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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03802201
Recruitment Status : Recruiting
First Posted : January 14, 2019
Last Update Posted : December 5, 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 . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.

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

Detailed Description:
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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 192 participants
Intervention Model: Sequential Assignment
Intervention Model Description:

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

  • Cohort 1: 3 mg subcutaneous (SC) weekly
  • Cohort 2: 10 mg SC weekly
  • Cohort 3: 20 mg SC weekly
  • Cohort 4a: 40 mg SC weekly
  • Cohort 4b: 40 mg SC every 2 weeks
  • Cohort 7: 80 mg SC weekly
  • Cohort 8: 40 mg SC twice a week

Additional Cohorts (Cohorts 5,6,9,10, and 11) will include subjects between 12 years of age 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 : August 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia Thalassemia

Arm Intervention/treatment
Experimental: PTG-300
PTG-300 Active
Drug: PTG-300
Subcutaneous (SC)
Other Name: PTG-300 Active




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. 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



Information from the National Library of Medicine

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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 screening with no transfusion free period > 45 days.
  2. Last RBC transfusion 5-10 days prior to dosing.

Main Exclusion Criteria:

  1. Subjects with Sickle Cell disease, Hgb H, Hb Bart's hydrops foetalis or hemoglobin S
  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. Subject has a concurrent clinically significant, unstable or uncontrolled cardiovascular, pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic or other medical disorder that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the subject by their participation in the study.
  4. Known primary or secondary immunodeficiency.
  5. History within 6 months of screening of any of the following:

    myocardial infarction, unstable angina, transient ischemic attack, decompensated heart failure requiring hospitalization, congestive heart failure (New York Heart Association Class 3 or 4), uncontrolled arrhythmias, cardiac revascularization, stroke, uncontrolled hypertension (resting systolic blood pressure [BP] > 160mmHg or resting diastolic BP > 100mmHg on more than one occasion) or uncontrolled diabetes (Hgb A1c > 9% or > one episode of severe hypoglycemia).

  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: Nishit Modi,, PhD 1-888-899-1543 ptgxclintrials@ptgx-inc.com

Locations
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Sponsors and Collaborators
Protagonist Therapeutics

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Responsible Party: Protagonist Therapeutics
ClinicalTrials.gov Identifier: NCT03802201    
Other Study ID Numbers: PTG-300-02
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: December 5, 2019
Last Verified: December 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