for people ages 12-65 (full criteria)
at Oakland, California and other locations
study started
estimated completion



This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort and with the potential for individual titration (dose increase or decrease) within each cohort. The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment of chronic anemia in β-thalassemia and to evaluate the appropriate dosing regimen for PTG-300 in the target population.

Official Title

A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia


β-thalassemia Ineffective Erythropoiesis chronic anemia Anemia Thalassemia beta-Thalassemia PTG-300


For people ages 12-65

Main Inclusion Criteria:

  1. Male and female subjects aged 18 to 65 years, inclusive (Cohorts 1-4b).
  2. Male and female subjects aged 12-<18 years, with a minimum weight of 30 kg (Cohorts 5 and 6).
  3. Documented diagnosis of β-thalassemia with no other Hgb abnormality.

Inclusion criteria applicable only for NTD β-thalassemia subjects:

  1. Mean Hgb < 10.0 g/dL of two measurements (one performed 7-28 days prior to dosing and the other performed within 7 days prior to dosing).
  2. Requirement of < 6 units RBC transfusion in a 24 week period with the last transfusion at least 8 weeks before screening.

Inclusion criteria applicable only for TD β-thalassemia subjects:

  1. Transfusion requirement of at least 6 units of RBC in the 24 weeks prior to randomization with no transfusion free period > 45 days.
  2. Last RBC transfusion 5-10 days prior to dosing.

Main Exclusion Criteria:

  1. Subjects with the diagnosis of β-thalassemia major (genotype homozygous β0/β0 or compound heterozygous β0/β+ with a major phenotype).
  2. Infection requiring hospitalization or IV antimicrobial therapy, or opportunistic infection within 6 months of dosing, any infection requiring antimicrobial therapy within 2 weeks of dosing; history of infection with human immunodeficiency virus (HIV).
  3. Treatment with hydroxyurea ≤ 24 weeks prior to randomization.
  4. Use of erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to randomization.
  5. Chronic use of systemic glucocorticoids (anti-inflammatory dose for more than 14 days) ≤ 12 weeks prior to randomization (physiologic replacement therapy for adrenal insufficiency is allowed).
  6. Pregnant or lactating females.


  • UCSF Benioff Children's Hospital accepting new patients
    Oakland California 94609 United States
  • Hospital of The University of Pennsylvania accepting new patients
    Philadelphia Pennsylvania 19104 United States


accepting new patients
Start Date
Completion Date
Protagonist Therapeutics
Phase 2
Study Type
Last Updated