Clinical Research Directory

Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders

The goal of Safety Lead-In is to confirm the safety of tafasitamab when given to patients with SSc, SLE, and LN. The goal of Phase 1 is to find the recommended dose of AD-PluReceptor-NK cells in combination with tafasitamab and lymphodepleting chemotherapy that can be given to patients with the disease. The goal of Phase 2 is to learn if the dose of AD-PluReceptor-NK cells found in Phase 1 in combination with tafasitamab and lymphodepleting chemotherapy can help to control the disease.

A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease

This is a first-in-human, single-arm, open-label study evaluating the safety, tolerability, and preliminary efficacy of ALLO-329 in adults with autoimmune diseases: systemic lupus erythematosus (SLE) with and without renal involvement, idiopathic inflammatory myopathy (IIM), and systemic sclerosis (SSc).The purpose of this trial is to evaluate the safety and tolerability of ALLO-329, an allogeneic anti-CD19, anti-CD70 dual chimeric antigen receptor (CAR) T cell therapy, in adults with autoimmune disorders, provide initial evidence of biological activity and clinical response to the treatment and determine the recommended Phase 2 regimen (RP2R).

A Study of AZD0120 in Autoimmune Diseases

This trial is a Phase 1b/2, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA).

A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2)

This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with autoimmune diseases.

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease

An exploratory clinical study of the safety and efficacy of YTS109 cell injection in subjects with recurrent/refractory autoimmune disease

Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell IV Infusion for Systemic Sclerosis

This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Systemic Sclerosis

Hyaluronidase for Sclerodactyly in Systemic Sclerosis Trial

Translational studies have demonstrated reduced hyaluronidase activity in the skin of patients with systemic sclerosis. It is thought this may contribute to the progressive fibrosis seen in this disease. Several studies have demonstrated that exogenous hyaluronidase is very effective at improving systemic sclerosis associated microstomia. Therefore, this study aims to explore hyaluronidase for systemic sclerosis associated sclerodactyly.

RESET-SSc: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T Cell Therapy, in Subjects With Systemic Sclerosis

RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis

Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis

This is a 52 week, single center, randomized, double-blind, placebo-controlled study. After patients maintain a stable dose of Mycophenolate Mofetil (MMF) for at least 1 month, they will be randomized to treatment with either Belimumab \& Rituximab or placebo.Patients in both groups will be on background MMF for the entirety of the study. Belimumab will be administered subcutaneously and Rituximab intravenously. Placebo injections and infusions will be of normal saline. Randomization will be done in a 2:1 manner to favor the treatment group. It is hypothesized that that Rituximab and Belimumab combination therapy with Mycophenolate Mofetil background therapy will improve fibrosis in SSc skin when compared to treatment with placebo and Mycophenolate Mofetil in a group of patients with early dcSSc.

A Study to Test Whether Nerandomilast Helps People With Systemic Sclerosis

Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open to adults who are at least 18 years old and have systemic sclerosis (SSc). People can join the study if they have limited or diffuse cutaneous SSc with disease onset within 7 years of the first non-Raynaud's symptom. The purpose of this study is to find out whether a medicine called nerandomilast helps people with systemic sclerosis. This study also aims to find out how well nerandomilast is tolerated in people with systemic sclerosis. Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take the tablets twice a day. Participants are in the study for 1 to about 4 years. During this time, they visit the study site regularly and get phone calls from the site staff. During study visits participants regularly have blood samples taken and doctors check changes in skin thickening, lung function, and internal organs, overall health and the safety and tolerability of study treatment in people with SSc. The results are compared between the groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

A Study of BMS-986553 in Participants With Refractory Autoimmune Diseases

The purpose of this study is to evaluate the safety and tolerability of BMS-986553, CD19 in vivo Chimeric Antigen Receptor (CAR) T cells, in participants with refractory autoimmune diseases including systemic lupus erythematosus (SLE), idiopathic inflammatory myopathies (IIM), systemic sclerosis (SSc), rheumatoid arthritis (RA), and Sjögren's disease (SjD).

C-CAR168, an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy, in Participants With Systemic Sclerosis (SSc) Refractory to Standard Therapy

This Phase 1b, multicenter, open-label study will evaluate the safety and efficacy of a single infusion of autologous anti-CD20/BCMA chimeric antigen receptor T cells (C-CAR168) following lymphodepleting chemotherapy in participants with refractory Systemic Sclerosis (SSc) who are not responding to standard therapy. Approximately 6-12 participants will undergo leukapheresis, lymphodepletion with fludarabine and cyclophosphamide, and infusion of C-CAR168. Participants will be followed for 24 months (2 years) to evaluate safety, CAR T-cell persistence, pharmacokinetics/pharmacodynamics, biomarkers, and preliminary efficacy results. Long-term safety follow-up for gene therapy-related events will continue for up to 15 years following CAR T-cell infusion.

A Trial in Healthy Adult Participants and Adults With Autoimmune Disease to Test How HBM7020 is Tolerated and Absorbed in the Body

This first-in-human study evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of HBM7020. The study will enroll healthy participants at low doses, followed by participants with moderate to severe autoimmune diseases with predominant B-cell involvement. Eligible participants include patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), Sjögren's disease (SjD), and rheumatoid arthritis (RA).

