Immunoglobulin Light-Chain AmyloidosisPhase 4NCT04612582
Comparison of BTD and BCD Based Regimens in the Treatment of AL Amyloidosis
A Phase 4 study testing Thalidomide and Cyclophosphamide in people with Immunoglobulin Light-Chain Amyloidosis, run by Guangdong Provincial People's Hospital at 1 site in China.
Trial summary
Research Objective:At present, there is no standard therapeutic regimen for monoclonal immunoglobulin light chain (AL) amyloidosis in the world. To compare the efficacy and safety of the regimens between bortezomib-thalidomide-dexamethasone (BTD) and bortezomib-cyclophosphamide-dexamethasone (BCD) in the treatment of… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Immunoglobulin Light-Chain Amyloidosis
- What is being tested?
- Thalidomide and Cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2020to 30 Jun 2023
- Phase
- Phase 4
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1.Signed the written informed consent; 2.18 years old ≤ age ≤ 80 years old, no restriction on gender; 3.AL amyloidosis was confirmed by pathological biopsy in the accumulated system or organ (kidney, heart, liver, skin), and excluded other secondary factors; 4.The proliferation of monoclonal plasma cells was confirmed by fixed electrophoresis of bone marrow or blood/urine.
Who cannot join
- Pathological biopsy showed non-AL amyloidosis;
- Abnormal proliferation of plasma cells reached the standard of multiple myeloma；
- Other hematological system tumors;
- Cushing's syndrome;
- Active hepatitis;
- 5.Pregnant or lactating women;
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Guangdong Provincial People'S Hospital
Guangzhou, Guangdong, 510080
Wenjian Wang, PhD, 0086-020-83827812-61421, wwjph@126.com
About this study
Official title: A Comparative Study of Bortezomib-Thalidomide-Dexamethason and Bortezomib-Cyclophosphamide-Dexamethason in the Treatment of Monoclonal Immunoglobulin Light Chain Amyloidosis: A Prospective Randomized Controlled Trial（BTD-CHINA-TRIAL）
Research Objective:At present, there is no standard therapeutic regimen for monoclonal immunoglobulin light chain (AL) amyloidosis in the world. To compare the efficacy and safety of the regimens between bortezomib-thalidomide-dexamethasone (BTD) and bortezomib-cyclophosphamide-dexamethasone (BCD) in the treatment of AL amyloidosis, so as to provide more clinical evidence for the standard treatment for the disease.
Research Design:This study was designed as a prospective, randomized and controlled clinical study. Patients who meet the inclusion criteria of this study will be randomized to the BTD scheme group or BCD scheme group.
Read the detailed description
Object and source:Patients meeting the inclusion criteria of AL amyloidosis are enrolled into the study. Screening failure rate ≤ 30%, follow-up dropout rate ≤ 10%.
Sample size evaluation: The complete remission rate (CR) and the very good partial remission rate (VGPR) were used as statistical indexes for hematology remission rate estimation. Referring to the previous literature, the CR and VGPR of bortezomib-thalidomide-dexamethasone (BTD) is 83%, and CR and VGPR of bortezomib-cyclophosphamide-dexamethasone (BCD) is 43%. The boundary value is 0.1, according to the level of significance level α = 0.05, test efficacy 1 - β = 0.8, using the method of effectiveness test, using pass11 software calculation to estimate the sample size to be included in the group, the expected dropout rate is 20%, the total sample size is 70, 35 in each group.
Observation index:Before and after treatment, physical examination should be carried out every month. The following test should be required. Blood pressure
, blood routine, liver function, electrolyte, 24h urine volume, 24h urine protein quantity, urine protein/creatinine ratio, blood uric acid, serum albumin, urea nitrogen, creatinine, eGFR(estimated Glomerular Filtration Rate), blood lipids, infection index, NT-proBNP (N terminal pro B type natriuretic peptide), TNT(Troponin-T), blood and urine free light chain should be detected respectively.
Quality assurance plan:The study shall be conducted in accordance with the current approved protocol and the July 1996 ICH drug clinical study management code (CPMP/ICH / 135/95) (ICH GCP), the Helsinki declaration, regulations and standard operating procedures.
