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Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (TRANSCEND)

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ClinicalTrials.gov Identifier: NCT03802201
Recruitment Status : Recruiting
First Posted : January 14, 2019
Last Update Posted : April 22, 2019
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
β-thalassemia Ineffective Erythropoiesis Drug: PTG-300 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 84 participants
Intervention Model: Sequential Assignment
Intervention Model Description:

Five PTG-300 dose levels/regimens are planned to be tested for each subpopulation of β thalassemia (NTD and TD) on separate arms:

  • Cohort 1: low dose subcutaneous (SC) weekly
  • Cohort 2: low intermediate dose SC weekly
  • Cohort 3: high intermediate dose SC weekly
  • Cohort 4a: high dose SC weekly
  • Cohort 4b: high dose SC every 2 weeks

Two additional cohorts (Cohorts 5 and 6) will include subjects between 12 and less than 18 years of age.

Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia
Actual Study Start Date : December 19, 2018
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : July 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia Thalassemia

Arm Intervention/treatment
Experimental: PTG-300 low dose (Cohort 1) Drug: PTG-300
subcutaneous (SC) weekly

Experimental: PTG-300 low intermediate dose (Cohort 2) Drug: PTG-300
SC weekly

Experimental: PTG-300 high intermediate dose (Cohort 3) Drug: PTG-300
SC weekly

Experimental: PTG-300 high dose (Cohort 4a) Drug: PTG-300
SC weekly

Experimental: PTG-300 high dose (Cohort 4b) Drug: PTG-300
SC every 2 weeks




Primary Outcome Measures :
  1. NTD: Proportion of responders at each dose [ Time Frame: 4 week period ]
    NTD subjects who achieve an increase in Hgb without transfusion

  2. NTD: Mean Hgb change from baseline [ Time Frame: 4 week period ]
  3. TD: Proportion of clinical responders at each dose [ Time Frame: 8 week period ]
    TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period

  4. TD: Mean change from baseline in the number of units of RBC required under each dose [ Time Frame: 8 week period ]


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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03802201


Contacts
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Contact: Roya Nawabi +1 510 474 0170 r.nawabi@ptgx-inc.com
Contact: Frank Valone +1 510 474 0170 f.valone@ptgx-inc.com

Locations
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United States, California
UCSF Benioff Children's Hospital Recruiting
Oakland, California, United States, 94609
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
United States, Pennsylvania
Hospital of The University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Greece
University General Hospital of Patras Recruiting
Patras, Achaia, Greece
Laiko General Hospital of Athens Recruiting
Athens, Attiki, Greece, 11562
Athens General Hospital 'G Gennimatas' Active, not recruiting
Athens, Attiki, Greece
Hippokration Hospital Recruiting
Thessaloníki, Greece
Lebanon
Chronic Care Center Recruiting
Beirut, Lebanon
Thailand
Chulalongkorn University Recruiting
Bangkok, Thailand
Chulalongkorn University Recruiting
Pathum Wan, Thailand
United Kingdom
Barts Health NHS Trust Recruiting
London, United Kingdom
Sponsors and Collaborators
Protagonist Therapeutics

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Responsible Party: Protagonist Therapeutics
ClinicalTrials.gov Identifier: NCT03802201     History of Changes
Other Study ID Numbers: PTG-300-02
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: April 22, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Protagonist Therapeutics:
chronic anemia
β-thalassemia

Additional relevant MeSH terms:
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Anemia
Thalassemia
beta-Thalassemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn