CLINICAL TRIALS

KYSA-5: A Phase 1/2, Open-Label, Multicentre Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Subjects with Systemic Sclerosis  

Sponsor: Kyverna Therapeutics

SSc is an immune-mediated rheumatic disease that is characterized by fibrosis of the skin and internal organs and vasculopathy. B-cells play a role in SSc, and the disease is characterized by the presence of autoantibodies such as anti-Scl-70 and anti-RNAP III antibodies. CD19-targeted chimeric antigen receptor (CAR) T-cells harness the ability of cytotoxic T-cells to directly and specifically lyse target cells to effectively deplete B-cells in the circulation and in lymphoid and potentially non-lymphoid tissues. KYV-101, a fully human anti-CD19 CAR T-cell therapy, will be investigated in adult subjects with systemic sclerosis.

Study Contact: Kyverna Therapeutics, Inc.

Phone Number: 510-925-2484

Email: Clinicaltrials@kyvernatx.com

Participating Site: 

Stanford University Medical Center

Dr. Lorinda Chung

A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease (BLISSc-ILD) 

Sponsor: GlaxoSmithKline

This study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).

Participating Sites:

-Los Angeles, California, United States, 90045

Contact: US GSK Clinical Trials Call Center    877-379-3718    GSKClinicalSupportHD@gsk.com   

Principal Investigator: Daniel Furst         

-Los Angeles, California, United States, 90095

Contact: US GSK Clinical Trials Call Center    877-379-3718    GSKClinicalSupportHD@gsk.com   

Principal Investigator: Elizabeth Volkmann         

-Los Angeles, California, United States, 90095

Contact: US GSK Clinical Trials Call Center    877-379-3718    GSKClinicalSupportHD@gsk.com   

Principal Investigator: Suzanne Kafaja         

-Upland, California, United States, 91786

Contact: US GSK Clinical Trials Call Center    877-379-3718    GSKClinicalSupportHD@gsk.com   

Principal Investigator: Ebrahim Sadeghi-Najafabadi       

Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD) Sponsor: aTyr Pharma, Inc.

We are testing whether an investigational drug, called efzofitimod, can improve (or be an effective form of treatment for) the skin and lung effects caused by systemic sclerosis-related interstitial lung disease (SSc-ILD)This is a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. 

The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20.

What will be involved? Throughout the study, you will:

• Attend a total of 9 visits to the study center

• Complete questionnaires to let us know how you are feeling

• Undergo monthly clinic visits where you will have physical exams, lung function tests, skin biopsies (only one at the beginning of the study and then at 3 months) and blood tests during these visits.

There will be no charge for any study-related treatment or procedures. Travel reimbursement for attending study visits will be provided. Please contact the study team for more information

Participating Sites: 

-Los Angeles, California, United States, 90024

Contact    877-689-4494    SScILD@cssienroll.com   

-San Diego, California, United States, 92093

Contact    877-689-4494    SScILD@cssienroll.com

Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Systemic Sclerosis Associated With Interstitial Lung Disease (SSc-ILD) (MK-7240-007) (ATHENA-SSc-ILD)

Sponsor: Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) 

PRA023 has previously been given to healthy adults and adults with inflammatory bowel disease. However, this is the first study of PRA023 in patients with Systemic Sclerosis and Interstitial Lung Disease. The study seeks to understand whether PRA023 can slow or halt disease in the lungs and improve quality of life. The study will also examine the potential benefit for skin involvement and overall Systemic Sclerosis disease severity. Key study requirements are: • Adult patients 18 years of age or older • Diagnosis of the diffuse form of scleroderma within 5 years • Interstitial lung disease, a common form of lung disease associated with inflammation and scarring • Patients who complete the study are offered access to continued treatment For More Information, This Trial is Posted on the Following Public Website: ClinicalTrials.gov Identifier: NCT05270668 

Prometheus Biosciences Selected Participating Sites: 

Contact: Prometheus Biosciences Call Center at 855-422-4300  

-Los Angeles, California, United States, 90045

-Los Angeles, California, United States, 90048-1804

-Los Angeles, California, United States, 90095

Investigator: Dr. Elizabeth Volkmann

Study Coordinator: Nancy R. Lopez Phone: 310-794-1638 

Email: nancyrlopez@mednet.ucla.edu

-Palo Alto, California, United States, 94304 

Investigator: Dr. Lorinda Chung

Study Coordinator: Kathryn Jee Email: kjee@stanford.edu

A Study in People With Systemic Sclerosis to Test Whether Avenciguat (BI 685509) Has an Effect on Lung Function and Other Systemic Sclerosis Symptoms (VITALISScE™) 

Sponsor: Boehringer Ingelheim

This study is open to adults aged 18 and older or above legal age who have systemic sclerosis. People can participate if they have a specific subtype called diffuse cutaneous systemic sclerosis. People with another subtype called limited cutaneous systemic sclerosis can also participate if they are anti Scl-70 antibody positive. Systemic sclerosis is also called scleroderma.

The purpose of this study is to find out whether a medicine called Avenciguat (BI 685509) helps people with scleroderma who have symptoms due to lung fibrosis or vascular problems.

