Clinical trial NCT03943615: Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Official title: An Open-Label, Expanded Access Protocol for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Sickle Cell Disease
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2019-05-09
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

Brief summary: The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization

Detailed description: The purpose of this program is to provide early access to voxelotor for patients with sickle cell disease who have exhausted alternative treatment options and are ineligible for actively-recruiting clinical trials of voxelotor.

This EAP may continue until such time that voxelotor is commercially available or the Sponsor discontinues the voxelotor EAP. Discontinuation may occur earlier if an individual participant or physician or Sponsor Medical Monitor decides to discontinue treatment.

Eligibility criteria: Inclusion Criteria

1. Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening
2. Ineligible or unable to participate in actively recruiting clinical studies of voxelotor
3. Hemoglobin (Hb) ≤ 10.5 g/dL during screening
4. No alternative treatment options in the judgment of the treating investigator
5. Participants, who if female and of child bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of voxelotor, and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of voxelotor
6. Patient has provided documented informed consent (the informed consent form \[ICF\] must be reviewed and signed by each participant),or for participants under 18 years of age, signed parental/legal guardian consent and participant assent.

Exclusion Criteria

1. Female who is breast feeding or pregnant
2. Hepatic dysfunction characterized by alanine aminotransferase (ALT) \>4 × ULN
3. Patients with clinically significant bacterial, fungal, parasitic, or viral infection which require 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
4. Renal dialysis
5. Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator
6. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
7. Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)
8. Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, interfere with compliance or preclude informed consent

The Sponsor Medical Monitor makes the sole and final enrollment determination for any patient.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Alabama Oncology
state: Alabama
city: Mobile
country: United States
facility: University of South Alabama
state: Alabama
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Baton Rouge
country: United States
facility: Our Lady of the Lake Physician Group
state: Louisiana
city: New Orleans
country: United States
facility: University Medical Center New Orleans
state: Louisiana
city: Baltimore
country: United States
facility: The John Hopkins Hospital
state: Maryland
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center PRIME
state: New York
city: Jamaica
country: United States
facility: Queens Hospital Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke Department of Pediatrics
state: North Carolina
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center Health System
state: Pennsylvania
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Richmond
country: United States
facility: VCU Health
state: Virginia
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: