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

Study Overview
=================
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.

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

Conditions
-----------------
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease

Intervention / Treatment
-----------------
* Drug: Inclacumab


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Inclacumab 30 mg/kg<br>Inclacumab 30 mg/kg administered intravenously (IV) | Drug: Inclacumab<br>* 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.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events (TEAEs). |  | Day 1 through study completion, an estimate of 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
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