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Sickle Cell DiseasePhase 2NCT06023199

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

A Phase 2 study testing Voxelotor in people with Sickle Cell Disease, run by Inova Health Care Services at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on… Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
Voxelotor
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
23 Oct 2023to 5 Mar 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • Male or female,
  • aged 18-65 years
  • Diagnosis of sickle cell disease with HbSS, HbSc, HbS-beta thalassemia genotypes
  • Hemoglobin level of 6 g/dL - 10.5 g/dL
Show all 11Show fewer
  • Between 0 and 10 vaso-occlusive (VOC) episodes in previous 12 months
  • Ability to take oral medication and be willing to adhere to the voxelotor regimen
  • For females of reproductive potential, they will be educated to avoid pregnancy during study period and for one month after due to limited data surrounding pregnancy and voxelotor
  • For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
  • Agreement to adhere to Lifestyle Considerations (see section 5.3) throughout study duration

Who cannot join

  • Hemoglobin results < 6 g/dL or > 10.5 g/dL
  • Patients receiving hydroxyurea who have had a dose change within the prior 3 months
  • Females who are breastfeeding or pregnant
  • Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) within 30 days of 6MWT
  • Any form of hepatic dysfunction as demonstrated by alanine aminotransferase (ALT) >4 × ULN or Child-Pugh Class C
  • Acute kidney injury or chronic renal disease with estimated glomerular filtration rate<30mL/min/1.73 m2
Show all 13Show fewer
  • Current or previous diagnosis of malignancy within the last two years excluding local therapy for non-melanoma skin malignancy
  • Unstable cardiopulmonary disease within the last 6 months prior to study enrollment
  • Any conditions including behavioral or medical that in the opinion of the investigator, may negatively impact informed consent, participation and study interpretation
  • Current use of commercially available voxelotor
  • Prior hypersensitivity to voxelotor or its excipients
  • Patients with baseline gait and other physical impairments that would limit 6MWT and other functional assessment at discretion of the investigator
  • Patients who on day of assessment present with vaso-occlusive crisis or rate their pain greater than 6 on a scale of 1-10 using the Numeric Rating Scale will not be assessed that day and re-screened at the investigator's discretion.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Inova Schar Cancer Institute

    Falls Church, Virginia, 22042

Other trials in United States

About this study

Official title: Assessing Functional Capacity in Sickle Cell Patients Receiving Voxelotor

Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment.

Being tested
Voxelotor
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VoxelotorDrug

    Also called Oxbryta

    Voxelotor is administered orally at a dose of 1500mg daily.

Study groups in NCT06023199
Study groupRoleWhat they receive
VoxeletorExperimentalNot described
Receives: Drug: Voxelotor

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Participant's Physical Performance Using 6 Minutes Walk Test (6MWT) Pre and Post Treatment with Voxelotor.

    The 6MWT is a sensitive objective assessment of functional exercise capacity which assess distance walked over 6 minutes.This test detects minor and moderate functional impairment resulting from treatment with Voxelotor.

    Measured: 6 months from the treatment initiation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Sep 2023
  2. Study start23 Oct 2023
  3. Primary completion17 Sep 2025
  4. Study completion5 Mar 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06023199
Sponsor's protocol ID
U23-02-4988
Status last verified
Aug 2026
First submitted
7 Aug 2023
Last updated
31 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Eisenstaedt R, Penninx BW, Woodman RC. Anemia in the elderly: current understanding and emerging concepts. Blood Rev. 2006 Jul;20(4):213-26. doi: 10.1016/j.blre.2005.12.002. Epub 2006 Feb 10.

    PubMed 16472893

  • Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.

    PubMed 31199090

  • Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.

    PubMed 33838113

  • Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.

    PubMed 30655275

  • Inusa BPD, Hsu LL, Kohli N, Patel A, Ominu-Evbota K, Anie KA, Atoyebi W. Sickle Cell Disease-Genetics, Pathophysiology, Clinical Presentation and Treatment. Int J Neonatal Screen. 2019 May 7;5(2):20. doi: 10.3390/ijns5020020. eCollection 2019 Jun.

    PubMed 33072979

  • Grosse SD, Odame I, Atrash HK, Amendah DD, Piel FB, Williams TN. Sickle cell disease in Africa: a neglected cause of early childhood mortality. Am J Prev Med. 2011 Dec;41(6 Suppl 4):S398-405. doi: 10.1016/j.amepre.2011.09.013.

    PubMed 22099364

  • Piel FB, Patil AP, Howes RE, Nyangiri OA, Gething PW, Dewi M, Temperley WH, Williams TN, Weatherall DJ, Hay SI. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013 Jan 12;381(9861):142-51. doi: 10.1016/S0140-6736(12)61229-X. Epub 2012 Oct 25.

    PubMed 23103089

  • Anie KA, Green J. Psychological therapies for sickle cell disease and pain. Cochrane Database Syst Rev. 2002;(2):CD001916. doi: 10.1002/14651858.CD001916.

    PubMed 12076428

  • Crawford J, Cella D, Cleeland CS, Cremieux PY, Demetri GD, Sarokhan BJ, Slavin MB, Glaspy JA. Relationship between changes in hemoglobin level and quality of life during chemotherapy in anemic cancer patients receiving epoetin alfa therapy. Cancer. 2002 Aug 15;95(4):888-95. doi: 10.1002/cncr.10763.

    PubMed 12209734

  • Corona LP, Andrade FCD, da Silva Alexandre T, de Brito TRP, Nunes DP, de Oliveira Duarte YA. Higher hemoglobin levels are associated with better physical performance among older adults without anemia: a longitudinal analysis. BMC Geriatr. 2022 Mar 21;22(1):233. doi: 10.1186/s12877-022-02937-4.

    PubMed 35313814

  • Yenamandra A, Marjoncu D. Voxelotor: A Hemoglobin S Polymerization Inhibitor for the Treatment of Sickle Cell Disease. J Adv Pract Oncol. 2020 Nov-Dec;11(8):873-877. doi: 10.6004/jadpro.2020.11.8.7. Epub 2020 Nov 1.

    PubMed 33489427

  • Agarwala P, Salzman SH. Six-Minute Walk Test: Clinical Role, Technique, Coding, and Reimbursement. Chest. 2020 Mar;157(3):603-611. doi: 10.1016/j.chest.2019.10.014. Epub 2019 Nov 2.

    PubMed 31689414

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Voxelotor in people with Sickle Cell Disease, run by Inova Health Care Services at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Participant's Physical Performance Using 6 Minutes Walk Test (6MWT) Pre and Post Treatment with Voxelotor. (time frame: 6 months from the treatment initiation). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06023199, which also lists 12 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2026.

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