- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT03041909
An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
An Open Label Single Arm Extension Study to Further Evaluate the Safety, Tolerability and Treatment Response of GBT440 in Patients With Sickle Cell Disease Who Participated in the Phase 1 Study GBT440-001
Studieöversikt
Detaljerad beskrivning
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001 (NCT02285088).
The primary objective of the study was to evaluate the safety and tolerability of up to a total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study (NCT02285088).
Studietyp
Inskrivning (Faktisk)
Fas
- Fas 2
Kontakter och platser
Studieorter
-
-
-
London, Storbritannien, SE1 9RT
- The BRC Research Facility, Floor 15 The Tower Wing
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Beskrivning
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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).
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Övrig
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Single Arm
Single Arm / open label
|
Oral drug
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
Tidsram: 2 - 6 months
|
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
|
2 - 6 months
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Tidsram: 2 - 6 months
|
Data presented are hemoglobin value collected at specific time points.
|
2 - 6 months
|
|
To Observed Pharmacokinetics in Plasma and Whole Blood.
Tidsram: 2 - 6 months
|
Measure maximum plasma concentration (Cmax)
|
2 - 6 months
|
|
To Characterize the Effect of GBT440 on Hemolysis.
Tidsram: 2 - 6 months
|
Data presented for unconjugated bilirubin at specific time point.
|
2 - 6 months
|
Samarbetspartners och utredare
Sponsor
Utredare
- Studierektor: Josh Lehrer-Graiwer, MD, Global Blood Therapeutics, Inc.
- Huvudutredare: Timothy Mant, FRCP, FFPM, Quintiles, Inc.
Publikationer och användbara länkar
Studieavstämningsdatum
Studera stora datum
Studiestart
Primärt slutförande (Faktisk)
Avslutad studie (Faktisk)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Uppskatta)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- GBT440-024
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
IPD-delning som stöder informationstyp
- Studieprotokoll
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