A SAD/MAD to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
| Status: | Recruiting |
|---|---|
| Conditions: | Healthy Studies, Anemia |
| Therapuetic Areas: | Hematology, Other |
| Healthy: | No |
| Age Range: | 12 - 60 |
| Updated: | 3/17/2019 |
| Start Date: | December 11, 2018 |
| End Date: | December 2019 |
| Contact: | Lindsey Wilson |
| Email: | lwilson@formarx.com |
| Phone: | 617-679-1970 |
A Randomized, Placebo-controlled, Double Blind, Single Ascending and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR)
being developed for the treatment of hemolytic anemias. This initial study will characterize
the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single
ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in
healthy volunteers and sickle cell disease patients. The effects of food on the absorption of
FT-4202 will also be evaluated in healthy volunteers.
being developed for the treatment of hemolytic anemias. This initial study will characterize
the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single
ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in
healthy volunteers and sickle cell disease patients. The effects of food on the absorption of
FT-4202 will also be evaluated in healthy volunteers.
This is a first-in-human (FIH), Phase 1 study of FT-4202 that will characterize the safety,
PK and PD of FT-4202 after a single dose and after repeated dosing first in healthy adult
volunteers and then in adolescents or adults with sickle cell disease (SCD). Initially, a
dose range of FT-4202 in single ascending dose (SAD) escalation cohorts will be explored in
healthy subjects. Enrollment of healthy subjects into 2-week multiple ascending dose (MAD)
escalation cohorts will be initiated once the safety and PK from at least two SAD cohorts is
available to inform the doses for the 2-week MAD portion of the study. The MAD cohorts will
then run in parallel to the single dose cohorts. A single dose cohort of healthy subjects is
planned to understand food effects (FE) on the PK of FT-4202. After the SAD and FE studies in
healthy subjects are completed, the safety, PK, and PD of a single dose of FT-4202 that was
found to be safe in healthy subjects will then be evaluated in SCD subjects. Multiple dose
studies in SCD subjects will then be initiated upon completion of MAD studies in healthy
volunteers.
PK and PD of FT-4202 after a single dose and after repeated dosing first in healthy adult
volunteers and then in adolescents or adults with sickle cell disease (SCD). Initially, a
dose range of FT-4202 in single ascending dose (SAD) escalation cohorts will be explored in
healthy subjects. Enrollment of healthy subjects into 2-week multiple ascending dose (MAD)
escalation cohorts will be initiated once the safety and PK from at least two SAD cohorts is
available to inform the doses for the 2-week MAD portion of the study. The MAD cohorts will
then run in parallel to the single dose cohorts. A single dose cohort of healthy subjects is
planned to understand food effects (FE) on the PK of FT-4202. After the SAD and FE studies in
healthy subjects are completed, the safety, PK, and PD of a single dose of FT-4202 that was
found to be safe in healthy subjects will then be evaluated in SCD subjects. Multiple dose
studies in SCD subjects will then be initiated upon completion of MAD studies in healthy
volunteers.
Inclusion Criteria:
- Healthy volunteer: subjects must be between 18 and 60 years of age; SCD: subjects must
be between 12 and 50 years of age
- Subjects must have the ability to understand and sign written informed consent, which
must be obtained prior to any study-related procedures being completed
- Subjects must be in general good health, based upon the results of medical history, a
physical examination, vital signs, laboratory profile, and a 12-lead ECG
- All males and females of child bearing potential must agree to use medically accepted
contraceptive regimen during study participation and up to 90 after
- Subjects must be willing to abide by all study requirements and restrictions
Exclusion Criteria:
- Evidence of clinically significant medical condition or other condition that might
significantly interfere with the absorption, distribution, metabolism, or excretion of
study drug, or place the subject at an unacceptable risk as a participant in this
study
- History of clinically significant cardiac diseases including condition disturbances
- Abnormal hematologic, renal and liver function studies
- History of drug or alcohol abuse
We found this trial at
1
site
Cincinnati, Ohio 45212
Principal Investigator: Lukasz Biernat, MD
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