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Voxelotor Sickle Cell Exercise Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04581356
Recruitment Status : Recruiting
First Posted : October 9, 2020
Last Update Posted : October 12, 2020
Sponsor:
Collaborators:
Mednax Center for Research, Education, Quality and Safety
University of California, San Francisco
Global Blood Therapeutics
Information provided by (Responsible Party):
Elizabeth Yang, MD, PhD, Pediatric Specialists of Virginia

Tracking Information
First Submitted Date  ICMJE September 11, 2020
First Posted Date  ICMJE October 9, 2020
Last Update Posted Date October 12, 2020
Actual Study Start Date  ICMJE September 21, 2020
Estimated Primary Completion Date August 20, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 2, 2020)
Peak oxygen consumption (VO2) [ Time Frame: 8 weeks ]
Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 2, 2020)
  • Change in Biochemical markers of red cell sickling: Hemoglobin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Reticulocyte Count [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Bilirubin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH) [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Haptoglobin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: P50 oxygen dissociation [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Point of Sickling (POS) [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
  • Change in Biochemical markers of red cell sickling: Dense Cells [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: October 8, 2020)
  • HRQOL: Patient's Global Impression of Change (PGIC) [ Time Frame: 8 weeks ]
    Health-related quality of life (HRQoL) as assessed by patient reported outcome via Patient's Global Impression of Change (PGIC) at end of treatment study visit. The PGIC asks patients to rate their overall improvement relative to their baseline state at the beginning of the study on a 7-point scale.
    1. No change (or condition has got worse)
    2. Almost the same, hardly any change at all
    3. A little better, but not noticeable change at all
    4. Somewhat better, but the change has not made any real difference
    5. Moderately better, and a slight but noticeable change
    6. Better, and a definite improvement that has made a real and worthwhile difference
    7. A great deal better, and a considerable improvement that has made all the difference
  • HRQOL: Clinical Global Impression of Change (CGIC) [ Time Frame: 8 weeks ]
    Health-related quality of life (HRQoL) as assessed by clinician-reported outcome via Clinical Global Impression of Change-Improvement (CGIC-I) scale at end of treatment study visit. The CGIC-I is a one-item questionnaire that requires the clinician to assess how much the patient's illness has improved or worsened relative to their baseline state at the beginning of the intervention on a 7-point scale:
    1. = Very much improved
    2. = Much improved
    3. = Minimally improved
    4. = No change
    5. = Minimally worse
    6. = Much worse
    7. = Very much worse
Original Other Pre-specified Outcome Measures
 (submitted: October 2, 2020)
  • HRQOL: Patient's Global Impression of Change (PGIC) [ Time Frame: 8 weeks ]
    Health-related quality of life (HRQoL) as assessed by patient reported outcome via Patient's Global Impression of Change (PGIC) at end of treatment study visit
  • HRQOL: Clinical Global Impression of Change (CGIC) [ Time Frame: 8 weeks ]
    Health-related quality of life (HRQoL) as assessed by clinician-reported outcome via Clinical Global Impression of Change (CGIC) at end of treatment study visit
 
Descriptive Information
Brief Title  ICMJE Voxelotor Sickle Cell Exercise Study
Official Title  ICMJE The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia
Brief Summary This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).
Detailed Description

This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy.

Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
single-arm, open-label, pilot study in which each subject compared to him/herself
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Anemia
Intervention  ICMJE Drug: Voxelotor
daily voxelotor 1500mg oral medication
Other Name: GBT440, Oxbryta
Study Arms  ICMJE Experimental: voxelotor
Voxelotor 1500mg daily orally
Intervention: Drug: Voxelotor
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 2, 2020)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 20, 2021
Estimated Primary Completion Date August 20, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Provision of signed and dated informed consent form
  2. Stated willingness to comply with all study procedures and availability for the duration of the study
  3. Male or female, age > 12 years
  4. In good general health as evidenced by medical history and diagnosed with a genetically severe form of sickle cell anemia (Hgb SS, Hgb S beta 0 thalassemia, Hgb SCHarlem, and others)
  5. Patients who are on Hydroxyurea need to be on a stable dose for at least 3 months without anticipated change in dosing until the study is completed.
  6. Ability to take oral medication and willingness to adhere to daily voxelotor and 2 CPETs at scheduled intervals.
  7. For females of reproductive potential who are sexually active: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 30 days after the end of study.
  8. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner

Exclusion Criteria:

  1. Patients on chronic transfusions or who received a transfusion within last 8 weeks
  2. Patients who had hospitalization for vaso-occlusive crisis or acute chest syndrome within 30 days prior to informed consent/assent.
  3. Patients who have screening alanine aminotransferase (ALT) > 4X upper limit of normal
  4. Patients who suffer from physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
  5. Patients already taking commercially available voxelotor
  6. Prior hypersensitivity to voxelotor or excipients.
  7. Pregnant patients
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Elizabeth Yang, MD, PhD 571-472-1717 eyang@psvcare.org
Contact: Vivian Phan, MS vphan@psvcare.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04581356
Other Study ID Numbers  ICMJE ESR-C006
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Elizabeth Yang, MD, PhD, Pediatric Specialists of Virginia
Study Sponsor  ICMJE Elizabeth Yang, MD, PhD
Collaborators  ICMJE
  • Mednax Center for Research, Education, Quality and Safety
  • University of California, San Francisco
  • Global Blood Therapeutics
Investigators  ICMJE
Principal Investigator: Elizabeth Yang, MD, PhD. Pediatric Specialists of Virginia
Study Director: Vivian Phan, MS Pediatric Specialists of Virginia
Study Director: Kari Wheeler, BSN, RN Pediatric Specialists of Virginia
PRS Account Pediatric Specialists of Virginia
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP