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NCT03815695

A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients

Completed Phase 1 Last updated 10 April 2024
What this trial tests

Phase 1 trial testing FT-4202/Placebo in Healthy Volunteers in 130 participants. Completed in 17 December 2021.

Timeline
11 December 2018
Primary endpoint
17 December 2021
17 December 2021

Quick facts

Lead sponsorForma Therapeutics, Inc.
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designsequential
Maskingquadruple
Primary purposetreatment
Enrollment130
Start date11 December 2018
Primary completion17 December 2021
Estimated completion17 December 2021
Sites18 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Forma Therapeutics, Inc. — full company profile →

Who can join

Adults 12 to 65, any sex, with Healthy Volunteers or Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Drug Therapies for the Management of Sickle Cell Disease.
    Rai P, Ataga KI. · · 2020 · cited 32× · PMID 32765834 · DOI 10.12688/f1000research.22433.1
  2. FT-4202, an oral PKR activator, has potent antisickling effects and improves RBC survival and Hb levels in SCA mice.
    Shrestha A, Chi M, Wagner K, Malik A, et al · · 2021 · cited 26× · PMID 33944896 · DOI 10.1182/bloodadvances.2020003604
  3. Sickle cell disease: progress towards combination drug therapy.
    Pace BS, Starlard-Davenport A, Kutlar A. · · 2021 · cited 24× · PMID 33471938 · DOI 10.1111/bjh.17312
  4. Safety, Pharmacokinetics, and Pharmacodynamics of Etavopivat (FT-4202), an Allosteric Activator of Pyruvate Kinase-R, in Healthy Adults: A Randomized, Placebo-Controlled, Double-Blind, First-in-Human Phase 1 Trial.
    Forsyth S, Schroeder P, Geib J, Vrishabhendra L, et al · · 2022 · cited 23× · PMID 35019238 · DOI 10.1002/cpdd.1058
  5. Management of Sickle Cell Disease Complications Beyond Acute Chest Syndrome.
    Ogu UO, Badamosi NU, Camacho PE, Freire AX, et al · · 2021 · cited 23× · PMID 33658881 · DOI 10.2147/jbm.s291394
  6. Pyruvate Kinase Deficiency: Current Challenges and Future Prospects.
    Fattizzo B, Cavallaro F, Marcello APML, Vercellati C, et al · · 2022 · cited 20× · PMID 36072510 · DOI 10.2147/jbm.s353907
  7. Research in Sickle Cell Disease: From Bedside to Bench to Bedside.
    Salinas Cisneros G, Thein SL. · · 2021 · cited 19× · PMID 34095767 · DOI 10.1097/hs9.0000000000000584
  8. Fatty acid nitroalkenes - Multi-target agents for the treatment of sickle cell disease.
    Chowdhury FA, Colussi N, Sharma M, Wood KC, et al · · 2023 · cited 12× · PMID 37907055 · DOI 10.1016/j.redox.2023.102941

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Other recruiting trials for Healthy Volunteers

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03815695.

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