A Phase 1b, Randomized, Double-Blind (Sponsor Open), Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PF-04447943, Co-Administered with and Without Hydroxyurea, in Subjects with Stable Sickle Cell Disease

A Phase 1b, Randomized, Double-Blind (Sponsor Open), Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PF-04447943, Co-Administered with and Without Hydroxyurea, in Subjects with Stable Sickle Cell Disease - PF-04447943 in SCD patients

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON44079

Enrollment

Registered

2015-06-01

Start date

2016-01-06

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle cell anemia

Interventions

Multiple doses of PF-04447943 or placebo.

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: - Stable SCD patients (Hbss or HbSB-thalasamia) - Male/female, age 18-65, BMI 17.5-35, incl (see protool p31)

Exclusion criteria

Exclusion criteria: - Recent Vaso occlusive crisis (

Design outcomes

Primary

Measure	Time frame
Safety will be assessed through adverse events, changes in laboratory results, changes in ECG measurements, and changes in vital sign measurements	—

Secondary

Measure	Time frame
Pharmacokinetic endpoints will include plasma PF-04447943 AUC(0-12h), C12h, Cmax, and Tmax on Days 1. C1h and C2h will also be measured on Days 7 to assess steady-state Cmax. Pharmacodynamic endpoints will include: Plasma cGMP Markers associated with cellular adhesion: E-selectin, P-selectin ICAM, VCAM Platelet-monocyte aggregates, platelet neutrophil aggregates MAC-1 expression on monocytes and neutrophils Markers associated with coagulation: Tissue Factor Thrombin-Antithrombin Complexes Prothrombin Fragments F1 + F2 D-dimers; Circulating endothelial microparticles Hemoglobin F	—

Measure

Time frame

Pharmacokinetic endpoints will include plasma PF-04447943 AUC(0-12h), C12h, Cmax, and Tmax on Days 1. C1h and C2h will also be measured on Days 7 to assess steady-state Cmax. Pharmacodynamic endpoints will include: Plasma cGMP Markers associated with cellular adhesion: E-selectin, P-selectin ICAM, VCAM Platelet-monocyte aggregates, platelet neutrophil aggregates MAC-1 expression on monocytes and neutrophils Markers associated with coagulation: Tissue Factor Thrombin-Antithrombin Complexes Prothrombin Fragments F1 + F2 D-dimers; Circulating endothelial microparticles Hemoglobin F

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)