Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Conditions
Keywords
blood disorders, hemoglobin, red blood cells, RBCs, sickle-like shape, mutation in hemoglobin gene, Sickle Cell Disease, SCD, Vaso-occlusive Crisis, VOC, SCA, Open Label
Brief summary
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
Detailed description
The study will include approximately 520 adult and adolescent participants (≥ 12 years of age) with SCD. All participants will receive inclacumab 30 mg/kg administered intravenously every 12 weeks.
Interventions
DRUGInclacumab
Inclacumab will be supplied in single use 10 mL vials at a concentration of 50 mg/mL. One vial contains 500 mg of inclacumab. This is a liquid concentrate for IV infusion.
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Participants who meet all the following criteria will be eligible for study enrollment: 1. Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study. 2. Participant has completed the originating inclacumab study within 30 calendar days of the Day 1 Visit. Participants who discontinued study drug in the originating study due to a non-study drug-related AE, but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with inclacumab. 3. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become of childbearing potential during the study must be willing to have a negative urine pregnancy test to remain in the study. 4. If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 165 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 165 days after the last dose of study drug. 5. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirement.
Exclusion criteria
Participants meeting any of the following
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events (TEAEs). | Day 1 through study completion, an estimate of 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Annualized rate of VOCs | Day 1 through study completion, an estimate of 5 years |
| Annualized rate of VOCs that require admission to a healthcare facility and treatment. | Day 1 through study completion, an estimate of 5 years |
| Annualized number of days of inpatient hospitalization for a VOC. | Day 1 through study completion, an estimate of 5 years |
| Annualized rate of all SCD-related urgent care visits to the clinic, emergency room, and hospital. | Day 1 through study completion, an estimate of 5 years |
| Proportion of total days missed from school or work due to SCD pain symptoms for the first 48 weeks. | Day 1 through study completion, an estimate of 5 years |
| Annualized rate of complicated VOCs. | Day 1 through study completion, an estimate of 5 years |
| Annualized rate of RBC transfusions. | Day 1 through study completion, an estimate of 5 years |
Other
| Measure | Time frame | Description |
|---|---|---|
| PD parameter (P-selectin inhibition) | Day 1 through Week 48 | — |
| PD parameter (Platelet Leukocyte Aggregation) | Day 1 through Week 48 | — |
| Plasma pharmacokinetic (PK) of inclacumab as assessed by population PK analysis using nonlinear mixed-effects modeling. | Day 1 through Week 48 | Inclacumab concentrations will be measured from plasma samples. Population PK analysis using nonlinear mixed effects modeling will be performed to characterize inclacumab PK in plasma. |
| Incidence of anti-drug antibodies (ADA) to inclacumab. | Day 1 through Week 48 | — |
Countries
Brazil, Colombia, Kenya, Lebanon, Nigeria, Oman, Saudi Arabia, Tanzania, Turkey (Türkiye), United States
Outcome results
None listed