Primary Systemic Amyloidosis (AL)Phase 3NCT00344526
Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis
A Phase 3 study testing Melphalan, Dexamethasone and Autologous stem cell transplantation in people with Primary Systemic Amyloidosis (AL), run by University Hospital, Limoges at 8 sites in France.
Trial summary
AL amyloidosis is caused by a clonal plasma cell dyscrasia and characterized by progressive deposition of amyloid fibrils derived from monoclonal Ig light chains, leading to multisystem organ failure and death. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Primary Systemic Amyloidosis (AL)
- What is being tested?
- Melphalan, Dexamethasone and Autologous stem cell transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 100 participants
- Where
- 8 sitesFrance
- Study dates
- Jan 2000to Jun 2006
- Phase
- Phase 3
- 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
Who may be able to join
- below 70 years of age
- biopsy proven systemic AL amyloidosis
- no more than 2 prior courses of chemotherapy
- ECOG performance status < 3
- Informed written consent
Who cannot join
- localized amyloidosis
- HIV seropositivity
- previous myelodysplasia
- concomitant serious disease
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
France8 sites
Service des Maladies du Sang
Lille
Service d'Hématologie et de Thérapie cellulaire
Limoges
Service d'hématologie clinique
Nantes
Service d'hématologie Clinique, Groupe Hospitalier Pitié-Salpétrière
Paris
Service d'hématologie clinique, Hôpital Necker
Paris
Service d'immuno-hématologie, Hôpital Saint-Louis
Paris
Service d'hématologie
Toulouse
Hématologie Clinique
Tours
About this study
Official title: Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients With AL (Primary)Amyloidosis. A Prospective Randomized Trial .
AL amyloidosis is caused by a clonal plasma cell dyscrasia and characterized by progressive deposition of amyloid fibrils derived from monoclonal Ig light chains, leading to multisystem organ failure and death. The prognosis for AL amyloidosis with conventional treatment remains poor, Autologous stem cell transplantation (ASCT) for AL amyloidosis produces high hematologic and organ responses. However, treatment-related mortality remains high and reported series are subject to selection bias.
Read the detailed description
A prospective randomized trial was conducted to compare in AL amyloidosis ASCT (melphalan 140 or 200 mg/m2 depending on age and clinical status supported with ASCT collected with G-CSF alone) and the oral regimen M-Dex (melphalan 10 mg/m2 and dexamethasone 40 mg for 4 days each months up to 18 months). The objectives were to compare survival and hematologic and clinical responses.
- Conditions
- Primary Systemic Amyloidosis (AL)
- Being tested
- Melphalan, Dexamethasone, Autologous stem cell transplantation
- Keywords
- AL amyloidosis, Autologous stem cell transplantation, Melphalan, Dexamethasone
- 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.
MelphalanDrug
DexamethasoneDrug
Autologous stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
survival
Secondary outcomes (2)
hematologic responses
clinical responses
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2006 (estimated)
- Study startJan 2000
- Study completionJun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Systemic Amyloidosis (AL)Studies of the same condition, open and under way.
- More trials run by University Hospital, LimogesThe 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
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants
Sponsor and investigators
- Lead sponsor
- University Hospital, Limoges
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, France
- Investigators
- Arnaud Jaccard, MD, Principal investigator, CH Limoges
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00344526
- Sponsor's protocol ID
- I00001
- Status last verified
- Jun 2007
- First submitted
- 22 Jun 2006
- Last updated
- 28 Jun 2007
- Results posted
- No
Publications
Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O, Recher C, Asli B, Lioure B, Royer B, Jardin F, Bridoux F, Grosbois B, Jaubert J, Piette JC, Ronco P, Quet F, Cogne M, Fermand JP; Myelome Autogreffe (MAG) and Intergroupe Francophone du Myelome (IFM) Intergroup. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2007 Sep 13;357(11):1083-93. doi: 10.1056/NEJMoa070484.
PubMed 17855669
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients With AL (Primary)Amyloidosis. A Prospective Randomized Trial .
- ClinicalTrials.gov ID
- NCT00344526
- Acronym / sponsor ID
- I00001
- Conditions
- Primary Systemic Amyloidosis (AL)
- Keywords
- AL amyloidosis, Autologous stem cell transplantation, Melphalan, Dexamethasone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants
- Lead sponsor
- University Hospital, LimogesUniversity, hospital or other
- Collaborators
- Ministry of Health, France
- Study start
- Jan 2000
- Study completion
- Jun 2006 (actual)
- First posted
- 26 Jun 2006 (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.
AL amyloidosis is caused by a clonal plasma cell dyscrasia and characterized by progressive deposition of amyloid fibrils derived from monoclonal Ig light chains, leading to multisystem organ failure and death. The prognosis for AL amyloidosis with conventional treatment remains poor, Autologous stem cell transplantation (ASCT) for AL amyloidosis produces high hematologic and organ responses. However, treatment-related mortality remains high and reported series are subject to selection bias.
Detailed description
A prospective randomized trial was conducted to compare in AL amyloidosis ASCT (melphalan 140 or 200 mg/m2 depending on age and clinical status supported with ASCT collected with G-CSF alone) and the oral regimen M-Dex (melphalan 10 mg/m2 and dexamethasone 40 mg for 4 days each months up to 18 months). The objectives were to compare survival and hematologic and clinical responses.
Study design
- Primary purpose
- Treatment
- Allocation
- 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
Key inclusion criteria (5 listed)
- below 70 years of age
- biopsy proven systemic AL amyloidosis
- no more than 2 prior courses of chemotherapy
- ECOG performance status < 3
- Informed written consent
Key exclusion criteria (4 listed)
- localized amyloidosis
- HIV seropositivity
- previous myelodysplasia
- concomitant serious disease
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 |
|---|---|---|
| Melphalan | Drug | – |
| Dexamethasone | Drug | – |
| Autologous stem cell transplantation | Procedure or surgery | – |
Primary outcome
survival
2 secondary outcomes
hematologic responses
clinical responses
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 28 Jun 2007.
- Derived from this study: PubMed 17855669Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O, Recher C, Asli B, Lioure B, Royer B, Jardin F, Bridoux F, Grosbois B, Jaubert J, Piette JC, Ronco P, Quet F, Cogne M, Fermand JP; Myelome Autogreffe (MAG) and Intergroupe Francophone du Myelome (IFM) Intergroup. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2007 Sep 13;357(11):1083-93. doi: 10.1056/NEJMoa070484.
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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.
Arnaud Jaccard, MD principal investigator
CH Limoges
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Melphalan, Dexamethasone and Autologous stem cell transplantation in people with Primary Systemic Amyloidosis (AL), run by University Hospital, Limoges at 8 sites in France.
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 5 inclusion and 4 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: finished, as of the sponsor's last update on 28 Jun 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in France. 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?
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?
survival. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00344526, which also lists 1 linked publication.
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 28 Jun 2007.