Status:
UNKNOWN
Autologous Tregs for Aplastic Anaemia
Lead Sponsor:
King's College Hospital NHS Trust
Conditions:
Aplastic Anemia
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
This Phase I study will determine the safety and optimal dose of expanded autologous Tregs to treat patients with Aplastic Anaemia (AA) (who have failed, or are considered ineligible for IST (immunosu...
Detailed Description
The clinical trial will examine the safety of giving AA patients who have failed other treatment(s), their own ('autologous') expanded Tregs - a form of 'cellular therapy - to treat the AA. The invest...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 14 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 30 2025
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT05386264
Start Date
July 14 2022
End Date
April 30 2025
Last Update
October 5 2023
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
King's College Hospital
London, United Kingdom