Participants are put into 2 groups by chance. One group takes Avenciguat (BI 685509) tablets 3 times a day and the other group takes placebo tablets 3 times a day. Placebo tablets look like BI 685509 tablets but do not contain any medicine. Participants take the tablets for at least 11 months. Afterwards, participants can continue to take the tablets until the last participant has completed the 11-months treatment period. This means that the time in the study and duration of treatment is different for each participant, depending on when they start the study. At the beginning of the study, participants visit the study site every 2 weeks. The time between the visits to the study site gets longer over the course of the study. After the 11-months treatment period, participants visit the study site every 3 months.

During the study, participants regularly do lung function tests. The results are compared between the 2 groups to see whether the treatment works. The participants also regularly fill in questionnaires about their scleroderma symptoms. The doctors regularly check participants' skin condition and general health and take note of any unwanted effects.

Participating Sites:

-Medvin Clinical Research

Covina, California, United States, 91722

Contact: Boehringer Ingelheim    833-602-2368    unitedstates@bitrialsupport.com   

-University of California Los Angeles

Los Angeles, California, United States, 90095

Contact: Boehringer Ingelheim    833-602-2368    unitedstates@bitrialsupport.com   

-Medvin Clinical Research

Whittier, California, United States, 90602

Contact: Boehringer Ingelheim    833-602-2368    unitedstates@bitrialsupport.com   

A Study Evaluating the Efficacy and Safety of Vixarelimab in Participants With Idiopathic Pulmonary Fibrosis and in Participants With Systemic Sclerosis-Associated Interstitial Lung Disease. Sponsor: Genentech, Inc. 

The main purpose of the study is to evaluate the efficacy of vixarelimab compared with placebo on lung function in participants with idiopathic pulmonary fibrosis (IPF) and in participants with systemic sclerosis-associated interstitial lung disease (SSc-ILD). Participants who complete 52-weeks of treatment in the Double-blind Treatment (DBT) period can choose to enroll in the optional Open-label Extension (OLE) period to receive treatment with vixarelimab for another 52 weeks.

Participating Sites: (Contact: Reference Study ID Number: GB44496 https://forpatients.roche.com/ 888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com)

-University of California, San Francisco-Fresno

Fresno, California, United States, 93701

University of California, San Francisco Medical Center

-San Francisco, California, United States, 94115

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7303509 in Participants With Systemic Sclerosis. Sponsor: Genentech, Inc. 

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of RO7303509 treatment in participants with systemic sclerosis (SSc) during a multiple-ascending-dose (MAD) portion of the trial. In the MAD phase, increasing doses of study drug will be tested sequentially. For each dose tested, the MAD stage will consist of a treatment period of 12 weeks followed by either a safety follow-up period of 13 weeks or continued treatment in an optional open-label safety extension (OSE) stage of 52 weeks to assess the long-term safety. All patients in the OSE stage will receive RO7303509 and no patient will receive placebo.

Participating Sites: (Contact: Reference Study ID Number: GA43360 https://forpatients.roche.com/ 888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com)

-Stanford University

Palo Alto, California, United States, 94304-1808

Oral Ifetroban to Treat Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension. Sponsor: Cumberland Pharmaceuticals

The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH).

This study is a randomized, placebo-controlled, double-blind phase 2 trial of patients with dcSSc or SSc-PAH. Twenty participants with SSc-PAH and 14 participants with dcSSc will be randomized to receive either oral ifetroban daily or matching placebo. Study participants will be treated for 12 months, followed by a 30-day follow-up period. The study will test whether ifetroban is safe and statistically superior to placebo in reducing the effects of their disease at month 12 and explore the ability of ifetroban to prevent or reverse progression in patients with early disease duration and reverse established disease in patients with longer disease duration.

Participating Sites:

-UCLA Los Angeles, California, United States, 90095-1670

Contact: Nashla Barroso: 310-825-9682      

Principal Investigator: Suzanne Kafaja, MD  

Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY) Sponsor: AstraZeneca

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. 

(Open Label Treatment Period: At Week 52, all participants will be given anifrolumab 120 mg (subcutaneous) once weekly for 52 weeks (last dose at Week 103). Participation will involve in-clinic study visits at Weeks 52, 56, 64, 76. 88 and 104.) 

Safety Follow-up Period: All participants will return to the clinic for a 12-week post treatment visit. This will occur post Double Blind Treatment Period (Week 52 or Double Blind Period early discontinuation) or post Open Label Treatment Period (Week 104 or Open Label Period early discontinuation).

Participating Sites: 

-Los Angeles, California, United States, 90045

-Los Angeles, California, United States, 90095

-Orange, California, United States, 92868

-San Diego, California, United States, 92108

A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis. Sponsor: Horizon Therapeutics Ireland DAC

This is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial. Participants will be screened within 4 weeks prior to the Baseline (Day 1) Visit. Approximately 300 participants who meet the trial eligibility criteria will be randomized on Day 1 in a 1:1:1 ratio to receive HZN-825 300 mg QD, HZN-825 300 mg BID or placebo for 52 weeks. 

