Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Study Overview
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Brief Summary
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The intent of this open-label, multicenter Expanded Access Program (EAP) is to provide early access to treatment with voxelotor prior to market authorization for pediatric patients age 6 months to 11 years with sickle cell disease (SCD) who have no alternative treatment options and are ineligible to participate in clinical trials of voxelotor. Cohort A, ages 4 to 11 years is now closed to enrollment.

Detailed Description
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Pediatric patients who are eligible for participation in this EAP will be treated with voxelotor, dispersible tablets or powder for oral suspension (Cohort A only) administered orally once daily (QD) at a weight-based dose, and followed by the treating physicians at the participating sites. Participants will receive standard of care treatment and procedures for management of SCD, including an initial visit and routine visits at least every 12 weeks (± 7 days) for clinical and laboratory assessments per standard of care and re-supply of voxelotor. A safety follow-up visit will be conducted 28 days (± 7 days) after the last dose of investigational product. This EAP may continue until such time that voxelotor is commercially available for patients aged 6 months to 11 years, or the Sponsor discontinues the voxelotor EAP.

Official Title
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An Open-label, Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: Voxelotor


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Documented diagnosis of sickle cell disease of any genotype Ineligible or unable to participate in actively recruiting clinical studies of voxelotor Baseline hemoglobin (Hb) ≤10.5 g/dL No alternative treatment options in the judgement of the treating Investigator Participants who, if female of childbearing potential (post-menarche), and are sexually active, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor Written informed parental/guardian consent and participant assent (if applicable) has been obtained per Institutional Review Board (IRB) policy and requirements, consistent with International Council for Harmonisation (ICH) guidelines Exclusion Criteria: Receiving chronic red blood cell (RBC) transfusion therapy for primary or secondary stroke prevention Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × the upper limit of normal (ULN) for age Severe renal dysfunction (estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m2 by Schwartz formula) Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy: Patients with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed. Patients with known active hepatitis A, B, or C or who are known to be Human Immunodeficiency Virus (HIV) positive Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable) Female who is pregnant or breastfeeding Participated in another clinical trial of an investigational drug or medical device, within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to informed consent for the EAP, or is currently participating in another trial of an investigational drug or medical device Medical, psychological, or behavioral conditions, that, in the opinion of the Investigator, may preclude informed consent, safe participation, or compliance with the protocol procedures Use of herbal medications (eg, St. John's wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or strong CYP3A4 inducers Active symptomatic COVID-19 infection

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Voxelotor|synthetic small molecule|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |