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Light Chain AmyloidosisPhase 1NCT05978661

FKC288 for Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

A Phase 1 study testing FKC288 in people with Light Chain Amyloidosis, run by Nanjing University School of Medicine at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This study is a single-center exploratory clinical trial. It is estimated that 6-12 subjects will be enrolled. The "BOIN" dose escalation design is adopted. The main purpose is to evaluate the safety of FKC288 in the treatment of subjects with relapsed or refractory AL amyloidosis and explore the recommended phase II… Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Light Chain Amyloidosis
What is being tested?
FKC288
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteChina
Study dates
29 Aug 2023to Jun 2026
Phase
Phase 1
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

  • The subject must personally sign a written informed consent form approved by the ethics committee before the start of the study;
  • The subject's age is ≥18 years old and <70 years old;
  • The subject must be diagnosed with light chain amyloidosis by pathological examination, with at least one major organ involved (heart, kidney, or liver);
  • The subject with recurrent/refractory light chain amyloidosis that achieved no response with conventional treatment;
  • dFLC > 50mg/L;
  • Expected survival ≥ 12 weeks;
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  • ECOG score ≤ 2 points;
  • Female subjects with fertility should agree to practice an effective method of contraception from the day of signing the ICF until 365 days after the infusion. An effective method of contraception is defined as abstinence or contraceptive methods with an annual failure rate of <1% specified in the plan.
  • Before enrollment, the subject must have appropriate organ function and meet all the following criteria:
  • 1) Absolute neutrophil count ≥ 1.0×109/L (use of granulocyte colony-stimulating factor (G-CSF) support is allowed, but must be without supportive treatment within 7 days before the examination); 2) Platelet count ≥ 75×109/L (no transfusion support [including component transfusion] or treatments aimed at raising platelets such as thrombopoietin [TPO] should be received within 7 days before the examination); 3) Hemoglobin ≥ 9 g/dl (no transfusion support [including component transfusion] should be received within 7 days before the examination); 4) Bilirubin value ≤ 1.5× upper limit of normal (ULN) (except bile duct obstruction caused by tumor compression); 5) Creatinine clearance rate ≥ 40 ml/min; 6) ALT or AST ≤ 2.5× ULN (≤5 times the upper limit of normal in patients with liver involvement); 7) Echocardiography results indicate left ventricular ejection fraction ≥ 50% with no significant pericardial effusion; 8) NTproBNP < 1800pg/ml, TNT < 0.06ng/ml; 9) Stable coagulation function: INR ≤ 1.5, APTT ≤ 1.2× ULN (excluding tumor-related anticoagulant therapy); 10) >95% basic blood oxygen saturation in the natural indoor air environments.

Who cannot join

  • Subjects who have received any of the following treatments prior to enrollment: 1) Subjects who have received gene therapy before enrollment; 2) Subjects who have received live vaccines within 4 weeks prior to enrollment; 3) Subjects has received other interventional clinical research drugs within 12 weeks before apheresis.
  • Subjects with central metastasis or complete intestinal obstruction.
  • Subject with moderate or more severe hydrothorax and ascites which are hard to control by conventional treatment and require continuous catheter drainage.
  • With an active malignant tumor in the past 5 years, unless it is a curable tumor and has been obviously cured.
  • Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and have abnormal peripheral blood HBV DNA test results (HBV DNA test abnormality is defined as HBV DNA quantitative detection is higher than the detection center's detection lower limit or higher than the detection center's normal reference range or HBV DNA qualitative detection is positive); hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; human immunodeficiency virus (HIV) antibody positive; the cytomegalovirus (CMV) DNA positive; syphilis testing RPR positive.
  • Presence of uncontrollable active infections (excluding <CTCAE grade 2 urinary and respiratory tract infections).
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  • Severe cardiovascular diseases, including but not limited to unstable angina pectoris, myocardial infarction (within 6 months prior to screening), congestive heart failure (New York Heart Association [NYHA] classification ≥ III), and severe arrhythmias.
  • Subjects with hypertension that cannot be controlled by medication.
  • Toxicity reactions that have not been relieved to baseline or ≤ grade 1 (NCI-CTCAE version 5.0, except for hair loss and laboratory test abnormalities without clinical significance) from past treatments.
  • Major surgery within 2 weeks before enrollment, or has a surgery planned during the time the subject is expected to be infused with FKC288 or within 12 weeks after FKC288 infusion (except planned surgery under local anesthesia).
  • Subject who has a solid organ transplant.
  • Women who are pregnant or breastfeeding.
  • Subjects with previous central nervous system diseases (such as cerebral aneurysm, epilepsy, stroke, dementia, psychosis, etc.) or conscious disorders.
  • Other systemic diseases that the investigator judges as unstable, including but not limited to severe liver, kidney, or metabolic diseases that require medication.
  • Known life-threatening allergic reactions, hypersensitivity reactions, or intolerance to FKC288 cell preparations or their components.
  • Subjects judged by the investigator to have bleeding or severe thrombosis, or have inherited/acquired bleeding and severe thrombosis (including hemophilia, coagulation dysfunction, thrombocytopenia, splenomegaly, etc.), or are receiving thrombolysis or anticoagulation therapy.
  • Other situations deemed inappropriate for inclusion by the investigator.

