A multicenter, Phase 1b, open label, nonrandomized, single dose study evaluating the safety, tolerability and activity of BIVV020 in adults with cold agglutinin disease
- Conditions
- CADcold agglutinin disease10003816
- Registration Number
- NL-OMON55174
- Lead Sponsor
- Genzyme Europe BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
- Male and/or female patients, * 18 years of age with cold agglutinin disease
as defined by:
a) Chronic hemolysis per Investigator*s judgement,
b) Polyspecific direct antiglobulin test (DAT) positive,
c) Monospecific DAT strongly positive for C3d,
d) Cold agglutinin (CAg) titer * 64 at 4°C; and,
e) IgG DAT *1+.
- A hemoglobin level *11 mg/dL.
- A total bilirubin level above the normal reference range that is thought to
be due to hemolysis.
- Documented vaccinations against encapsulated bacterial pathogens (Neisseria
meningitidis,
including serogroup B meningococcus and Streptococcus pneumoniae) within five
years of
screening or willing to complete protocol specified vaccinations.
- Having given written informed consent prior to undertaking any study-related
procedure.
- Cold agglutinin syndrome secondary to infection, rheumatologic disease, or
known high grade
hematologic malignancy, or known solid organ tumor.
- Clinically relevant infection of any kind within one month preceding
screening.
- Treatment with anti-CD20 monotherapy within three months or anti CD20
combination therapies
within six months prior to screening.
- Concurrent treatment with systemic immunosuppressive agents targeting B- or
T-cell function
and/or cytotoxic agents within 3 months prior to screening. Concurrent
treatment with other
systemic immunosuppressants within 5.5 half-lives of the drug prior to
screening.
- Any specific complement system inhibitor within three months prior to
screening.
- Concurrent treatment with systemic corticosteroids other than a stable daily
dose equivalent to
*10 mg/day prednisone within three months prior to screening.
- If female, pregnant or lactating.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety<br /><br>* Assessment of adverse events (AE)/treatment-emergent adverse events (TEAE).<br /><br>* Clinical laboratory evaluations including hematology, biochemistry, systemic<br /><br>lupus erythematosus (SLE) panel testing, and urinalysis.<br /><br>* Electrocardiographic (ECG) intervals (heart rate, PR, QRS, QT, and QTcF).<br /><br>* Vital signs (blood pressure and heart rate).</p><br>
- Secondary Outcome Measures
Name Time Method <p>Hematologic BA<br /><br>* Total bilirubin.<br /><br>* Hemoglobin.<br /><br><br /><br>Pharmacodynamics<br /><br>* CP.<br /><br>* Complement alternative pathway (AP).<br /><br>* CH50.<br /><br>* Total C4.<br /><br><br /><br>Pharmacokinetics<br /><br>* Parameters (not limited to): Cmax, tmax, tlast, AUClast, AUC0-*, t1/2z, CL,<br /><br>Vd.<br /><br><br /><br>Immunogenicity<br /><br>* Anti-BIVV020 antibodies (ADA).</p><br>