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Trial · Interventional

CompletedPhase 2Registry data

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

Short name TRANSCEND

Overview

Status
Completed
Phase
Phase 2
Enrollment
63 participants (actual)
Sponsor
Protagonist Therapeutics, Inc. · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
9 countries

United States, Greece, Italy, Lebanon, Malaysia, Thailand, Tunisia, Turkey (Türkiye), United Kingdom

Start
19 Dec 2018
Primary completion
31 Jul 2020
Completion
31 Jul 2020
First posted
14 Jan 2019
Last update posted
16 Jul 2021
Conditions
β-thalassemia · Ineffective Erythropoiesis · Chronic Anemia

Timeline

1 trial · 1 completed

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2018201920202021
  1. Ph 2

    TRANSCEND

    NCT03802201

    19 Dec 2018 to 31 Jul 2020

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • PTG-300Drug

Arms1

  • Experimental

    PTG-300 Active

    Drug: PTG-300 Subcutaneous

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. NTD: Proportion of responders at each dose

    Time frame · 4 week period

    Registry description

    NTD subjects who achieve an increase in Hgb without transfusion

  2. TD: Proportion of clinical responders at each dose

    Time frame · 8 week period

    Registry description

    TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

Completed trials do not always post results.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 12 Years up to 65 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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