Study to Evaluate Effects of Investigational Drug on Oxygenation in Healthy Volunteers Under Hypoxic Conditions

NCT03051711 | Terminated Phase 1

• 1 other identifier: GBT440-0111
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the effect of GBT440 on oxygen saturation at rest and exercise, under hypoxic conditions, at Day 15 compared to Baseline.

Conditions

Cardio-pulmonary Function

Outcome Measures

Primary Outcomes (1)

• The change in oxygen saturation (%) at rest and exercise, under hypoxic conditions

  Day 15

Secondary Outcomes (5)

• Maximal oxygen uptake VO2 max (mL/kg/min) under normoxic and hypoxic conditions

  Day 15

• Cardiac output (L/min) under normoxic and hypoxic conditions

  Day 15

• Oxy-hemoglobin dissociation curve p50, under hypoxic conditions

  Day 15

• Perceived dyspnea score (1-10), under normoxic and hypoxic conditions

  Day 15

• Frequency and severity of treatment-emergent adverse events (TEAEs) as assessed by (NCI CTCAE Version 4.03)

  Day 15

Study Arms (2)

GBT440 Dose 1

EXPERIMENTAL

Dose 1

Drug: GBT440

GBT440 Dose 2

EXPERIMENTAL

Dose 2

Drug: GBT440

Interventions

GBT440 DRUG

Capsules which contain GBT440 drug substance in Swedish Orange

Also known as: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde

GBT440 Dose 1; GBT440 Dose 2

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Males or females aged 18 - 50 years inclusive
• Able and willing to provide signed informed consent to participate in this study
• VO2 max ≥ 40 mL/kg/min for males, and ≥ 35 mL/kg/min for females
• Weight ≥ 40 kg
• Male or female of child bearing potential willing and able to use highly effective methods of contraception during the study to 30 days after the last dose of study drug.

You may not qualify if:

• Subjects whose exercise regimen at Screening is, in the Investigator's opinion, expected to change significantly during the study
• Family or personal history of congenital long QT syndrome
• Participated in another clinical trial of an investigational drug (or medical device) within 30 days or 5-half-lives, whichever is longer, prior to Screening, or is currently participating in another trial of an investigational drug (or medical device)
• Clinically significant medical disease that is likely, in the Investigator's opinion, to significantly impact the study's efficacy and safety assessments (e.g., history significant cardio-pulmonary disease or arrhythmias) within 6 months of Screening.
• AST, ALT or total bilirubin \>2 × ULN
• Serum creatinine \>1.5 mg/dL
• Clinical evidence of active infection, within 21 days of Screening, which may include but is not limited to bronchitis, pneumonia, urinary tract infection, or cellulitis.
• Female who is breast-feeding or pregnant
• Current smoker or history of smoking within 3 months from Screening
• Any condition possibly affecting drug absorption, including but not limited to previous surgery on the stomach or small intestine
• Known hypersensitivity to any component of the study drug

Sponsors & Collaborators

• Global Blood Therapeutics: lead
• Mayo Clinic: collaborator

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Interventions

voxelotor

Study Officials

• Ganesh Balaratnam, MBChB, BAO

  Global Blood Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: January 18, 2017
• First Posted: February 14, 2017
• Study Start: December 1, 2016
• Primary Completion: December 1, 2017
• Study Completion: February 21, 2018
• Last Updated: October 4, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)