Immune Thrombocytopenia (ITP), Warm Autoimmune Hemolytic Anemia (WAIHA)
Conditions
Keywords
Autoimmune disease, Blood platelet disorder, Thrombocytopenia, Anemia, Hemolytic anemia, Red blood cell disorder, Autoimmune cytopenia, Warm Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, Immune Thrombocytopenic Purpura
Brief summary
A Phase 1b/2a, open-label dose escalation and dose expansion study of subcutaneously (SC) administered velinotamig for the treatment of adults with relapsing and refractory immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA) to evaluate safety and tolerability.
Interventions
DRUGVelinotamig specified dose on specified days.
Velinotamig is an engineered bispecific antibody directed against BCMA and CD3.
Sponsors
Cullinan Therapeutics Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Age ≥18 to 80 years * Active autoimmune cytopenia * Relapsed/refractory after standard of care therapy * ECOG performance status 2 or lower Laboratory parameters including the following: * Absolute lymphocyte count (ALC) ≥0.5 × 109/L * Absolute neutrophil count (ANC) ≥1.0 × 109/L * Hemoglobin ≥6.5 g/dL * Total bilirubin ≤1.5 × ULN unless related to Gilbert's syndrome * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.0 × ULN, unless attributable to hemolysis * Estimated glomerular filtration rate (eGFR) based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula ≥30 mL/min/1.73m2
Exclusion criteria
* Pregnant or lactating women * History of clinically significant disease, condition, or medical history that, in the opinion of the Investigator, would interfere with subject safety, study evaluations, and/or study procedures, would put the patient at undue risk or confound study results * Evidence of active hepatitis B virus (HBV), hepatitis C virus (HCV), HIV, Epstein-Barr virus (EBV), or cytomegalovirus (CMV) infection * Active or latent tuberculosis (TB) evidenced by a positive or indeterminate interferon gamma release assay (IGRA), unless the patient has documented previous completion of TB treatment and no current clinical indication of TB * Presence of New York Heart Association class III or IV congestive heart * Primary immunodeficiency or history of recurrent infections * Previous treatment with a BCMA-targeted therapy * Receipt of an investigational therapy within 30 days or 5 drug-elimination half-lives (whichever is longer) prior to Day 1 * History of solid organ transplant * Planned major surgery in the timeframe of the dosing period
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability | 48 weeks | Incidence and severity of all adverse events, serious adverse events, adverse events of special interest, adverse events leading to treatment discontinuation, and laboratory abnormalities. |
Countries
United States
Contacts
CONTACTShane McLoughlin
Outcome results
None listed