Phase I/II Study of QEL-005 in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc) and in Patients With Difficult to Treat Rheumatoid Arthritis (D2TRA).
New cell therapy QEL-005 tested in autoimmune diseases
Plain English Summary
Controlling Hyperactive Immunity With Long-lived Lymphocytes is a Phase 2 clinical trial sponsored by Quell Therapeutics Limited studying Diffuse Cutaneous Systemic Sclerosis, Rheumatoid Arthritis (RA), Systemic Sclerosis (SSc), Autoimmune Rheumatologic Disease. This trial tests QEL-005, a new treatment made from a patient's own cells, for adults with severe systemic sclerosis or difficult-to-treat rheumatoid arthritis. Participants will have their white blood cells collected, modified in a lab to target specific immune cells, and then reinfused. The study aims to determine if QEL-005 is safe and well-tolerated, and if it can help reduce disease activity. Alternative treatments may include existing medications for rheumatoid arthritis and systemic sclerosis, or other investigational therapies. The trial aims to enroll 16 participants.
Official Summary
This study is a Phase 1/2, open-label clinical trial to test an experimental treatment called QEL-005 in adults with two autoimmune conditions: diffuse cutaneous systemic sclerosis (dcSSc) and difficult-to-treat rheumatoid arthritis (D2TRA). The main goals are to find out whether QEL-005 is safe, how well people tolerate it, and whether it may help reduce disease activity or improve symptoms. QEL-005 is made from a participant's own white blood cells (autologous cells). These cells are collected and then changed in a laboratory using genetic methods to create specialized immune cells called CAR-T regulatory cells that target a protein on B cells called CD19. These modified cells are then given back to the participant by intravenous (IV) infusion. To take part, eligible participants will first have a procedure called leukapheresis, where some of their white blood cells are removed from the blood. The study team will use these cells to manufacture QEL005. After QEL005 is ready, participants will receive an IV infusion of their modified cells, stay in hospital overnight for monitoring, and will then be followed closely in the clinic. Throughout the trial, participants will have regular safety checks, which may include blood tests, imaging scans, questionnaires about symptoms and daily functioning, and biopsies taken from involved tissues, to help understand how QEL005 is working in the body. Detailed follow up will be for 1 year after QEL-005 infusion, and there is long-term follow up for a total of 15 years, which is standard for cell therapies. The information from this Phase 1/2 study will help determine an appropriate dose and dosing schedule of QEL005 for future studies.
Who Can Participate
Here is what you need to know about eligibility for this trial. Adults aged 18 and older with a confirmed diagnosis of diffuse cutaneous systemic sclerosis or difficult-to-treat rheumatoid arthritis. Patients must have active disease despite current treatments and meet specific criteria for their condition. Individuals with significant other medical conditions, active infections, immunodeficiency, or a history of certain cancers may not be eligible. Participants must have adequate organ function (blood, liver, kidney) and be willing to undergo long-term follow-up. This trial is studying Diffuse Cutaneous Systemic Sclerosis, Rheumatoid Arthritis (RA), Systemic Sclerosis (SSc), Autoimmune Rheumatologic Disease, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcome measures will assess the safety of QEL-005 by looking for any serious side effects or toxicities within the first month after treatment, and will continue to monitor for any advers The specific primary outcome measures are: D2TRA & SSc: Incidence of Protocol-Defined Dose Limiting Toxicities (DLTs) to Assess Safety and Tolerability (Dose Escalation Phase Only) (up to 28 days post-infusion); D2TRA & SSc: Incidence and grade of treatment-emergent adverse events (TEAEs), adverse events (AEs) and adverse events of special interest (AESIs) (From time of signing the informed consent form (ICF) through to week 52); D2TRA & SSc: Incidence of clinically significant abnormalities in safety laboratory parameters, electrocardiogram (ECG) findings, and vital signs (Up to week 52). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.
Why This Trial Matters
This trial addresses a significant unmet need for patients with severe autoimmune diseases like systemic sclerosis and rheumatoid arthritis who have not responded to standard therapies, by exploring a Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Diffuse Cutaneous Systemic Sclerosis, Rheumatoid Arthritis (RA), Systemic Sclerosis (SSc), Autoimmune Rheumatologic Disease, where improved treatment options are needed.