Plan for missing data:(1)When the subject falls off, the researcher should contact the subject as soon as possible by means of visiting, making an appointment for follow-up, making phone calls, sending letters, etc., asking the reason, making record as much detail as possible.(2)All patients with abscission should be recorded for ITT analysis. There is no need for replacement for exfoliated patients. (3)If a patient withdraws from the study due to anaphylaxis, adverse reactions or ineffective treatment, the investigator shall take corresponding treatment measures according to the actual situation of the subject.
Statistical analysis plan:The main evaluation indexes were hematology CR and VGPR, organ function CR and VGPR. The statistical index of treatment effect is the number of patients who meet the criteria of CR and VGPR. Conclusion: if P > 0.05, H0 hypothesis cannot be rejected according to the test level of single side α = 0.05, that is to say, it cannot be judged that the treatment of light amyloidosis in BTD group is better than that in BCD group; if P ≤ 0.05, it can be considered that the treatment of light amyloidosis in BTD group is better than that in BCD group. The difference between the two groups was analyzed by chi square test or Fisher exact probability test as a secondary evaluation index (the overall survival rate and progression free survival period were observed after one year follow-up after six courses of treatment).The safety analysis set includes all cases entering the study and using at least one dose of the study drug. Chi square test or Fisher exact probability test were used to analyze the incidence of adverse events in the two groups. Chi square test or McNemar test were used to analyze the change of abnormal rate of laboratory indexes from baseline.
- Conditions
- Immunoglobulin Light-Chain Amyloidosis
- Being tested
- Thalidomide, Cyclophosphamide
- Keywords
- AL Amyloidosis, Bortezomib, cyclophosphamide, thalidomide, randomized controlled trial, prognosis, Side effect
- 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.
ThalidomideDrug
Also called Bortezomib, Dexamethason
Bortezomib (d 1, 8, 15, 22, 1.3mg /m2, subcutaneous injection); thalidomide (d 1-28 50-100mg, oral); dexamethason (d 1, 2, 8, 9, 15, 16, 22, 23, 20mg, oral/intravenous injection) as a course of treatment, the patients will complete six courses of treatment after entering the group, and the total observation time is one year after the end of treatment.
CyclophosphamideDrug
Also called Bortezomib, Dexamethason
Bortezomib (d 1, 8, 15, 22,1.3mg / m2, subcutaneous injection); cyclophosphamide (d 1, 2,900mg / m2, intravenous drip); dexamethason (d 1, 2, 8, 9,15, 16, 22, 23, 20mg, oral/intravenous injection) as a course of treatment, patients will complete six courses of treatment after entering the group, the overall observation time is one year after the end of treatment.
| Study group | Role | What they receive |
|---|---|---|
| Group1 | Experimental | Group1 is the AL amyloidosis patients who have bortezomib-thalidomide-dexamethason-based regimens for their treatment. Receives: Drug: Thalidomide |
| Group2 | Experimental | Group2 is the AL amyloidosis patients who have bortezomib-cyclophosphamide-dexamethason-based regimens for their treatment. Receives: Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic Response
According to the criteria of hematologic response of AL amyloidosis.
Measured: 1 year
Secondary outcomes (1)
Organ Response
According to the criteria of organ response of AL amyloidosis.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2020
- Study start1 Jan 2020
- Primary completion31 Jan 2023 (estimated)
- Study completion30 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Immunoglobulin Light-Chain AmyloidosisStudies of the same condition, open and under way.
- More trials run by Guangdong Provincial People's HospitalThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients who meet the entry criteria will randomly enter the BTD or BCD treatment group according to the proportion of 1:1. The random code will be generated by computer and the random envelope will be made according to the random code. Treatment group included. Each code has a corresponding random envelope. According to the time sequence of subjects meeting the entry criteria, the random number of subjects will be given from small to large. The corresponding random envelope can only be opened during grouping. If a random number falls off in the middle of use, it can no longer be used.
