Health TrialsbyHealthPanel

Autoimmune Cytopenia Elimination by Chimeric Antigen Receptor T-cell Therapy

ConditionCytopenia
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-80
SponsorSaurabh Dahiya

About this trial

This is a phase 1 study to evaluate the safety and tolerability of AZD0120 in patients with relapsed or refractory immune mediated cytopenia (primary chronic Immune Thrombocytopenia (ITP) or autoimmune hemolytic anemia (wAIHA)).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of primary chronic immune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA) according to the published consensus criteria

1st line treatment includes steroids (dexamethasone, prednisone or equivalent), IVIG, and/or Anti-Rhd Ig. These medications may be administered in combination or in series.

2nd line treatment includes the use of thrombopoietic agents, and anti-CD20 mAbs.

A failure to respond to splenectomy is considered an additional line of therapy.

Disqualifiers

For ITP - ITP Bleeding Scale Grade 3 or higher at the screening visit (with the exception of menorrhagia controlled with hormonal therapy +/- tranexamic acid)

For ITP - Hgb < 8 g/dL (NOTE: Exclusion does not apply to Evans Syndrome).

For wAIHA - subjects with signs of hemolysis not attributable to wAIHA are excluded.

For wAIHA - platelet count <100,000/uL (NOTE: ITP subjects may be below this threshold if due to active ITP. Exclusion does not apply to Evans Syndrome).

Trial design

Design model

Sequential

Treatments tested in this trial

  • AZD0120

    Drug

    One time administration on Day 0.

  • Bendamustin

    Drug

    Participants will be given bendamustin for 2 days (study Day -5 and Day -4) followed by 3 days of rest.

Treatment groups

16 Participants
are divided into 2 treatment groups
Group A: AZD0120 (cell therapy product) + BendamustineExperimental treatment 2 interventions
Group B: AZD0120 (cell therapy product)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of adverse events

The incidence of adverse events defined as dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS).

Time frame
within 28 days after the CAR-T administration

Secondary outcomes

1

Incidence of adverse events and serious adverse events following infusion

Evaluate the safety of AZD0120 cells by the incidence and severity of adverse events, and serious adverse events, following infusion. Events will be recorded and graded according to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 or ASTCT Consensus Grading.

Time frame
until 28 days after the CAR-T administration
2

Number of CAR-T cells in blood

The CAR T cell expansion and persistence will be followed at designated time points after infusion. Study participants should have detectable CAR T cells in the blood in at least 2 consecutive time points (Day +3, +7, +10, +14, +21, +28).

Time frame
Day +3, +7, +10, +14, +21, +28
3

Clinical response rate

Measured by complete response (CR), response (R), durable response (DR), and treatment-free remission for each disease arm as determined by published guidelines.

Time frame
At 3, 6, 9, and 12-months post infusion

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Saurabh Dahiya

Lead sponsor

Stanford University

Sponsor institution

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: