Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: U23-02-4988
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (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.

  6 months from the treatment initiation

Other Outcomes (1)

• The Change in the Quality of Life as assessed by Sheehan Disability Scale (SDS) During the 6 Months Trial Period.

  6 months from the treatment initiation

Study Arms (1)

Voxeletor

EXPERIMENTAL

Drug: Voxelotor

Interventions

Voxelotor DRUG

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

Also known as: Oxbryta

Voxeletor

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Pfizer: collaborator
• Inova Health Care Services: lead

Study Sites (1)

Inova Schar Cancer Institute

Falls Church, Virginia, 22042, United States

Location

Related Publications (12)

• 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.

  PMID: 16472893 BACKGROUND

• 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.

  PMID: 31199090 BACKGROUND

• 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.

  PMID: 33838113 BACKGROUND

• 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.

  PMID: 30655275 BACKGROUND

• 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.

  PMID: 33072979 BACKGROUND

• 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.

  PMID: 22099364 BACKGROUND

• 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.

  PMID: 23103089 BACKGROUND

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

  PMID: 12076428 BACKGROUND

• 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.

  PMID: 12209734 BACKGROUND

• 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.

  PMID: 35313814 BACKGROUND

• 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.

  PMID: 33489427 BACKGROUND

• 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.

  PMID: 31689414 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sheinei Alan

  Inova Schar Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2023
• First Posted: September 5, 2023
• Study Start: October 23, 2023
• Primary Completion: September 17, 2025
• Study Completion (Estimated): June 15, 2026
• Last Updated: February 2, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)