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangdong Provincial People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wenjian Wang, PhD, Study chair, Guangdong Provincial People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04612582
- Sponsor's protocol ID
- WJWang001
- Status last verified
- Apr 2020
- First submitted
- 15 Jun 2020
- Last updated
- 3 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Huang XH, Liu ZH. The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China. Kidney Dis (Basel). 2016 Apr;2(1):1-9. doi: 10.1159/000444287. Epub 2016 Feb 25.
PubMed 27536686Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med. 2003 Aug 7;349(6):583-96. doi: 10.1056/NEJMra023144. No abstract available.
PubMed 12904524Wechalekar AD, Gillmore JD, Hawkins PN. Systemic amyloidosis. Lancet. 2016 Jun 25;387(10038):2641-2654. doi: 10.1016/S0140-6736(15)01274-X. Epub 2015 Dec 21.
PubMed 26719234Perfetto F, Moggi-Pignone A, Livi R, Tempestini A, Bergesio F, Matucci-Cerinic M. Systemic amyloidosis: a challenge for the rheumatologist. Nat Rev Rheumatol. 2010 Jul;6(7):417-29. doi: 10.1038/nrrheum.2010.84. Epub 2010 Jun 8.
PubMed 20531382Gertz MA. Immunoglobulin light chain amyloidosis: 2016 update on diagnosis, prognosis, and treatment. Am J Hematol. 2016 Sep;91(9):947-56. doi: 10.1002/ajh.24433.
PubMed 27527836Dingli D, Tan TS, Kumar SK, Buadi FK, Dispenzieri A, Hayman SR, Lacy MQ, Gastineau DA, Hogan WJ, Gertz MA. Stem cell transplantation in patients with autonomic neuropathy due to primary (AL) amyloidosis. Neurology. 2010 Mar 16;74(11):913-8. doi: 10.1212/WNL.0b013e3181d55f4d.
PubMed 20231668Kumar S, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Colby C, Laumann K, Zeldenrust SR, Leung N, Dingli D, Greipp PR, Lust JA, Russell SJ, Kyle RA, Rajkumar SV, Gertz MA. Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements. J Clin Oncol. 2012 Mar 20;30(9):989-95. doi: 10.1200/JCO.2011.38.5724. Epub 2012 Feb 13.
PubMed 22331953Palladini G, Hegenbart U, Milani P, Kimmich C, Foli A, Ho AD, Vidus Rosin M, Albertini R, Moratti R, Merlini G, Schonland S. A staging system for renal outcome and early markers of renal response to chemotherapy in AL amyloidosis. Blood. 2014 Oct 9;124(15):2325-32. doi: 10.1182/blood-2014-04-570010. Epub 2014 Aug 12.
PubMed 25115890Palladini G, Perfetti V, Obici L, Caccialanza R, Semino A, Adami F, Cavallero G, Rustichelli R, Virga G, Merlini G. Association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis who are ineligible for stem cell transplantation. Blood. 2004 Apr 15;103(8):2936-8. doi: 10.1182/blood-2003-08-2788. Epub 2003 Dec 18.
PubMed 15070667Jaccard 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 17855669Kastritis E, Wechalekar AD, Dimopoulos MA, Merlini G, Hawkins PN, Perfetti V, Gillmore JD, Palladini G. Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis. J Clin Oncol. 2010 Feb 20;28(6):1031-7. doi: 10.1200/JCO.2009.23.8220. Epub 2010 Jan 19.
PubMed 20085941Huang X, Wang Q, Chen W, Zeng C, Chen Z, Gong D, Zhang H, Liu Z. Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment of renal AL amyloidosis: a randomized controlled trial. BMC Med. 2014 Jan 6;12:2. doi: 10.1186/1741-7015-12-2.
PubMed 24386911Cavo M, Pantani L, Pezzi A, Petrucci MT, Patriarca F, Di Raimondo F, Marzocchi G, Galli M, Montefusco V, Zamagni E, Gamberi B, Tacchetti P, Brioli A, Palumbo A, Sonneveld P. Bortezomib-thalidomide-dexamethasone (VTD) is superior to bortezomib-cyclophosphamide-dexamethasone (VCD) as induction therapy prior to autologous stem cell transplantation in multiple myeloma. Leukemia. 2015 Dec;29(12):2429-31. doi: 10.1038/leu.2015.274. Epub 2015 Oct 7. No abstract available.
