AL AmyloidosisNCT04210791
Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
An observational study of AL Amyloidosis and Plasma Cell Dyscrasia, run by Nanjing University School of Medicine at 1 site in China.
Trial summary
This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- AL Amyloidosis, Plasma Cell Dyscrasia
- How many people
- 500 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2010to 31 Dec 2030
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: All patients had an amyloid disease, which was confirmed by biopsy and documented plasma cell dyscrasia; the AL amyloidosis diagnosis and the assessment of organ involvement were based on previous consensus criteria. All patients had received autologous stem cell transplantation.
Who may be able to join
- Male or female;
- aged 18-75 years;
- Patients with newly diagnosed AL;
- Appropriate for autologous hematopoietic stem cell transplantation;
- Abnormal M protein or free light chain detected in serum and/or urine
- ECOG score 0-2 points;
- Subjects (or their legal representatives) must sign an informed consent document indicating understanding the purpose of and procedures required for the study and willingness to participate in the study.
Who cannot join
- Pregnant and breastfeeding women;
- Subjects suffering from multiple myeloma;
- hypersensitivity to any treatment drugs;
- Subjects have severe cardiovascular disease;
- Subjects have a serious physical disease and mental illnesses;
- Other conditions that researchers consider are not suitable for transplantation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
National Clinical Research Center of Kidney Diseases, Jinling Hospital
Nanjing, Jiangsu, 210016
About this study
This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.
Read the detailed description
This study is divided into two parts. In the first part, the investigators retrospectively analyze the data of patients with AL amyloidosis who treated with autologous stem cell transplantation from July 2010 to December 2019. All patients had a biopsy-proven disease by positive Congo red stain with a concomitant demonstration of plasma cell dyscrasia. Organ involvement was established according to the criteria established at the 10th International Symposium on Amyloid and Amyloidosis. The protocol of ASCT included mobilization with colony-stimulating factor alone and conditioning with high-dose melphalan 140 or 200 mg/m2. In addition to analyzing the efficacy and safety of all the patients, the investigators also analyzed the difference in efficacy between patients in different subgroups. For example, subgroups divided according to different induction treatment regimens; subgroups divided according to different plasma cell FISH data and FCM data; subgroups divided according to the degree of organ involvement, and subgroups divided according to different maintenance treatment regimens.
In the second part of the study, the investigators will conduct a prospective study to explore the best autologous stem cell transplantation treatment protocol for AL amyloidosis. The protocol includes the induction therapy with bortezomib and daratumumab before ASCT, the maintenance treatment with lenalidomide after ASCT, and the treatment options for recurrence after transplantation.
- Conditions
- AL Amyloidosis, Plasma Cell Dyscrasia
- Keywords
- AL amyloidosis, autologous stem cell transplantation, induction therapy, bortezomib, lenalidomide, ixazomib, daratumumab, melphalan
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis, Paraproteinemias
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival
the 5 years overall survival after autologous stem cell transplantation.
Measured: 5 years
progression-free survival
the 5 years progression-free survival after autologous stem cell transplantation.
Measured: 5 years
hematological complete response rate
the 1 year hematologic complete response rate after autologous stem cell transplantation.
Measured: 1 year
organ response rate
the 1 years organ response rate after autologous stem cell transplantation.
Measured: 1 year
Secondary outcomes (4)
hematological overall response rate
the 1 years hematologic overall response rate after autologous stem cell transplantation.
Measured: 1 year
the relapse rate of complete remission participants
the relapse rate of complete remission participants after autologous stem cell transplantation.
Measured: 5 years
the rate of Minimal Residual Disease-negative participants
the rate of Minimal Residual Disease-negative participants after autologous stem cell transplantation.
Measured: 5 years
the median time form stem cell transplantation to next treatment
the median time form stem cell transplantation to next chemotherapy or antibody treatment.
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 posted26 Dec 2019
- Study start1 Jul 2010
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2030 (estimated)
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 AL 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 500 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Nanjing University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhi-Hong Liu, MD, Nanjing University School of Medicine
- Investigators
- Zhihong Liu, MD, Study director, Nanjing University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04210791
- Sponsor's protocol ID
- NJCT-1901
- Status last verified
- Feb 2024
- First submitted
- 14 Dec 2019
- Last updated
- 26 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Zhang Y, Guo J, Chen W, Zhao L, Huang X. Risk factors, treatments and outcomes of patients with light chain amyloidosis who relapse after autologous stem cell transplantation. Bone Marrow Transplant. 2024 Mar;59(3):350-358. doi: 10.1038/s41409-023-02185-z. Epub 2023 Dec 26.
