A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Safety and Efficacy of Inclacumab in Participants with Sickle Cell Disease Experiencing Vaso-occlusive Crises

Phase 3

Recruiting

• Conditions: Patients with a diagnosis of sickle cell disease who have experienced between 2 and 10 vaso-occlusive crises in the 12 months preceding enrollment in this study.; SCD, vaso-occlusive crisis, blood disorders, hemoglobin, red blood cells, sickle-like shape, mutation in hemoglobin gene, sickle-cell trait, sickle-cell crisis, Sickle Cell Disease SCD, Hydroxyurea/ Hydroxycarbamide Therapy, SCA

• Registration Number: LBCTR2021064791
• Lead Sponsor: Global Blood Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-05-23
• Study Completion: 2024-01-31
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 240

Inclusion Criteria

Inclusion Criteria:
1. Participant has a confirmed diagnosis of SCD (HbSS, HbSC, HbSß0
thalassemia, or HbSß+ thalassemia genotype).
Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing
during Screening.
2. Participant is male or female, = 12 years of age at the time of informed
consent.
NOTE: Initial study enrollment will include participants = 16 years of age until the DMC determines that adequate safety and PK data support the
enrollment of participants 12 to 15 years of age. Sites will be informed by the Sponsor when participants 12 to 15 years of age may be enrolled.
3. Participant has experienced between 2 and 10 VOCs within the 12 months prior to the Screening Visit as determined by documented medical history.
A prior VOC is defined as an acute episode of pain which:
- Has no medically determined cause other than a vaso-occlusive event,
and
- Results in a visit to a medical facility (hospital, emergency department, urgent care center, outpatient clinic, or infusion center) or
results in a remote contact with a healthcare provider; and
- Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral
narcotics.
4. Participants receiving erythropoiesis-stimulating agents (ESA, erythropoietin [EPO]) must be on a stable dose for at least 90 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.
5. Participants receiving HU, L-glutamine, or voxelotor (Oxbryta) must be on a stable dose for at least 30 days prior to the Screening Visit and expected to
continue with the stabilized regimen throughout the course of the study.
6. Participant has adequate venous access, in the opinion of the Investigator, to comply with study procedures.
7. Participant understands the study procedures and agrees to participate in the study by giving written informed consent or parental permission/written assent.
8. Women of childbearing potential (WOCBP) are required to have a
negative serum pregnancy test at the Screening visit and negative urine
pregnancy test on all subsequent clinic visits and must agree to use a
highly effective method of contraception throughout the study period and
for at least 165 days after dosing.
Female participants will not be considered of childbearing potential if
they are pre-menarchal, surgically sterile (hysterectomy, bilateral
salpingectomy, tubal ligation, or bilateral oophorectomy) or
postmenopausal (no menses for 12 months without an alternative medical
cause, confirmed by follicle-stimulating hormone test results).

Exclusion Criteria

Exclusion Criteria
Candidates will be excluded from study entry if any of the following exclusion criteria exist at
Screening or Baseline visits or at the timepoint specified in the individual criterion listed.
1. Participant is receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion).
2. Participant is taking or has received crizanlizumab (ADAKVEO) within 90 days prior to the Screening Visit.
3. Participant weighs > 133 kg (292 lbs.).
4. Participant has a significant active and poorly controlled (unstable) hepatic disorder clearly unrelated to SCD
5. Participant has any of the following laboratory values at screening:
a. Absolute neutrophil count (ANC) < 1.0 × 109/L
b. Platelet count < 80 × 109/L
c. Hemoglobin < 4.0 g/dL for adults and < 5.0 g/dL for participants ages 12 to < 18 years of age
d. Estimated glomerular filtration rate (eGFR) < 30 mL/min using Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) formula in adults, and Schwartz formula in adolescents
6. Participant has known active (symptomatic) COVID infection or tests positive for COVID-19 during Screening.
7. Participant has a history of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including severe or unstable pulmonary hypertension.
8. Participant has had treatment for a malignancy within the 12 months prior to the Screening Visit (except non-melanoma skin cancer and in situ cervical cancers).
9. Participant has had a stroke within the 2 years prior to the Screening Visit.
10. Participant has a positive test indicative of an active malaria infection at Screening. Testing to be conducted at local laboratories in malaria-endemic regions at the discretion of the Investigator.
11. Participant has any confirmed clinically significant drug allergy and/or known hypersensitivity to monoclonal antibody therapeutics or formulation components of the study drug or a related drug.
12. Participant has been in another investigational trial within 30 days or 5 half-lives of the investigational agent (whichever is greater) prior to the Screening Visit.
13. Participant has had a major surgery within 8 weeks prior to the Screening Visit.
14. Participant is pregnant, breastfeeding, or planning to become pregnant during the 48-week treatment period.
15. Participant, parent, or legal guardian are unlikely to comply with the study procedures.
16. Participant has other medical, psychological, or addictive condition that, in the opinion of
the Investigator, would confound or interfere with evaluation of safety, efficacy, and/or PK of the investigational drug; prevent compliance with the study protocol; preclude informed consent; or render the participant, parent, or caretaker unable/unlikely to
comply with the study procedures.

Study & Design

• Study Type: Interventional
• Study Design: Not specified