PubMed 26442610Dispenzieri A, Buadi F, Kumar SK, Reeder CB, Sher T, Lacy MQ, Kyle RA, Mikhael JR, Roy V, Leung N, Grogan M, Kapoor P, Lust JA, Dingli D, Go RS, Hwa YL, Hayman SR, Fonseca R, Ailawadhi S, Bergsagel PL, Chanan-Khan A, Rajkumar SV, Russell SJ, Stewart K, Zeldenrust SR, Gertz MA. Treatment of Immunoglobulin Light Chain Amyloidosis: Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) Consensus Statement. Mayo Clin Proc. 2015 Aug;90(8):1054-81. doi: 10.1016/j.mayocp.2015.06.009.
PubMed 26250727
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Comparative Study of Bortezomib-Thalidomide-Dexamethason and Bortezomib-Cyclophosphamide-Dexamethason in the Treatment of Monoclonal Immunoglobulin Light Chain Amyloidosis: A Prospective Randomized Controlled Trial（BTD-CHINA-TRIAL）
- ClinicalTrials.gov ID
- NCT04612582
- Acronym / sponsor ID
- WJWang001
- Conditions
- Immunoglobulin Light-Chain Amyloidosis
- Keywords
- AL Amyloidosis, Bortezomib, cyclophosphamide, thalidomide, randomized controlled trial, prognosis, Side effect
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Guangdong Provincial People's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2020 (actual)
- Primary completion
- 31 Jan 2023 (estimated)
- Study completion
- 30 Jun 2023 (estimated)
- First posted
- 3 Nov 2020 (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.
Research Objective:At present, there is no standard therapeutic regimen for monoclonal immunoglobulin light chain (AL) amyloidosis in the world. To compare the efficacy and safety of the regimens between bortezomib-thalidomide-dexamethasone (BTD) and bortezomib-cyclophosphamide-dexamethasone (BCD) in the treatment of AL amyloidosis, so as to provide more clinical evidence for the standard treatment for the disease.
Research Design:This study was designed as a prospective, randomized and controlled clinical study. Patients who meet the inclusion criteria of this study will be randomized to the BTD scheme group or BCD scheme group.
Detailed description
Object and source:Patients meeting the inclusion criteria of AL amyloidosis are enrolled into the study. Screening failure rate ≤ 30%, follow-up dropout rate ≤ 10%.
Sample size evaluation: The complete remission rate (CR) and the very good partial remission rate (VGPR) were used as statistical indexes for hematology remission rate estimation. Referring to the previous literature, the CR and VGPR of bortezomib-thalidomide-dexamethasone (BTD) is 83%, and CR and VGPR of bortezomib-cyclophosphamide-dexamethasone (BCD) is 43%. The boundary value is 0.1, according to the level of significance level α = 0.05, test efficacy 1 - β = 0.8, using the method of effectiveness test, using pass11 software calculation to estimate the sample size to be included in the group, the expected dropout rate is 20%, the total sample size is 70, 35 in each group.
Observation index:Before and after treatment, physical examination should be carried out every month. The following test should be required. Blood pressure
, blood routine, liver function, electrolyte, 24h urine volume, 24h urine protein quantity, urine protein/creatinine ratio, blood uric acid, serum albumin, urea nitrogen, creatinine, eGFR(estimated Glomerular Filtration Rate), blood lipids, infection index, NT-proBNP (N terminal pro B type natriuretic peptide), TNT(Troponin-T), blood and urine free light chain should be detected respectively.
Quality assurance plan:The study shall be conducted in accordance with the current approved protocol and the July 1996 ICH drug clinical study management code (CPMP/ICH / 135/95) (ICH GCP), the Helsinki declaration, regulations and standard operating procedures.
