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An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001

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ClinicalTrials.gov Identifier: NCT03041909
Recruitment Status : Completed
First Posted : February 3, 2017
Results First Posted : January 2, 2019
Last Update Posted : January 2, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition Sickle Cell Disease
Intervention Drug: GBT440
Enrollment 5
Recruitment Details Subjects enrolled in this study participated in GBT440-001 study (NCT02285088).
Pre-assignment Details There was no screening period as subjects transitioned directly from the GBT440-001 study (NCT02285088).into this study.
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088). The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088). This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
Period Title: Overall Study
Started 3 1 1
Completed 3 1 1
Not Completed 0 0 0
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months Total
Hide Arm/Group Description These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3). The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1). This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1). Total of all reporting groups
Overall Number of Baseline Participants 3 1 1 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 3 participants 1 participants 1 participants 5 participants
35
(25 to 42)
26 21
30
(21 to 42)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 1 participants 1 participants 5 participants
Female
0
   0.0%
0
   0.0%
1
 100.0%
1
  20.0%
Male
3
 100.0%
1
 100.0%
0
   0.0%
4
  80.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 1 participants 1 participants 5 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
3
 100.0%
1
 100.0%
1
 100.0%
5
 100.0%
White
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
Hide Description The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Time Frame 2 - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description:
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
Overall Number of Participants Analyzed 3 1 1
Measure Type: Count of Participants
Unit of Measure: Participants
2
  66.7%
1
 100.0%
1
 100.0%
2.Secondary Outcome
Title To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Hide Description Data presented are hemoglobin value collected at specific time points.
Time Frame 2 - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description:
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
Overall Number of Participants Analyzed 3 1 1
Measure Type: Number
Unit of Measure: g/dL
NA [1]  NA [1]  NA [1] 
[1]
Data is not reported due to participant privacy.
3.Secondary Outcome
Title To Observed Pharmacokinetics in Plasma and Whole Blood.
Hide Description Measure maximum plasma concentration (Cmax)
Time Frame 2 - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description:
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
Overall Number of Participants Analyzed 3 1 1
Measure Type: Number
Unit of Measure: ug/mL
NA [1]  NA [1]  NA [1] 
[1]
Data is not reported due to participant privacy.
4.Secondary Outcome
Title To Characterize the Effect of GBT440 on Hemolysis.
Hide Description Data presented for unconjugated bilirubin at specific time point.
Time Frame 2 - 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description:
These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).
Overall Number of Participants Analyzed 3 1 1
Measure Type: Number
Unit of Measure: umol/L
NA [1]  NA [1]  NA [1] 
[1]
Data is not reported due to participant privacy.
Time Frame 10 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Hide Arm/Group Description These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3). The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1). This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).
All-Cause Mortality
GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      0/1 (0.00%)      0/1 (0.00%)    
Hide Serious Adverse Events
GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/3 (0.00%)      1/1 (100.00%)      1/1 (100.00%)    
Blood and lymphatic system disorders       
Sickle Cell Anemia with Crisis * 1  0/3 (0.00%)  0 1/1 (100.00%)  1 0/1 (0.00%)  0
Surgical and medical procedures       
Cholecystectomy * 1  0/3 (0.00%)  0 0/1 (0.00%)  0 1/1 (100.00%)  1
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GBT440 - 2 Months GBT440 - 6 Months GBT440 - 4 Months
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/3 (66.67%)      1/1 (100.00%)      1/1 (100.00%)    
Gastrointestinal disorders       
Diarrhea * 1  1/3 (33.33%)  1 0/1 (0.00%)  0 1/1 (100.00%)  1
Toothache † 1  0/3 (0.00%)  0 1/1 (100.00%)  1 0/1 (0.00%)  0
Abdominal Pain † 1  1/3 (33.33%)  1 0/1 (0.00%)  0 0/1 (0.00%)  0
Gingival Swelling † 1  0/3 (0.00%)  0 1/1 (100.00%)  1 0/1 (0.00%)  0
Infections and infestations       
Upper Respiratory Tract Infection † 1  1/3 (33.33%)  1 0/1 (0.00%)  0 1/1 (100.00%)  1
Nervous system disorders       
Headache † 1  0/3 (0.00%)  0 0/1 (0.00%)  0 1/1 (100.00%)  1
1
Term from vocabulary, MedDRA (17.1)
*
Indicates events were collected by non-systematic assessment
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The sponsor and the PI will have to agree on any publications regarding the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Margaret Tonda, Sr. Director, Clinical Science
Organization: Global Blood Therapeutics
Phone: 650-741-7761
EMail: mtonda@gbt.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03041909    
Other Study ID Numbers: GBT440-024
First Submitted: January 19, 2017
First Posted: February 3, 2017
Results First Submitted: October 2, 2018
Results First Posted: January 2, 2019
Last Update Posted: January 2, 2019