A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

Sponsor

Global Blood Therapeutics (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05348915

Collaborator

(none)

520

Enrollment

Locations

Arm

59.1

Anticipated Duration (Months)

173.3

Patients Per Site

2.9

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease Vaso-occlusive Crisis Vaso-occlusive Pain Episode in Sickle Cell Disease	Drug: Inclacumab	Phase 3

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

520 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial

Actual Study Start Date :

Mar 29, 2022

Anticipated Primary Completion Date :

Mar 1, 2027

Anticipated Study Completion Date :

Mar 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Inclacumab 30 mg/kg Inclacumab 30 mg/kg administered intravenously (IV)	Drug: Inclacumab 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.

Arm

Intervention/Treatment

Experimental: Inclacumab 30 mg/kg

Inclacumab 30 mg/kg administered intravenously (IV)

Drug: Inclacumab

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.

Outcome Measures

Primary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs). [Day 1 through study completion, an estimate of 5 years]

Secondary Outcome Measures

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 Outcome Measures

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]
PD parameter (P-selectin inhibition) [Day 1 through Week 48]
PD parameter (Platelet Leukocyte Aggregation) [Day 1 through Week 48]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Participants who meet all the following criteria will be eligible for study enrollment:

Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study.
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.
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.

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.
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 exclusion criteria will not be eligible for study enrollment:

Female participant who is breastfeeding or pregnant.
Participant had an infusions-related reaction (IRR) in the originating inclacumab clinical study.
Participant withdrew consent from the originating inclacumab clinical study.
Participant was lost to follow-up from the originating inclacumab clinical study.
Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.