A Phase 3, Double-blind, Randomized, Placebo-controlled, MulticenterStudy of GBT440 Administered Orally to Patients With Sickle Cell Disease
- Conditions
- Sickle Cell DiseaseTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2016-003370-40-IT
- Lead Sponsor
- GLOBAL BLOOD THERAPEUTICS, INC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 274
1. Male or female study participants with Sickle Cell Disease:
• Documentation of SCD genotype (HbSS, HbSC, HbSß thalassemia or
other sickle cell syndrome variants) may be based on history of
laboratory testing or must be confirmed by
laboratory testing during screening
2. Participants have had at least 1 episode of VOC in the past 12 months.
For study eligibility, VOC is defined as a previously documented episode
of ACS or acute painful crisis (for which there was no explanation other
than VOC) which required prescription or healthcare professional instructed
use of analgesics for moderate to severe pain (documentation
must exist in the patient medical record prior to Screening)
3. Age 12 to 65 years
4. Hemoglobin (Hb) =6.0 and =10.5 g/dL during screening
5. Absolute reticulocyte count and % reticulocyte count must be >1.5 ×
ULN during Screening
6. For participants taking hydroxyurea (HU), the dose of HU (mg/kg)
must be stable for at least 3 months prior to signing the ICF and with no
anticipated need for dose adjustments during the study, in the opinion of the Investigator
7. Participants must demonstrate 75% compliance with ePRO measure
completion to be enrolled (participants will be given an ePRO device for
at least 28 days during Screening; participants who are 60 to 74%
compliant can re-screen once with Investigator approval; re-screening is
not allowed for participants who are <60% compliant)
8. Participants, who if female and of child bearing potential, are using
highly effective methods of contraception from study start to 3 months
after the last dose of study drug, and who if male are willing to use
barrier methods of contraception, from study start to 3 months after the
last dose of study drug
9. Participant has provided documented informed consent or assent (the
informed consent form [ICF] must be reviewed and signed by each
participant; in the case of pediatric participants, both the consent of the
participant's legal representative or legal guardian, and the participant's
assent must be obtained)
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 290
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. More than 10 VOCs within the past 12 months that required a hospital,
emergency room or clinic visit
2. Female who is breast feeding or pregnant
3. Patients who are receiving regularly scheduled blood (RBC)
transfusion therapy (also termed chronic, prophylactic, or preventive
transfusion) or have received a RBC transfusion for any reason within 28
days of signing the ICF
4. Hospitalized for sickle cell crisis or other vaso-occlusive event within
14 days of signing the ICF
(i.e., a vaso-occlusive event cannot be within 14 days prior to ICF)
5. Hepatic dysfunction characterized by alanine aminotransferase (ALT)
>4 × ULN
6. Participants with clinically significant bacterial, fungal, parasitic or
viral infection which require therapy:
• Participants with acute bacterial infection requiring antibiotic use
should delay screening/enrollment until the course of antibiotic therapy
has been completed.
• Participants with known active hepatitis A, B, or C or who are known to
be human immunodeficiency virus (HIV) positive
7. Severe renal dysfunction (estimated glomerular filtration rate at the
Screening visit; calculated by the central laboratory) <30mL/min/1.73²
or on chronic dialysis
8. History of malignancy within the past 2 years prior to treatment Day 1
requiring chemotherapy and/or radiation (with the exception of local
therapy for non-melanoma skin malignancy)
9. History of unstable or deteriorating cardiac or pulmonary disease
within 6 months prior to consent including but not limited to the
following:
• Unstable angina pectoris or myocardial infarction or elective coronary
intervention
• Congestive heart failure requiring hospitalization
• Uncontrolled clinically significant arrhythmias
10. Any condition affecting drug absorption, such as major surgery
involving the stomach or small intestine (prior cholecystectomy is
acceptable)
11. Participated in another clinical trial of an investigational agent (or
medical device) within 30 days or 5 half-lives of date of informed
consent, whichever is longer, or is currently participating in another trial
of an investigational agent (or medical device) 12. Inadequate venous access as determined by the Investigator/site
staff
13. Medical, psychological, or behavioral conditions, which, in the
opinion of the Investigator, may confound study interpretation, interfere
with compliance, or preclude informed consent
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method