CLINICAL TRIALS

A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis (eSScape)

Sponsor: argenx

Official Title
A Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Systemic Sclerosis

The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months.

Enrolling Sites:

Arizona Locations

Phoenix, Arizona, United States, 85032-9306

Recruiting Arizona Arthritis and Rheumatology Associates

Contact : Saima Chohan, MD

857-350-4834  clinicaltrials@argenx.com

California Locations

Los Angeles, California, United States, 90095

Recruiting UCLA Ronald Reagan University of California Los Angeles Medical Center

Contact : Suzanne Kafaja, MD

857-350-4834  clinicaltrials@argenx.com

Tibulizumab Systemic Sclerosis Understanding and Response Evaluation (TibuSURE)

Sponsor: Zura Bio Inc

The study is a Phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the effects of tibulizumab over 24 weeks (Period 1) in adult participants with systemic sclerosis, followed by an open-label extension period where all active participants will receive tibulizumab and will be evaluated for an additional 28 weeks (Period 2)

Official Title

A Phase 2, Multi-Center Study Consisting of a Randomized, Double-Blind, Placebo-Controlled Period, Followed by an Open-Label Extension Period, to Assess the Efficacy, Safety, and Tolerability of Tibulizumab in Adults With Systemic Sclerosis

California Enrolling Site:

La Jolla, California, United States, 92037

UCSD Altman Clinical and Translational Research Institute Center for Clinical Research

Contact : Principal Investigator

702-825-9872  clinicaltrial@zurabio.com

A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis

Sponsor: Novartis Pharmaceuticals

The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with diffuse cutaneous systemic sclerosis relative to placebo

Official Title:

A Randomized, Double-blind, Parallel Group, Placebo-controlled Multicenter Study to Evaluate Efficacy, Safety and Tolerability of Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis

Detailed Description: The study consists of the following periods:

Screening Period, with a duration of up to 6 weeks;

Treatment Period 1, with a duration of 52 weeks;

Treatment Period 2 (Open-label treatment), with a duration of 52 weeks;

Post-treatment Follow-up Period, with a duration of at least 20 weeks post last dose and up to 2 years.

Enrolling sites:

Arizona Locations

Mesa, Arizona, United States, 85202

Arizona Arthritis and Rheumatology Research PLLC

Contact : Debra Mc Dermed

602-386-4970  Debra.McDermed@azarthritis.com

Principal Investigator : Nehad Soloman

California Locations

Los Angeles, California, United States, 90095

UCLA

Contact : Lauren Nam

310-488-0162  lnam@mednet.ucla.edu

Principal Investigator : Suzanne Kafaja

Newport Beach, California, United States, 92663

Hoag Hospital

Contact : Vicki Tan

949-791-3049  vicki.tan@hoag.org

Principal Investigator : Christine Thai

EncompaSSc: Evaluation of MTX-474 in Participants With Diffuse Cutaneous Systemic Sclerosis (dcSSc)

Sponsor: Mediar Therapeutics

Brief Summary: A Phase 2 Randomized, Double-blind, Placebo-Controlled Study of the Safety and Efficacy of MTX-474 in Participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc)

Detailed Description:Participants with dcSSc who meet the study's inclusion and exclusion criteria will be randomly assigned in a 3:2 ratio to receive MTX-474 or a matching placebo by intravenous (IV) infusion. Concomitant use of one of the approved dcSSc therapies (immunosuppressive therapy, systemic glucocorticoids or other antifibrotic agents) is permitted under certain criteria. Participants randomized to the MTX-474 arm of the study will receive an IV infusion every 4 weeks, beginning at Day 0 and ending at Week 20. The End of Treatment Visit will occur at Week 24, and a Safety Follow-Up Visit will occur at Week 28, 8 weeks after the final infusion. mRSS assessments will occur at Screening, Baseline, and at all subsequent treatment visits up to and including Week 24. Spirometry will be performed at screening and Weeks 12. HRCT will be performed at screening and week 24. DLCO will be performed at Screening. Skin biopsies will be performed at Baseline and Week 12. Participants will have blood drawn for safety assessment and to assess Ephrin receptor levels at Screening, Baseline and every 4 weeks until Week 28. Blood will be drawn for serum PK analyses relative to the first and last doses of MTX-474.