Trial locations and contacts

1 site in 1 country.

Xianghua Huang, MD contact

86-25-80862002

hxhszb@163.com

China1 site, 1 recruiting

  • Jinling Hospital

    Nanjing, Jiangsu, 210016

    Xianghua Huang, MD, 862580862351, hxhszb@163.com

    Xianghua Huang, MD

    Recruiting

Other trials in China

About this study

Official title: A Single-Center Exploratory Study to Evaluate the Safety and Efficacy of FKC288 in Subjects With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

This study is a single-center exploratory clinical trial. It is estimated that 6-12 subjects will be enrolled. The "BOIN" dose escalation design is adopted. The main purpose is to evaluate the safety of FKC288 in the treatment of subjects with relapsed or refractory AL amyloidosis and explore the recommended phase II dose of FKC288 in the treatment of patients with relapsed/refractory systemic Light Chain (AL) amyloidosis.

Read the detailed description

A leukapheresis procedure will be performed to manufacture FKC288 chimeric antigen receptor (CAR) modified T cells. Bridging therapy is allowed between PBMC collection and lymphodepletion. Lymphodepletion with fludarabine and cyclophosphamide was performed for three consecutive days. After 1-day rest, subjects will receive a single dose infusion of FKC288 at 0.1, 0.3, 1.0, or 3.0x 10^6 CAR+ T cells/Kg. Subjects will be followed in the study for a minimum of 2 years after FKC288 infusion.

Being tested
FKC288
Keywords
AL Amyloidosis, BCMA/CD19-CAR-T cell
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.

  • FKC288Drug

    Administration of FKC288 Four dose groups of 0.1×10^6 CAR-T/kg, 0.3×10^6 CAR-T/kg, 1.0×10^6 CAR-T/kg, and 3.0×10^6 CAR-T/kg FKC288 are designed in this study. 3 to 6 subjects are expected to be enrolled in each dose group according to observed DLT.

Study groups in NCT05978661
Study groupRoleWhat they receive
Treatment armExperimentalNot described
Receives: Drug: FKC288

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The proportion of subjects with dose-limiting toxicity

    The number of participants with dose limiting toxicity in each dose group and the type of dose limiting toxicity that occurred.

    Measured: Within 28 days after FKC288 injection infusion

  2. The proportion of subjects with adverse events

    All adverse events were evaluated according to NCI-CTCAE v5.0 criteria.

    Measured: Within 24 weeks after FKC288 injection infusion

Secondary outcomes (4)

  1. Proportion of subjects achieving hematologic response

    The proportion of subjects who achieve any type of hematologic response according to the 2023 National Comprehensive Cancer Network (NCCN) systemic light-chain amyloidosis guidelines within 6 months after receiving FKC288 infusion.

    Measured: Within 6 months after FKC288 injection infusion

  2. Proportion of subjects achieving organ response

    The proportion of subjects who achieve any type of organ response according to the 2023 National Comprehensive Cancer Network (NCCN) systemic light-chain amyloidosis guidelines within 2 years after receiving FKC288 infusion.

    Measured: Within 2 years after FKC288 injection infusion

  3. Progression-free survival (PFS) of all subjects

    Time from the first FKC288 infusion to the first assessment of disease progression/relapse or death from any cause.

    Measured: Within 2 years after FKC288 injection infusion

  4. Overall survival (OS) of all subjects

    Time from the first FKC288 infusion to death from any cause.

    Measured: Within 2 years after FKC288 injection infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Aug 2023
  2. Study start29 Aug 2023
  3. Primary completionJun 2025 (estimated)
  4. Study completionJun 2026 (estimated)

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05978661
Sponsor's protocol ID
NJCT-2301
Status last verified
Nov 2023
First submitted
7 May 2023
Last updated
15 Nov 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Huang X, Wu X, Chen W, Wang W, Xu W, Guo J, Zhao J, Xu X, Wang C, Shan A, Xu J, Liu Z. B -Cell Maturation Antigen-CD19 Dual-Targeted Chimeric Antigen Receptor- T -Cell Therapy for Relapsed or Refractory AL Amyloidosis. J Am Soc Nephrol. 2026 Apr 1. doi: 10.1681/ASN.0000001096. Online ahead of print.

    PubMed 41920709

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing FKC288 in people with Light Chain Amyloidosis, 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 10 inclusion and 17 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 15 Nov 2023. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 Xianghua Huang, MD (86-25-80862002, hxhszb@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The proportion of subjects with dose-limiting toxicity (time frame: Within 28 days after FKC288 injection infusion). The study lists 2 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 NCT05978661, 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 15 Nov 2023.

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