Status:
COMPLETED
A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
Lead Sponsor:
Forma Therapeutics, Inc.
Collaborating Sponsors:
Medpace, Inc.
Conditions:
Healthy Volunteers
Sickle Cell Disease
Eligibility:
All Genders
12-65 years
Phase:
PHASE1
Brief Summary
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, toler...
Detailed Description
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 the...
Eligibility Criteria
Inclusion
- SCD Key
- Must be between 12 and 65 years of age
- Previously diagnosed sickle cell disease (hemoglobin electrophoresis or genotype)
- Must have a minimum body weight of 40 kg (88 lbs) at the Screening Visit
- Must have the ability to understand and sign written informed consent (and assent where applicable), which must be obtained prior to any study-related procedures being completed
- All male and female patients of child bearing potential must agree to use medically accepted contraceptive regimen during study participation and for 90 days after last study drug administration
- Must be willing to abide by all study requirements and restrictions
- SCD Key
Exclusion
- Had more than 6 episodes of vaso-occlusive crisis (VOC) within the past 12 months that required a hospital, emergency room, or clinic visit
- Had a least one episode of acute chest syndrome in the last 6 months
- Received any of the following approved therapies for use in SCD:
- Hydroxurea (HU): excluded if started HU \< 90 days prior to Day 1 of study treatment
- Adakveo®: excluded if received an infusion within 14 days prior to Day 1 of study treatment
- Oxbryta®: excluded if received a dose within 7 days prior to start of Day 1 of study treatment
- Received a red blood cell transfusion within 30 days of starting the study drug
- Hemoglobin \< 7.0 g/dL or \> 10.5 g/dL
- Unable to take and absorb oral medications
- HEALTHY VOLUNTEER Inclusion Criteria: \[Note: no longer recruiting subjects for this portion of the study\]
- Subjects must be between 18 and 60 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 days after
- Subjects must be willing to abide by all study requirements and restrictions
- HEALTHY VOLUNTEER
Key Trial Info
Start Date :
December 11 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 17 2021
Estimated Enrollment :
130 Patients enrolled
Trial Details
Trial ID
NCT03815695
Start Date
December 11 2018
End Date
December 17 2021
Last Update
April 10 2024
Active Locations (18)
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1
Woodland International Research Group (SCD subjects only)
Little Rock, Arkansas, United States, 72211
2
Collaborative Neuroscience Research, LLC (SCD subjects only)
Long Beach, California, United States, 90806
3
Pacific Research Partners (SCD subjects only)
Oakland, California, United States, 94607
4
UCSF Benioff Children's Hospital Oakland (SCD subjects only)
Oakland, California, United States, 94609