Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Sickle Cell Disease Clinical Trial

Official title:

An Open-Label, Expanded Access Protocol for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT03943615
• Other study ID #: GBT440-037
• Secondary ID: C5341044
• Status: No longer available

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Voxelotor

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Augusta University	Augusta	Georgia
United States	The John Hopkins Hospital	Baltimore	Maryland
United States	Our Lady of the Lake Physician Group	Baton Rouge	Louisiana
United States	Alabama Oncology	Birmingham	Alabama
United States	Montefiore Medical Center PRIME	Bronx	New York
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Duke Department of Pediatrics	Durham	North Carolina
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Texas Children's Hospital	Houston	Texas
United States	Queens Hospital Center	Jamaica	New York
United States	University of South Alabama	Mobile	Alabama
United States	Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey
United States	University Medical Center New Orleans	New Orleans	Louisiana
United States	Thomas Jefferson University Hospital	Philadelphia	Pennsylvania
United States	University of Pittsburgh Medical Center Health System	Pittsburgh	Pennsylvania
United States	VCU Health	Richmond	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

External Links

•   To obtain contact information for a study center near you, click here.