Plan for missing data:(1)When the subject falls off, the researcher should contact the subject as soon as possible by means of visiting, making an appointment for follow-up, making phone calls, sending letters, etc., asking the reason, making record as much detail as possible.(2)All patients with abscission should be recorded for ITT analysis. There is no need for replacement for exfoliated patients. (3)If a patient withdraws from the study due to anaphylaxis, adverse reactions or ineffective treatment, the investigator shall take corresponding treatment measures according to the actual situation of the subject.
Statistical analysis plan:The main evaluation indexes were hematology CR and VGPR, organ function CR and VGPR. The statistical index of treatment effect is the number of patients who meet the criteria of CR and VGPR. Conclusion: if P > 0.05, H0 hypothesis cannot be rejected according to the test level of single side α = 0.05, that is to say, it cannot be judged that the treatment of light amyloidosis in BTD group is better than that in BCD group; if P ≤ 0.05, it can be considered that the treatment of light amyloidosis in BTD group is better than that in BCD group. The difference between the two groups was analyzed by chi square test or Fisher exact probability test as a secondary evaluation index (the overall survival rate and progression free survival period were observed after one year follow-up after six courses of treatment).The safety analysis set includes all cases entering the study and using at least one dose of the study drug. Chi square test or Fisher exact probability test were used to analyze the incidence of adverse events in the two groups. Chi square test or McNemar test were used to analyze the change of abnormal rate of laboratory indexes from baseline.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients who meet the entry criteria will randomly enter the BTD or BCD treatment group according to the proportion of 1:1. The random code will be generated by computer and the random envelope will be made according to the random code. Treatment group included. Each code has a corresponding random envelope. According to the time sequence of subjects meeting the entry criteria, the random number of subjects will be given from small to large. The corresponding random envelope can only be opened during grouping. If a random number falls off in the middle of use, it can no longer be used.
| Arm | Type | Receives |
|---|---|---|
| Group1 | Experimental | Drug: ThalidomideGroup1 is the AL amyloidosis patients who have bortezomib-thalidomide-dexamethason-based regimens for their treatment. |
| Group2 | Experimental | Drug: CyclophosphamideGroup2 is the AL amyloidosis patients who have bortezomib-cyclophosphamide-dexamethason-based regimens for their treatment. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 1.Signed the written informed consent; 2.18 years old ≤ age ≤ 80 years old, no restriction on gender; 3.AL amyloidosis was confirmed by pathological biopsy in the accumulated system or organ (kidney, heart, liver, skin), and excluded other secondary factors; 4.The proliferation of monoclonal plasma cells was confirmed by fixed electrophoresis of bone marrow or blood/urine.
Key exclusion criteria (6 listed)
- Pathological biopsy showed non-AL amyloidosis;
- Abnormal proliferation of plasma cells reached the standard of multiple myeloma；
- Other hematological system tumors;
- Cushing's syndrome;
- Active hepatitis;
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 |
|---|---|---|
| ThalidomideAlso: Bortezomib, DexamethasonBortezomib (d 1, 8, 15, 22, 1.3mg /m2, subcutaneous injection); thalidomide (d 1-28 50-100mg, oral); dexamethason (d 1, 2, 8, 9, 15, 16, 22, 23, 20mg, oral/intravenous injection) as a course of treatment, the patients will complete six courses of treatment after entering the group, and the total observation time is one year after the end of treatment. | Drug | Group1 |
| CyclophosphamideAlso: Bortezomib, DexamethasonBortezomib (d 1, 8, 15, 22,1.3mg / m2, subcutaneous injection); cyclophosphamide (d 1, 2,900mg / m2, intravenous drip); dexamethason (d 1, 2, 8, 9,15, 16, 22, 23, 20mg, oral/intravenous injection) as a course of treatment, patients will complete six courses of treatment after entering the group, the overall observation time is one year after the end of treatment. | Drug | Group2 |
Primary outcome
Hematologic Response
Time frame: 1 year
According to the criteria of hematologic response of AL amyloidosis.
1 secondary outcome
Organ Response
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2020.
- Background: PubMed 27536686Huang XH, Liu ZH. The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China. Kidney Dis (Basel). 2016 Apr;2(1):1-9. doi: 10.1159/000444287. Epub 2016 Feb 25.