Official Title:A Phase 2 Randomized, Double-blind, Placebo-Controlled Study to Assess the Safety and Efficacy of MTX-474 in the Treatment of Participants With Diffuse Cutaneous Systemic Sclerosis (dcSSc)

Enrolling Site in California:

Newport Beach, California, United States, 92663

Encompa 

Contact :EncompaSSc contact

617 802 6568  EncompaSSc@mediartx.com

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

Sponsor: CRISPR Therapeutics

Brief Summary:

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).

Detailed Description:

This study may enroll up to 80 subjects in total. CTX112 is a CD19 directed chimeric antigen receptor (CAR) T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are from healthy adult volunteer donors that are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease).

Official Title:

A Phase 1 Dose Evaluation Study of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Subjects With Refractory Autoimmune Disease

Enrolling Site:

Redwood City, California, United States, 94063

Research Site 4

Name: Clinical Trials Contact:

Phone Number: 1 877-214-4634

Email: medicalaffairs@crisprtx.com

A Study of CNTY-101 in Participants with Refractory B Cell-mediated Autoimmune Diseases (CALiPSO-1) (CALiPSO-1)

ClinicalTrials.gov ID NCT06255028

Sponsor: Century Therapeutics, Inc.

CALiPSO-1 is a Phase 1, multi-centre, dose-confirmation study to evaluate the safety and efficacy of CNTY-101 in participants with refractory B cell-mediated autoimmune diseases including those with moderate to severe systemic lupus erythematosus (SLE)/ lupus nephritis (LN), idiopathic inflammatory myopathies (IIM), and diffuse cutaneous systemic sclerosis (DcSSc).

Participating Sites:

-Los Angeles, California, United States, 90033 (Recruiting)

Keck School of Medicine of University of Southern California

Contact: Sara Madrigal at: 323-409-4349 smadriga@usc.edu

-Sacramento, California, United States, 957817 UC Davis (Not yet recruiting)

Contact: Elizabeth Robison at: 916-734-8101 eerobison@ucdavis.edu

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:

CALIFORNIA: 

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

-Los Angeles, California, United States, 90045

Principal Investigator: Daniel Furst         

-Los Angeles, California, United States, 90095

Principal Investigator: Elizabeth Volkmann         

-Los Angeles, California, United States, 90095

Principal Investigator: Suzanne Kafaja         

-Upland, California, United States, 91786

Principal Investigator: Ebrahim Sadeghi-Najafabadi       

ARIZONA: 

-Phoenix, Arizona, United States, 85027

Principal Investigator: Da-Wei Liao

-Scottsdale, Arizona, United States, 85258

Principal Investigator: Richard Sue

-Scottsdale, Arizona, United States, 85259

Principal Investigator: Vivek Nagaraja

-Tucson, Arizona, United States, 85724

Principal Investigator: Sachin Chaudhary

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

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)

Arizona Locations:

-Southern Arizona VA Health Care System NAVREF PPDS

California Locations:

-Fresno, California, United States, 93701-2302

University of California, San Francisco-Fresno

-Los Angeles, California, United States, 90033-1036

University of Southern California Keck School of Medicine

-Los Angeles, California, United States, 90095-8344

UCLA Rheumatology

-San Francisco, California, United States, 94143-2204

University of California, San Francisco Medical Center

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).

Contact: AstraZeneca Clinical Study Information Center

Phone Number: 1-877-240-9479

Email: information.center@astrazeneca.com

Participating Sites: 

-Los Angeles, California, United States, 90045

-Los Angeles, California, United States, 90095

-Orange, California, United States, 92868

-San Diego, California, United States, 9210

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: 

Arizona Locations

-St. Joseph's Hospital and Medical Center

Contact: Rajat Walia, Site 0314

602-406-4000

California Locations

-Scripps Clinic Torrey Pines

Contact: Jacqueline Chang, Site 0336 at: 858-824-5404

-University of Southern California (USC) - Keck School of Medicine (KSOM) - Transplant Clinic

Contact: Toby Maher, Site 0212 at: 323-865-9854

-David Geffen School of Medicine at UCLA

Contact: John Belperio, Site 0019 at: 310-968-6324

-Orange, California, United States, 92868-3201

UC Irvine Medical Center

Contact: Huawei Dong, Site 0366 at: 714-456-6776

-Sacramento, California, United States, 95817

University of California UC Davis Medical Center

Contact: Timothy Albertson, Site 0341 at: 916-734-3650

-San Francisco, California, United States, 94143-2202

University of California, San Francisco Medical Center- Pulmonary Practice

Contact: Jeffrey Golden, Site 0015 at: 415-353-2060

-Stanford Hospital and Clinics

Contact: Rishi Raj, Site 0352 at: 650-725-8083

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