The trial will include up to a 4-week Screening Period and a 52-week Double-blind Treatment Period. Participants will take their first dose of trial drug at the clinic and will return to the clinic for trial visits at Week 4 and every 6 weeks thereafter until Week 52. All participants who complete the Double-blind Treatment Period (Week 52) will be eligible to enter a 52-week extension trial (HZNP-HZN-825-302, NCT not available yet). Participants not entering the extension will return to the clinic for a Safety Follow-up Visit 4 weeks after the last dose of trial drug.

Contact: HorizonTherapeutics 866-479-6742 clinicaltrials@horizontherapeutics.com
Participating Sites:

-Pacific Arthritis Care Center 

Los Angeles, California, United States, 90045

Contact: Stanique Thomas    310-297-6812    stanique.pa.clinicalresearch@gmail.com 

Principal Investigator: Daniel Furst, MD         

-UCLA Department of Medicine

Los Angeles, California, United States, 90095-1670

Contact: Contact: Nancy R Lopez    310-794-6213    nancyrlopez@mednet.ucla.edu  

Principal Investigator: Elizabeth Volkmann, MD

-Stanford University School of Medicine

Palo Alto, California, 94304-1808

Contact Study Coordinator: Kathryn Jee Email: kjee@stanford.edu

Principal Investigator: Dr. Lorinda Chung

CONQUER Registry: COllaborative National QUality and Efficacy Registry (CONQUER) for Tracking Disease Progression in Systemic Sclerosis (systemic sclerosis with diffuse or limited skin disease) Sponsor: Scleroderma Research Foundation

This is an observational study (no intervention is given) where select Scleroderma Centers of Excellence in the US enter patient data into a registry to understand how the disease evolves and to discover novel biomarkers that predict organ involvement. Any patient with systemic sclerosis with a disease duration of less than 5 years is eligible to participate. 

Participating Sites:

-UCLA Department of Medicine

Los Angeles, California, United States, 90095-1670

Contact: Contact: Nancy R Lopez: 310-794-6213 Email: nancyrlopez@mednet.ucla.edu  

Principal Investigator: Elizabeth Volkmann, MD

-Stanford University

Principal Investigator: Lorinda Chung, MD, MS 

Contact Study Coordinator: Kathryn Jee Email: kjee@stanford.edu

A Study to Evaluate the Efficacy, Safety, and Tolerability of BMS-986278 in Participants With Progressive Pulmonary Fibrosis. Sponsor: Bristol-Myers Squibb

The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986278 in Participants with Progressive Pulmonary Fibrosis.

Participating Sites: 

-UC Irvine Medical Center

Orange, California, United States, 92868-3201

Contact: Huawei Dong:  714-456-6776      

-Stanford Hospital and Clinics

Stanford, California, United States, 94305

Contact: Rishi Raj: 650-736-8083   

TBI Using IMRT and Cyclophosphamide Prior to Stem Cell Transplant for the Treatment of Severe Systemic Sclerosis

This early phase I trial studies the side effects and feasibility of total body irradiation using intensity modulation radiation therapy (IMRT) when given in combination with cyclophosphamide prior to stem cell transplant to treat severe systemic sclerosis. IMRT delivers total body radiation therapy more precisely and may reduce radiation exposure to sensitive normal organs. Giving chemotherapy, such as cyclophosphamide, and total body irradiation before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the bone marrow for new blood-forming cells (stem cells) to grow. Giving IMRT and cyclophosphamide prior to stem cell transplant may work better in treating severe systemic sclerosis and reduce radiation doses to lung and kidneys compared to cyclophosphamide alone.

Patients undergo TBI using IMRT twice daily (BID) on days -5 and -4 in the absence of disease progression or disease progression. Patients then receive cyclophosphamide on days -3 and -2 and undergo HSCT on day 0 in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up on days 30 and 100.

Contact: City of Hope Medical Center

Duarte, California, United States, 91010

Contact: Jeffrey Y. Wong    626-218-2247    jwong@coh.org   

Principal Investigator: Jeffrey Y. Wong    

COMING SOON:

CONQUEST: Sponsor: Scleroderma Reaearch Foundation

The primary objective of CONQUEST is to rapidly evaluate potential treatments for interstitial lung disease associated with systemic sclerosis (SSc-ILD). Secondary objectives include global SSc outcomes, including some focused on skin and other aspects of the disease.

CONQUEST arrives at the right moment to address those challenges and offers unique benefits for scleroderma patients. It also offers huge efficiencies to encourage more clinical trials and more rapid evaluation of potential therapies.

CONQUEST is a unique opportunity for patients for the following reasons:

-Efficient trial design to study multiple therapies with a go/no-go decision

-Be part of drug discovery/biomarker validation/outcomes measures development and validation

-Highest possible chance of randomization to an active arm (compared with other trial designs)

-Availability of placebo data to the broad scleroderma medical science community (to improve design and timelines iteratively)

Though none of the trial sites is currently recruiting, it is anticipated that more than 130 sites worldwide will participate in CONQUEST. Trial centers will be based in Europe, North America, Asia, and South America.