Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

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).

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Drug: Voxelotor Phase 4

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
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
Official Title: The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia
Actual Study Start Date : September 21, 2020
Estimated Primary Completion Date : August 20, 2021
Estimated Study Completion Date : September 20, 2021


Arm Intervention/treatment
Experimental: voxelotor
Voxelotor 1500mg daily orally
Drug: Voxelotor
daily voxelotor 1500mg oral medication
Other Name: GBT440, Oxbryta




Primary Outcome Measures :
  1. Peak oxygen consumption (VO2) [ Time Frame: 8 weeks ]
    Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment


Secondary Outcome Measures :
  1. Change in Biochemical markers of red cell sickling: Hemoglobin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  2. Change in Biochemical markers of red cell sickling: Reticulocyte Count [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  3. Change in Biochemical markers of red cell sickling: Bilirubin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  4. Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH) [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  5. Change in Biochemical markers of red cell sickling: Haptoglobin [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  6. Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  7. Change in Biochemical markers of red cell sickling: P50 oxygen dissociation [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  8. Change in Biochemical markers of red cell sickling: Point of Sickling (POS) [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.

  9. Change in Biochemical markers of red cell sickling: Dense Cells [ Time Frame: 8 weeks ]
    Measured before and after treatment with voxelotor.


Other Outcome Measures:
  1. 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

  2. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT04581356


Contacts
Layout table for location contacts
Contact: Elizabeth Yang, MD, PhD 571-472-1717 eyang@psvcare.org
Contact: Vivian Phan, MS vphan@psvcare.org

Locations
Layout table for location information
United States, Virginia
Pediatric Specialist of Virginia Recruiting
Fairfax, Virginia, United States, 22031
Contact: Elizabeth Yang, MD, PhD         
Sponsors and Collaborators
Elizabeth Yang, MD, PhD
Mednax Center for Research, Education, Quality and Safety
University of California, San Francisco
Global Blood Therapeutics
Investigators
Layout table for investigator information
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
  Study Documents (Full-Text)

Documents provided by Elizabeth Yang, MD, PhD, Pediatric Specialists of Virginia:
Study Protocol  [PDF] June 5, 2020
Informed Consent Form  [PDF] July 23, 2020

Layout table for additonal information
Responsible Party: Elizabeth Yang, MD, PhD, Pediatric Hematologist, Director, Sickle Cell Program, Pediatric Specialists of Virginia
ClinicalTrials.gov Identifier: NCT04581356    
Other Study ID Numbers: ESR-C006
First Posted: October 9, 2020    Key Record Dates
Last Update Posted: October 12, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn