NCT03041909

Brief Summary

This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2016

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2016

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

January 19, 2017

Completed
15 days until next milestone

First Posted

Study publicly available on registry

February 3, 2017

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2017

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2017

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

January 2, 2019

Completed
Last Updated

January 2, 2019

Status Verified

December 1, 2018

Enrollment Period

10 months

First QC Date

January 19, 2017

Results QC Date

October 2, 2018

Last Update Submit

December 18, 2018

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.

    The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.

    2 - 6 months

Secondary Outcomes (3)

  • To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia

    2 - 6 months

  • To Observed Pharmacokinetics in Plasma and Whole Blood.

    2 - 6 months

  • To Characterize the Effect of GBT440 on Hemolysis.

    2 - 6 months

Study Arms (1)

Single Arm

EXPERIMENTAL

Single Arm / open label

Drug: GBT440

Interventions

GBT440DRUG

Oral drug

Single Arm

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Male or female subjects with SCD aged 18 to 60 years inclusive and \>50 kg who have participated in the GBT440-001 study.
  • Subjects, who if female and of child bearing potential, agree to continue to use highly effective methods of contraception prior to enrollment in this study and for 3 months after the last dose of study drug.
  • Subjects, who if male are willing to continue to use barrier methods of contraception, prior to enrollment in this study to 3 months after the last dose of study drug.

You may not qualify if:

  • Subjects requiring chronic transfusion therapy.
  • Subjects receiving a blood transfusion within 30 days of enrollment in this study.
  • Female subjects who are pregnant, trying to become pregnant or lactating.
  • Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, haematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders, or additional risk factors for torsades de pointe (e.g., heart failure, hypokalemia, personal or family history of long QTc interval).
  • Subjects who have a significant infection or known inflammatory process on admission to this study.
  • Subjects who have acute gastrointestinal symptoms at the time of admission (e.g. nausea, vomiting, diarrhoea, heartburn).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The BRC Research Facility, Floor 15 The Tower Wing

London, SE1 9RT, United Kingdom

Location

Related Publications (1)

  • 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: 30655275DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Margaret Tonda, Sr. Director, Clinical Science
Organization
Global Blood Therapeutics
mtonda@gbt.com
650-741-7761

Study Officials

  • Josh Lehrer-Graiwer, MD

    Global Blood Therapeutics, Inc.

    STUDY DIRECTOR

  • Timothy Mant, FRCP, FFPM

    Quintiles, Inc.

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

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

Study Record Dates

First Submitted

January 19, 2017

First Posted

February 3, 2017

Study Start

August 1, 2016

Primary Completion

June 1, 2017

Study Completion

August 1, 2017

Last Updated

January 2, 2019

Results First Posted

January 2, 2019

Record last verified: 2018-12

Data Sharing

IPD Sharing
Will share
Shared Documents
STUDY PROTOCOL

Locations

GB(1)