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A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.

ConditionsRelapsed AL AmyloidosisRefractory AL AmyloidosisLight Chain AmyloidosisAmyloidosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorAlexion Pharmaceuticals, Inc.

About this trial

Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Confirmed histopathological diagnosis of AL amyloidosis

One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines

Measurable hematologic disease: dFLC > 20 mg/L or serum M-protein > 5g/L

Relapsed or refractory disease with a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.

Disqualifiers

Have any other form of amyloidosis other than AL amyloidosis

Mayo Stage IIIb AL amyloidosis

Oxygen saturation < 95% on room air

Systolic blood pressure <100mmHg

Trial design

Design model

Single group

Treatments tested in this trial

  • AZD0120

    Drug

    Participants will receive AZD0120 via intravenous (IV) infusion.

Treatment groups

91 Participants
are divided into 1 treatment group
Group A: AZD0120Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Phase 1b: Number of Participants With incidence and severity of Treatment-emergent Adverse Events

Time frame
Through study completion, a minimum of 6 months
2

Phase 2: Proportion of Participants Achieving Complete Response

Time frame
Through study completion, a minimum of 6 months

Secondary outcomes

1

Phase 1b: Levels of AZD0120 in blood over time in participants with AL amyloidosis

Time frame
Through study completion, a minimum of 6 months
2

Phase 2: Percentage of participants achieving hematologic response

Time frame
Through study completion, a minimum of 6 months

Other outcomes

Sponsors and contacts

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

Alexion Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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