Using patients' own T cells to treat Aplastic Anemia
Production of Expanded Autologous Regulatory T Cells to Treat Patients With Refractory Aplastic Anaemia in a Phase I Dose Finding Study
This study is testing whether using patients' own T cells can help people with Aplastic Anemia who haven't responded to other treatments by improving their blood counts and helping their bone marrow recover.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | King's College Hospital NHS Trust Academic / other |
| Locations | 1 site (London) |
| Trial ID | NCT05386264 on ClinicalTrials.gov |
What this trial studies
This Phase I clinical trial aims to evaluate the safety and optimal dosage of expanded autologous T regulatory cells (Tregs) in patients with Aplastic Anemia (AA) who have not responded to or are ineligible for standard immunosuppressive therapies. The study will involve collecting Tregs from the patients themselves, which will be expanded in a licensed facility and then administered back to the patients. Researchers will monitor the immune system's response and assess whether this treatment can lead to improved blood counts and recovery of healthy bone marrow stem cells. This approach is novel for Aplastic Anemia, although similar therapies have shown promise in treating other autoimmune disorders.
Who should consider this trial
Good fit: Ideal candidates include adults with acquired idiopathic Aplastic Anemia who are transfusion-dependent and have failed or are ineligible for other treatments.
Not a fit: Patients with constitutional or inherited forms of Aplastic Anemia or those who have a matched sibling or unrelated donor may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with Aplastic Anemia who have limited treatment alternatives.
How similar studies have performed: While this specific application in Aplastic Anemia is novel, expanded autologous Tregs have shown safety and efficacy in treating other autoimmune diseases in previous studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Acquired idiopathic AA * No evidence of constitutional/inherited AA based on clinical findings, absence of family history of AA, normal DEB test and normal Kings bone marrow failure gene panel * Very severe, severe or non-severe AA * Lack a matched sibling donor (MSD) or matched unrelated donor (MUD), or ineligible for MSD/MUD HSCT * Transfusion dependent * Failed or ineligible for a course of ATG and CSA * Failed / intolerant or inappropriate to treat with Eltrombopag or fails to meet Blueteq approval for use of Eltrombopag using NHS England guidance * AST \< 3 x upper limit of normal (ULN), bilirubin \< 1.5 x ULN (unless Gilbert's syndrome) * eGFR \>50mL/min * Age ≥ 18 years, male or female * Willing and able to provide written and informed consent * If female of child-bearing potential, have a negative serum pregnancy test and agree to use adequate contraceptive methods if of reproductive age * Diffusing capacity for carbon monoxide (DLCO) ≥ 45% predicted corrected for haemoglobin * LVEF \> 40%. * Performance status ≤ 2 Exclusion Criteria: * Constitutional AA * Age \< 18 years' old * Have a MSD and are eligible for MSD HSCT * Have a MUD and are eligible for MUD HSCT * Hypocellular myelodysplastic syndrome (Hypo MDS) or AA/Hypo MDS overlap * Uncontrolled ongoing infection * Active malignancy * Treatment of cancer in the last 5 years (except in situ carcinoma of the cervix or basal cell carcinoma) * Unable to give informed consent * Active or uncontrolled infection not responding to appropriate antibiotics and antifungal agents. * Human immunodeficiency virus (HIV) sero-positivity, hepatitis B, hepatitis C or hepatitis E infection. * Abnormal organ function: AST/ALT \>3 x upper limit of normal (ULN), bilirubin \>1.5 x ULN, eGFR ≤50mL/min * Heart failure (= grade III New York Heart Association) * Pregnant or lactating women * Unable or unwilling to comply with the contraceptive requirements
Where this trial is running
London
- King's College Hospital — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Jen Lewis
- Email: jen.lewis@kcl.ac.uk
- Phone: 07890254538
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.