- Background: PubMed 12904524Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med. 2003 Aug 7;349(6):583-96. doi: 10.1056/NEJMra023144. No abstract available.
- Background: PubMed 26719234Wechalekar AD, Gillmore JD, Hawkins PN. Systemic amyloidosis. Lancet. 2016 Jun 25;387(10038):2641-2654. doi: 10.1016/S0140-6736(15)01274-X. Epub 2015 Dec 21.
- Background: PubMed 20531382Perfetto F, Moggi-Pignone A, Livi R, Tempestini A, Bergesio F, Matucci-Cerinic M. Systemic amyloidosis: a challenge for the rheumatologist. Nat Rev Rheumatol. 2010 Jul;6(7):417-29. doi: 10.1038/nrrheum.2010.84. Epub 2010 Jun 8.
- Background: PubMed 27527836Gertz MA. Immunoglobulin light chain amyloidosis: 2016 update on diagnosis, prognosis, and treatment. Am J Hematol. 2016 Sep;91(9):947-56. doi: 10.1002/ajh.24433.
- Background: PubMed 20231668Dingli D, Tan TS, Kumar SK, Buadi FK, Dispenzieri A, Hayman SR, Lacy MQ, Gastineau DA, Hogan WJ, Gertz MA. Stem cell transplantation in patients with autonomic neuropathy due to primary (AL) amyloidosis. Neurology. 2010 Mar 16;74(11):913-8. doi: 10.1212/WNL.0b013e3181d55f4d.
- Background: PubMed 22331953Kumar S, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Colby C, Laumann K, Zeldenrust SR, Leung N, Dingli D, Greipp PR, Lust JA, Russell SJ, Kyle RA, Rajkumar SV, Gertz MA. Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements. J Clin Oncol. 2012 Mar 20;30(9):989-95. doi: 10.1200/JCO.2011.38.5724. Epub 2012 Feb 13.
- Background: PubMed 25115890Palladini G, Hegenbart U, Milani P, Kimmich C, Foli A, Ho AD, Vidus Rosin M, Albertini R, Moratti R, Merlini G, Schonland S. A staging system for renal outcome and early markers of renal response to chemotherapy in AL amyloidosis. Blood. 2014 Oct 9;124(15):2325-32. doi: 10.1182/blood-2014-04-570010. Epub 2014 Aug 12.
- Background: PubMed 15070667Palladini G, Perfetti V, Obici L, Caccialanza R, Semino A, Adami F, Cavallero G, Rustichelli R, Virga G, Merlini G. Association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis who are ineligible for stem cell transplantation. Blood. 2004 Apr 15;103(8):2936-8. doi: 10.1182/blood-2003-08-2788. Epub 2003 Dec 18.
- Background: 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.
- Background: PubMed 20085941Kastritis E, Wechalekar AD, Dimopoulos MA, Merlini G, Hawkins PN, Perfetti V, Gillmore JD, Palladini G. Bortezomib with or without dexamethasone in primary systemic (light chain) amyloidosis. J Clin Oncol. 2010 Feb 20;28(6):1031-7. doi: 10.1200/JCO.2009.23.8220. Epub 2010 Jan 19.
- Background: PubMed 24386911Huang X, Wang Q, Chen W, Zeng C, Chen Z, Gong D, Zhang H, Liu Z. Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment of renal AL amyloidosis: a randomized controlled trial. BMC Med. 2014 Jan 6;12:2. doi: 10.1186/1741-7015-12-2.
- Individual participant dataSharing planned: No
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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.
Wenjian Wang, PhD study chair
Guangdong Provincial People's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Thalidomide and Cyclophosphamide in people with Immunoglobulin Light-Chain Amyloidosis, run by Guangdong Provincial People's Hospital 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 1 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: status not confirmed, as of the sponsor's last update on 3 Nov 2020. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wenjian Wang, PhD (0086-020-83827812-61421, wwjph@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematologic Response (time frame: 1 year). 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 NCT04612582, which also lists 14 linked publications.
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 3 Nov 2020.