PubMed 38148411
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
- ClinicalTrials.gov ID
- NCT04210791
- Acronym / sponsor ID
- NJCT-1901
- Conditions
- AL Amyloidosis, Plasma Cell Dyscrasia
- Keywords
- AL amyloidosis, autologous stem cell transplantation, induction therapy, bortezomib, lenalidomide, ixazomib, daratumumab, melphalan
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Nanjing University School of MedicineUniversity, hospital or other
- Study start
- 1 Jul 2010 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 26 Dec 2019 (actual)
- 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.
This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.
Detailed description
This study is divided into two parts. In the first part, the investigators retrospectively analyze the data of patients with AL amyloidosis who treated with autologous stem cell transplantation from July 2010 to December 2019. All patients had a biopsy-proven disease by positive Congo red stain with a concomitant demonstration of plasma cell dyscrasia. Organ involvement was established according to the criteria established at the 10th International Symposium on Amyloid and Amyloidosis. The protocol of ASCT included mobilization with colony-stimulating factor alone and conditioning with high-dose melphalan 140 or 200 mg/m2. In addition to analyzing the efficacy and safety of all the patients, the investigators also analyzed the difference in efficacy between patients in different subgroups. For example, subgroups divided according to different induction treatment regimens; subgroups divided according to different plasma cell FISH data and FCM data; subgroups divided according to the degree of organ involvement, and subgroups divided according to different maintenance treatment regimens.
In the second part of the study, the investigators will conduct a prospective study to explore the best autologous stem cell transplantation treatment protocol for AL amyloidosis. The protocol includes the induction therapy with bortezomib and daratumumab before ASCT, the maintenance treatment with lenalidomide after ASCT, and the treatment options for recurrence after transplantation.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All patients had an amyloid disease, which was confirmed by biopsy and documented plasma cell dyscrasia; the AL amyloidosis diagnosis and the assessment of organ involvement were based on previous consensus criteria. All patients had received autologous stem cell transplantation.
Key inclusion criteria (7 listed)
- Male or female;
- aged 18-75 years;
- Patients with newly diagnosed AL;
- Appropriate for autologous hematopoietic stem cell transplantation;
- Abnormal M protein or free light chain detected in serum and/or urine
Key exclusion criteria (6 listed)
- Pregnant and breastfeeding women;
- Subjects suffering from multiple myeloma;
- hypersensitivity to any treatment drugs;
- Subjects have severe cardiovascular disease;
- Subjects have a serious physical disease and mental illnesses;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
overall survival
Time frame: 5 years
the 5 years overall survival after autologous stem cell transplantation.
progression-free survival
Time frame: 5 years
the 5 years progression-free survival after autologous stem cell transplantation.
hematological complete response rate
Time frame: 1 year
the 1 year hematologic complete response rate after autologous stem cell transplantation.
organ response rate
Time frame: 1 year
the 1 years organ response rate after autologous stem cell transplantation.
4 secondary outcomes
hematological overall response rate
Time frame: 1 year
the relapse rate of complete remission participants
Time frame: 5 years
the rate of Minimal Residual Disease-negative participants
Time frame: 5 years
the median time form stem cell transplantation to next treatment
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2024.
- Derived from this study: PubMed 38148411Zhang Y, Guo J, Chen W, Zhao L, Huang X. Risk factors, treatments and outcomes of patients with light chain amyloidosis who relapse after autologous stem cell transplantation. Bone Marrow Transplant. 2024 Mar;59(3):350-358. doi: 10.1038/s41409-023-02185-z. Epub 2023 Dec 26.
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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.
Zhihong Liu, MD study director
Nanjing University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AL Amyloidosis and Plasma Cell Dyscrasia, run by Nanjing University School of Medicine at 1 site in China.
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 7 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: enrolling by invitation, as of the sponsor's last update on 26 Feb 2024. 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 China. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
overall survival (time frame: 5 years). The study lists 4 primary outcomes in all. 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 NCT04210791, 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 26 Feb 2024.