Investor Insight
This trial represents an early-stage investment in a potentially innovative cell therapy for autoimmune diseases, a large and growing market, with QEL-005 targeting B cells, a common strategy in this Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the specific risks and benefits of QEL-005, and how it compares to your current treatment options. Participation involves a procedure to collect your blood cells, a period for cell modification, an infusion of the modified cells, and regular clinic visits for monitoring over several years. Be prepared for potential hospital stays for monitoring after the infusion and for a commitment to long-term follow-up for up to 15 years. The trial is being conducted at 6 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NON_RANDOMIZED
- Model: SEQUENTIAL
- Masking: NONE
- Enrollment: 16 participants
Interventions
- DRUG: QEL-005 — QEL-005 is an autologous cell therapy that is composed of engineered regulatory T cells transduced with a lentiviral vector containing a CAR directed against the B cell marker CD19. Treatment will be given via an IV infusion.
Primary Outcomes
- D2TRA & SSc: Incidence of Protocol-Defined Dose Limiting Toxicities (DLTs) to Assess Safety and Tolerability (Dose Escalation Phase Only) (up to 28 days post-infusion)
- D2TRA & SSc: Incidence and grade of treatment-emergent adverse events (TEAEs), adverse events (AEs) and adverse events of special interest (AESIs) (From time of signing the informed consent form (ICF) through to week 52)
- D2TRA & SSc: Incidence of clinically significant abnormalities in safety laboratory parameters, electrocardiogram (ECG) findings, and vital signs (Up to week 52)
Secondary Outcomes
- D2TRA: Change from baseline in American College of Rheumatology (ACR) response criteria (Week 4,8,12,24,38 and 52)
- D2TRA: Change from baseline in a disease activity score-28 C-reactive protein (DAS28-CRP) (Week 4,8,12,38 and 52)
- D2TRA: Change from baseline in Health Assessment Questionnaire Disability Index (Week 4,8,12,24,38 and 52)
- D2TRA: Change from baseline in Ultrasound Outcome Measure in Rheumatology (OMERACT) score (Week 12 and 52)
- D2TRA: Clinical response status (Week 12 and 52)
Full Eligibility Criteria
Inclusion Criteria: * Participants must be at least 18 years of age at the time of signing the informed consent. * Up to date vaccination status and no planned vaccinations for post 3 months infusion * Adequate haematological, liver and renal function * Willing to undergo annual influenza vaccination * Willing to enter a 15-year follow-up * Eastern Cooperative Oncology Group (ECOG) performance status grade \< 3 * Able and willing to use a highly effective method of contraception * Stable dose of steroid prior to screening Specific inclusion criteria for participants with difficult to treat rheumatoid Arthritis (D2TRA) only: * Diagnosis of Rheumatoid Arthritis (RA) per 2010 ACR-EULAR criteria * Diagnosis of D2TRA per 2021 EULAR criteria * Evidence of clinically active disease a defined by validated clinical or laboratory results consistent with standard definitions of active RA * Evidence of inflammation in target joints used for the DAS28 CRP assessment Specific inclusion criteria for participants with diffuse cutaneous systemic sclerosis (dcSSc) only: * Diagnosis of dcSSc as per the 2013 ACR-EULAR criteria * Serologically positive for antinuclear antibodies * Failure to respond sufficiently to immunomodulatory disease modifying anti-rheumatic drugs (DMARDs). * Skin involvement with a total modified Rodnan Skin Score of at least 15 * Evidence of lung fibrosis based on imaging or pulmonary function testing * Evidence of active disease based on a validated SSc activity assessment Exclusion Criteria: * Presence of a significant medical condition(s), or clinically significant laboratory abnormality * History or concern of autoimmune diseases other than those under study * Active infection, or recurrent chronic infection requiring intervention * Immunodeficiency or receiving immunoglobulin replacement therapy * Past or current infection with hepatitis B or C, tuberculosis, syphilis, or HIV * Clinically significant cardiac dysfunction or severe pulmonary impairment * Use of investigational agents within a pre-defined period prior to study screening * Received a previous cell therapy * Received certain B cell related experimental therapies in a clinical trial with the past year * Any solid organ, bone marrow or stem cell transplant * History of malignancy in the past 5 years * Receiving prohibited medication that cannot be stopped at screening
Trial Locations
- University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom
- Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom
- Guy's & St Thomas NHS Foundation Trust, London, United Kingdom
- Royal Free London NHS Foundation Trust, London, United Kingdom
- Newcastle Upon Tyne NHS Foundation Trust, Newcastle, United Kingdom
- University of Oxford - The Kennedy Institute, Oxford, United Kingdom
Frequently Asked Questions
What is clinical trial NCT07473154?
