In short
Clinical trials are studying ALLOGENEIC UMBILICAL CORD BLOOD-DERIVED NATURAL KILLER CELLS, EX VIVO EXPANDED with rituximab in adults with hard-to-treat rheumatic diseases. These studies look at safety, tolerability, and early benefit after lymphodepletion. The target conditions include systemic sclerosis, rheumatoid arthritis, idiopathic inflammatory myopathies, and Sjögren’s disease.
Key points
- One Phase 2 clinical trial is studying ALLOGENEIC UMBILICAL CORD BLOOD-DERIVED NATURAL KILLER CELLS, EX VIVO EXPANDED with rituximab in adults with relapsing B-cell dependent rheumatologic diseases. The target conditions are systemic sclerosis, rheumatoid arthritis, idiopathic inflammatory myopathies, and Sjögren’s disease. The study is focused on safety, tolerability, and early benefit after cyclophosphamide and fludarabine are used for lymphodepletion. The main safety measure is dose-limiting toxicities, and disease-specific outcomes are checked at Week 52. The trial plans to use response scores such as TIS, rCRISS, DAS28-ESR, and ClinESSDAI to judge possible benefit.
Trial overview
This article describes one authorised interventional study of ALLOGENEIC UMBILICAL CORD BLOOD-DERIVED NATURAL KILLER CELLS, EX VIVO EXPANDED given with rituximab in adults with relapsing forms of B-cell dependent rheumatologic diseases.1 The study is designed to assess safety, tolerability, and early signs of benefit after lymphodepletion with cyclophosphamide and fludarabine.1
Who is being studied
The trial includes adult subjects with hard-to-treat rheumatic diseases.1 The conditions listed are systemic sclerosis (SSc), rheumatoid arthritis (RA), idiopathic inflammatory myopathies (IIMs), and Sjögren’s disease (SjD).1
The study summary says these are relapsing forms of B-cell dependent rheumatologic diseases.1 In simple terms, this means the diseases can flare up again after improvement, and the study focuses on illnesses where B cells are an important part of the disease process.1
Treatment plan in the trial
The study treatment is given with rituximab, and the trial also uses cyclophosphamide and fludarabine before the main study treatment.1 This before-treatment step is called lymphodepletion, which means lowering certain immune cells first.1
The intervention list shows cyclophosphamide and fludarabine as intravenous infusions, rituximab as an intravenous infusion, and AB-101 as an intravenous treatment.1 The source also names the study product as AlloNK®, which is the trial name used in the study summary.1
What the trial measures
The main safety goal is to find dose-limiting toxicities, meaning side effects that may stop the dose from being increased.1 The trial uses a 3+3 design to help determine the MTD (maximum tolerated dose) and/or MAD (maximum administered dose).1
The main disease response measures are checked at Week 52.1 For idiopathic inflammatory myopathies, the endpoint is TIS ≥ 60; for systemic sclerosis, it is rCRISS ≥ 50; for rheumatoid arthritis, it is DAS28-ESR < 2.6; and for Sjögren’s disease, it is an improvement of at least 4 points in ClinESSDAI from baseline.1
These scores are used to measure whether the disease is getting better.1 They are different tools for different diseases, so the study can judge benefit in a way that matches each condition.1
Trial phase and size
This is a Phase 2 study.1 Phase 2 trials usually look at both safety and early benefit in a group of patients who have the disease being studied.1
The planned enrollment is 92 participants.1 The status is listed as authorised.1
What patients should know
This study is not a general review of the treatment; it is a clinical trial focused on a specific patient group with difficult rheumatic diseases.1 The main question is whether the study approach can be given safely and whether it may help symptoms and disease activity.1
The trial uses several disease-specific outcome measures, which helps researchers look at benefit in a more precise way for each illness.1 Based on the source data, the study is centered on adult patients with relapsing B-cell dependent rheumatologic disease rather than on healthy volunteers.1