fraction (LVEF) > 45% by echocardiogram within 60 days of enrollment
- Pulmonary Function Tests must show diffusing capacity of lung for carbon monoxide (DLCO) > 50%.
Key exclusion criteria (6 listed)
- Patients with recent (< 6 months) myocardial infarction, congestive heart failure, New York Heart Association (NYHA) class III/IV or arrhythmia which are refractory to medical therapy are ineligible.
- Prior chemotherapy with alkylating agent allowed only if no evidence of Myelodysplastic Dysplastic Syndrome (MDS) morphologically or cytogenetically. Total cumulative dose of oral melphalan must be < 300 mg. Patients should not have received any cytotoxic therapy < 4 weeks prior to registration and should have fully recovered from the effects of such therapy.
- Patients must not have overt multiple myeloma (>30% bone marrow plasmacytosis and, extensive (>2) lytic lesions and hypercalcemia).
- No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years.
- Patients must not be HIV positive.
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 |
|---|---|---|
| BortezomibAlso: VELCADEConditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 | Drug | melphalan + Bortezomib |
| MelphalanAlso: AlkeranConditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 | Drug | melphalan, melphalan + Bortezomib |
| NeupogenAlso: granulocyte colony-stimulating factor (G-CSF)granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days | Drug | melphalan, melphalan + Bortezomib |
| Stem Cell CollectionAlso: apheresiscollect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells | Procedure or surgery | melphalan, melphalan + Bortezomib |
| Stem cell infusionAlso: infusioninfusion of previously collected autologous stem cells | Procedure or surgery | melphalan, melphalan + Bortezomib |
Primary outcome
Number of Participants With Hematologic Response
Time frame: 6 months
Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains
3 secondary outcomes
Toxicities
Time frame: 100 days
Overall Survival
Time frame: 5 years
Number of Participants With Organ Response
Time frame: 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for AL Amyloidosis
RecruitingPhase 1
Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis
Relapsed/Refractory AL Amyloidosis
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
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
RecruitingPhase 2
Study of HBI0101 (NXC-201) CAR-T Therapy in Multiple Myeloma and Light-Chain Amyloidosis
Multiple Myeloma, Refractory to Standard Treatment, Multiple Myeloma, Relapsed
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Belantamab Mafodotin in Combination With Cyclophosphamide, Bortezomib, and Dexamethasone in Adult Participants With Newly Diagnosed Amyloid Light Chain (AL) Amyloidosis
Amyloidosis
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.
Vaishali Sanchorawala, MD principal investigator
Boston Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Bortezomib, Melphalan and Neupogen in people with AL Amyloidosis, run by Boston Medical 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?
The sponsor lists 9 inclusion and 6 exclusion criteria. 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: stopped early, as of the sponsor's last update on 10 Sep 2018. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Aug 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Hematologic Response (time frame: 6 months). 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 10 Sep 2018.