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

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ClinicalTrials.gov Identifier: NCT03943615
Expanded Access Status : Available
First Posted : May 9, 2019
Last Update Posted : May 9, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

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

Condition or disease Intervention/treatment
Sickle Cell Disease Drug: Voxelotor

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.


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Study Type : Expanded Access
Expanded Access Type : Individual Patients
Official Title: An Open-Label, Expanded Access Protocol for Patient With Sickle Cell Disease Who Have No Alternative Treatment Options



Intervention Details:
  • Drug: Voxelotor
    Other Name: GBT440

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03943615


Contacts
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Contact: Carolyn Hoppe, MD 650-741-7741 choppe@gbt.com
Contact: Aimee Enriquez 650-741-7777 aenriquez@gbt.com

Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Jonathan Sorof, MD Global Blood Therapeutics, Inc.

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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03943615     History of Changes
Other Study ID Numbers: GBT440-037
First Posted: May 9, 2019    Key Record Dates
Last Update Posted: May 9, 2019
Last Verified: May 2019

Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn