The Safety of Repurposing Daratumumab for Relapsed or Refractory Autoimmune Antibody Mediated Hemolytic Anemia

STUDY02000992 21SUL992

A single-arm study utilizing a 6 x 4 expansion design using daratumumab SC treatment for patients with refractory Autoimmune Hemolytic Anemia.

2022-03-21

2024-12-01

2025-12-01

Active (not recruiting)

Early phase I

2

Inclusion Criteria: * All patients must have confirmed autoimmune hemolytic anemia, based on a Hemoglobin \<10 g/dL, a positive Direct Antiglobulin Test, elevated LDH, and elevated Reticulocyte count, low Haptoglobin test. * Patients must have been previously treated with both a course of steroids starting at a dose of at least 1mg/kg of Prednisone (or steroid equivalent) and at least 100mg of rituximab previously. They must show signs of ongoing hemolysis (as above) either 1) recurring after previous treatment, 2) or while on a Prednisone dose (or equivalent) of 10mg daily or greater. * Age ≥18 years * Patients are allowed to be on steroids, as per standard of care. The dosing regimens may include up to 1mg/kg of Prednisone followed by a Prednisone taper, or a regimen of 40mg of Dexamathasone for 4 days. (See Section 6.13 for recommended steroid taper.) * All patients must give informed consent indicating they are aware of the investigational nature of this treatment, as well as the study protocols and requirements. * Patients with Evan's syndrome are permissible * Patients must have performance status of ECOG 0-2 Exclusion Criteria: * Patients with active HIV, Hepatitis B, Hepatitis C. We define active as having a detectable viral load. Patients with a prior exposure or well controlled disease while on treatment will be permitted. More information regarding management of these infections can be found in the footnote of the schema in Section 6.1. * Patients with active Systemic Lupus Erythematosus with other systemic organ involvement requiring treatment * Patients with active lymphoid malignancy, other than Chronic Lymphoid Leukemia or other low grade lymphoproliferative disorders, not otherwise requiring treatment. Patients with a history of solid tumors are allowed, but must not have received treatment (chemotherapy, surgery, etc.) for a malignancy within 3 years before the date of randomization (exceptions are squamous and basal cell carcinomas of the skin, carcinoma in situ of the cervix or breast, or other non-invasive lesion), which is considered cured with minimal risk of recurrence within 3 years. * Patients with a serious uncontrolled medical disorder or active infection which would impair their ability to receive study treatment will be excluded. Significant cardiac disease, including uncontrolled high blood pressure, unstable angina, congestive heart failure, myocardial infarction within the previous 3 months or serious cardiac arrhythmias will be excluded. Dementia or significantly altered mental status that would prohibit the understanding or rendering of informed consent and compliance with the requirements of this protocol will be excluded. * Patients who are pregnant or breastfeeding * COPD with an FEV1 \<50% predicted, or moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification * Renal failure with GFR \<20 ml/min * End stage liver disease, as defined by local guidelinesEnd stage liver disease, as defined by local guidelines * Prior treatment with daratumumab or any other anti-CD38 therapies * Exposure to an investigational drug (including investigational vaccine) or invasive investigational medical device for any indication within 4 weeks or 5 pharmacokinetic half-lives, whichever is longer.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 2, 'type': 'ACTUAL'}}

2024-01-18