A Study to Learn About a Medicine Called PF-08154225 in Participants With Autoimmune Diseases

The purpose of this study is to learn about the safety and effects of the study medication called PF-08154225 for the potential treatment of autoimmune diseases. An autoimmune disease is a condition that makes a person's immune system attack its healthy cells by mistake. This study is particularly looking at autoimmune diseases called as Systemic Lupus Erythematosus (SLE), Rheumatoid Arthritis (RA), Idiopathic Inflammatory Myositis (IIM) or Systemic Sclerosis (SSc). This study is divided into 3 parts: Part 1a, Part 1b and Part 2. Parts 1a and 1b are seeking participants with Systemic Lupus Erythematosus (SLE) or Rheumatoid Arthritis and Part 2 with also Idiopathic Inflammatory Myositis (IIM) or Systemic Sclerosis (SSc). Participants can take part only in one part of the study. All participants in this study will receive PF-08154225 at the study clinic. In Part 1a participants will receive single administration of PF-08154225 after which they will be observed at the study clinic during regular visits through week 16 or longer. In Part 1b the participants will receive multiple administration of PF-08154225 after which they will be monitored in similar a manner as in Part 1a through week 24 or longer. In Part 2 participants will receive multiple administrations of PF-08154225. After the last injection they will be monitored for safety through week 52 or longer.

Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)

The purpose of this study is to evaluate the efficacy and safety of treatment with subcutaneous anifrolumab versus placebo in adult participants with systemic sclerosis. The target population for this study includes patients who meet the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification for systemic sclerosis, either limited or diffuse cutaneous subsets, with a disease duration of less than 6 years from first non-Raynaud's phenomenon symptom.

Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease

The goal of this pilot study is to assess the feasibility of a larger study on the efficacy of mycophenolate mofetil in people diagnosed with systemic sclerosis with mild lung involvement. Participants will be recruited over 12 months at 3 academic centers and assigned randomly to receive either mycophenolate mofetil or placebo, a look-alike substance that contains no active drug, for 96 weeks.

A Study of KITE-363 in Participants With Refractory Autoimmune Diseases

This study will have two Phases: Phase 1a and Phase 1b. The goal of this clinical study is to learn more about the study drug KITE-363, to establish dosing, tolerability, safety, and preliminary efficacy of KITE-363 in participants with refractory autoimmune diseases. The primary objectives of this study are: Phase 1a: To evaluate the safety and tolerability of KITE-363 in participants with autoimmune disease. To determine the recommended dose for Phase 1b. Phase 1b: To evaluate the safety and efficacy of KITE-363 in participants with autoimmune disease.

A Study to Compare the Efficacy and Safety of BMS-986353 (Zolacabtagene- Autoleucel / Zola-cel), CD19-CAR T Cells, Versus Standard of Care in Participants With Active Systemic Sclerosis

The purpose of this study is to compare the efficacy and safety of BMS-986353 versus standard of care in participants with active Systemic Sclerosis

Hyperpolarized Xenon MRI in Adults With Pulmonary Hypertension in Interstitial Lung Disease and Systemic Sclerosis

This study aims to evaluate a noninvasive magnetic resonance imaging technique using hyperpolarized xenon gas to measure lung function in adults with interstitial lung disease, pulmonary hypertension, and systemic sclerosis. Researchers will assess whether MRI measurements correlate with clinical measures of lung and vascular function.

Subcutaneous Injection of Sodium Thiosulfate for Ectopic Calcifications or Ossifications. A Pilot Study

Ectopic soft tissue calcifications or ossifications can complicate the course of numerous diseases; most of them are rare or very rare. Even if the clinical, radiological and pathological presentation of ectopic calcifications and ossifications are different, the same hypotheses are discussed considering their hypothetical pathophysiology. Indeed, high calcium phosphate product, local cellular lesions and abnormal transdifferentiation of mesenchymal cells are regularly evoked when pathophysiology of such calcifications or ossifications are discussed. Apart from several case reports that have not been confirmed so far, no medical treatments are available, leading to significant pain and impairment of quality of life for patients. Therefore, only surgical treatment can be proposed when the volume or the consequences of these calcifications/ossifications become too important. Sodium thiosulfate (STS) is currently used as a cyanide poisoning antagonist and a chemoprotectant against adverse effects of several chemotherapies such as Cisplatin. Numerous case reports and several studies have revealed the potential interest of STS in the treatment of uremic induced vascular or soft tissues calcifications. Recently, our group has developed an expertise in the use of STS for the treatment of ectopic soft tissue calcifications or ossifications. Considering these promising preliminary data, and their limits, we developed a strategy to treat soft tissue calcifications or ossifications based on a local administration of STS. The first results of this therapeutic strategy are highly promising and the local or systemic safety is satisfactory so far. These preliminary data also reported by others deserve to be confirmed in a prospective study. We propose in this project to conduct a prospective open controlled phase II trial in order to assess the efficacy and the safety of intralesional administration of STS for the treatment of calcifications secondary to dermatomyositis or systemic sclerosis and ectopic ossifications secondary to pseudo-hypoparathyroidism 1a type (PHP1A/iPPSD2) (inactivating parathyroid hormone / parathyroid-hormone-related peptid (PTH/PTHrP) signalling disorder).

Evaluating Tools to Communicate Scleroderma Research Results to Patients - Trial #1

Sharing research results with patients is required by ethical regulations. Yet, most researchers do not share results from their studies with patients. The investigators plan to conduct a series of randomized controlled trials among people with scleroderma, a rare autoimmune disease, in a large international cohort, to identify the most effective methods for communicating study results with patients. The first trial in the series will compare a research dissemination tool (infographic) against a plain-language summary comparator. Participants will be randomly assigned to receive the dissemination tool or comparator. Study participants will rate communication tools for (1) information completeness; (2) understandability; and (3) ease of use of format. Our results can be used by researchers and patient organizations who disseminate research results so that they can tailor the way they disseminate results to patient needs.

KYSA-5: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis

A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects with Systemic Sclerosis

Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis

The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.