NCT07473154 is a Phase 2 INTERVENTIONAL study titled "Controlling Hyperactive Immunity With Long-lived Lymphocytes." It is currently not yet recruiting and is sponsored by Quell Therapeutics Limited. The trial targets enrollment of 16 participants.
What conditions does NCT07473154 study?
This trial investigates treatments for Diffuse Cutaneous Systemic Sclerosis, Rheumatoid Arthritis (RA), Systemic Sclerosis (SSc), Autoimmune Rheumatologic Disease. The primary condition under study is Diffuse Cutaneous Systemic Sclerosis.
What treatments are being tested in NCT07473154?
The interventions being studied include: QEL-005 (DRUG). QEL-005 is an autologous cell therapy that is composed of engineered regulatory T cells transduced with a lentiviral vector containing a CAR directed against the B cell marker CD19. Treatment will be given via an IV infusion.
What does Phase 2 mean for NCT07473154?
Phase 2 trials test whether the treatment works for the intended condition. They involve 100-300 patients and continue to evaluate safety while measuring effectiveness.
What is the current status of NCT07473154?
This trial is currently "Not Yet Recruiting." It started on 2026-04. The estimated completion date is 2028-08.
Who is sponsoring NCT07473154?
NCT07473154 is sponsored by Quell Therapeutics Limited. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT07473154?
The trial aims to enroll 16 participants. The trial has not yet started recruiting.
How is NCT07473154 designed?
This is a interventional study, uses non_randomized allocation, follows a sequential design, employs none masking.
What are the primary outcomes being measured in NCT07473154?
The primary outcome measures are: D2TRA & SSc: Incidence of Protocol-Defined Dose Limiting Toxicities (DLTs) to Assess Safety and Tolerability (Dose Escalation Phase Only) (up to 28 days post-infusion); D2TRA & SSc: Incidence and grade of treatment-emergent adverse events (TEAEs), adverse events (AEs) and adverse events of special interest (AESIs) (From time of signing the informed consent form (ICF) through to week 52); D2TRA & SSc: Incidence of clinically significant abnormalities in safety laboratory parameters, electrocardiogram (ECG) findings, and vital signs (Up to week 52). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT07473154 being conducted?
This trial is being conducted at 6 sites, including Birmingham; Leeds; London; Newcastle and 2 more sites (United Kingdom).
Where can I find official information about NCT07473154?
The official record for NCT07473154 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT07473154. This government database provides the most up-to-date and detailed information about the trial.
What is NCT07473154 testing in simple terms?
This trial tests QEL-005, a new treatment made from a patient's own cells, for adults with severe systemic sclerosis or difficult-to-treat rheumatoid arthritis. Participants will have their white blood cells collected, modified in a lab to target specific immune cells, and then reinfused.
Why is this trial significant?
This trial addresses a significant unmet need for patients with severe autoimmune diseases like systemic sclerosis and rheumatoid arthritis who have not responded to standard therapies, by exploring a
What are the potential risks of participating in NCT07473154?
Potential risks include side effects related to the cell infusion, such as fever, fatigue, or flu-like symptoms. There is a risk of cytokine release syndrome, a serious immune reaction that can cause fever, low blood pressure, and breathing difficulties. Other potential side effects may include infections, low blood cell counts, or effects on organ function, which will be closely monitored. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT07473154?
Ask your doctor about the specific risks and benefits of QEL-005, and how it compares to your current treatment options. Participation involves a procedure to collect your blood cells, a period for cell modification, an infusion of the modified cells, and regular clinic visits for monitoring over several years. Be prepared for potential hospital stays for monitoring after the infusion and for a commitment to long-term follow-up for up to 15 years. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT07473154 signal from an investment perspective?
This trial represents an early-stage investment in a potentially innovative cell therapy for autoimmune diseases, a large and growing market, with QEL-005 targeting B cells, a common strategy in this This is a Phase 2 trial, which is focused on confirming efficacy before larger pivotal studies.
What happens if the treatment in this trial doesn't work?
The study aims to determine if QEL-005 is safe and well-tolerated, and if it can help reduce disease activity. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.