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

Primary Purpose

Sickle Cell Disease

Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Voxelotor
Sponsored by
Pfizer
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Sickle Cell Disease

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Alabama Oncology
  • University of South Alabama
  • Children's Healthcare of Atlanta
  • Augusta University
  • Our Lady of the Lake Physician Group
  • University Medical Center New Orleans
  • The John Hopkins Hospital
  • Rutgers Cancer Institute of New Jersey
  • Montefiore Medical Center PRIME
  • Queens Hospital Center
  • University of North Carolina at Chapel Hill
  • Duke Department of Pediatrics
  • The Ohio State University Wexner Medical Center
  • Thomas Jefferson University Hospital
  • University of Pittsburgh Medical Center Health System
  • Cook Children's Medical Center
  • Texas Children's Hospital
  • VCU Health

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
May 7, 2019
Last Updated
July 14, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT03943615
Brief Title
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

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Voxelotor
Other Intervention Name(s)
GBT440

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria Documented diagnosis of sickle cell disease of any genotype confirmed by laboratory testing during screening Ineligible or unable to participate in actively recruiting clinical studies of voxelotor Hemoglobin (Hb) ≤ 10.5 g/dL during screening No alternative treatment options in the judgment of the treating investigator 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 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 Female who is breast feeding or pregnant Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN 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 Renal dialysis Non-SCD conditions or complications of SCD for which survival beyond 3 months is unlikely, in the opinion of the treating Investigator Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable) 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) 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Alabama Oncology
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35211
Country
United States
Facility Name
University of South Alabama
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36693
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Augusta University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Our Lady of the Lake Physician Group
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70809
Country
United States
Facility Name
University Medical Center New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
The John Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Rutgers Cancer Institute of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901-1914
Country
United States
Facility Name
Montefiore Medical Center PRIME
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
Queens Hospital Center
City
Jamaica
State/Province
New York
ZIP/Postal Code
11432
Country
United States
Facility Name
University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Duke Department of Pediatrics
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Thomas Jefferson University Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
University of Pittsburgh Medical Center Health System
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
VCU Health
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States

12. IPD Sharing Statement

Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-037
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

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