Voxelotor for Improving Oxygen Saturation in Adults

NCT05289570 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: Pro00109353
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to evaluate the efficacy of voxelotor for increasing oxygen saturation in 20 patients with hypoxemia. Specifically, the SpO2/FiO2 ratio will be compared before and after voxelotor use at rest and during exercise (ambulatory patients only). The primary study objective is to evaluate the efficacy of voxelotor for increasing oxygen saturation in patients with hypoxic hypoxemia as a result of end-stage lung disease or acute lung injury. The secondary objective is to evaluate the efficacy of voxelotor on allowing de-escalation of supplemental oxygen support.

Conditions

Acute Lung Injury; End Stage Lung Disease

Outcome Measures

Primary Outcomes (1)

• Change in SpO2/FiO2 (S/F) Ratio From Baseline to 2 Days After Initiation of Voxelotor Treatment

  SpO2 (oxygen saturation) is the percentage of oxygen in the blood. The fraction of inspired oxygen (FiO2) is the concentration of oxygen a person inhales.

  baseline to 2 days after initiation of voxelotor treatment

Secondary Outcomes (1)

• Change in SpO2/FiO2 (S/F) Ratio From Baseline to 5 Days After Initiation of Voxelotor Treatment

  baseline to 5 days after initiation of voxelotor treatment

Study Arms (1)

Voxelotor Arm

EXPERIMENTAL

500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor.

Drug: Voxelotor

Interventions

Voxelotor DRUG

500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).

Also known as: Oxbryta®

Voxelotor Arm

Eligibility Criteria

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

You may qualify if:

• Oxygen dependency due to an end-staged lung disease (pre-lung transplant population) or ALI/acute lung injury (for example but not limited to primary allograft dysfunction, infectious pneumonia, aspiration, non-cardiogenic pulmonary edema). ALI will be defined as per Berlin criteria with a P/F ratio \<100 denoting severe ARDS, \<200 denoting moderate and \<300 mild ARDS. ALI and mild ARDS are considered synonymous. In the event of inability to obtain arterial blood gas analysis to calculate a P/F ratio, we will consider a range of patients requiring standard nasal cannulae flowing at 6l/min to maintain SaO2 \>90% as ALI, and Salter High -Flow nasal cannulae at 12-15l/min in order to maintain SaO2\>85% as severe ARDS.
• At least 48 hours of stable, increased oxygen requirement or ventilatory support prior to the start of drug administration if consented.

You may not qualify if:

• Minors (\<18 years)
• Pre-existing congestive cardiac failure (NYHA III or IV)
• Medically significant, non-revascularized coronary artery disease
• Inability to obtain informed consent from LAR
• Pregnancy
• Incarcerated individual.
• Failure of another vital organ.
• Severe hepatic impairment (Childs-Pugh C) or liver enzymes \> 4x upper limit of normal (ULN) at screening.
• Unstable acute kidney injury/rising creatinine.
• Chronic neuromuscular disease requiring mechanical ventilation
• Not anticipated to survive \>48 hours
• Limited therapeutic goals (do not resuscitate, etc.)
• History of Pulmonary Embolism (PE)
• Requires treatment with Fluconazole or other moderate and strong CYP3A4 inhibitors listed in section 5.6
• A patient with active bleeding complications requiring more than 1 unit of blood transfusion per day, as the PK and PD of Voxelotor in the setting of blood loss and blood transfusion is unknown.

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Health System

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Acute Lung Injury

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Lung Injury; Lung Diseases; Respiratory Tract Diseases

Results Point of Contact

• Title: Ian Welsby, MD
• Organization: Duke University

ian.welsby@duke.edu

919-681-6752

Study Officials

• Ian Welsby

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 11, 2022
• First Posted: March 21, 2022
• Study Start: May 3, 2022
• Primary Completion: June 28, 2023
• Study Completion: July 4, 2023
• Last Updated: July 24, 2024
• Results First Posted: July 24, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)