β-thalassemiaPhase 2TRANSCENDNCT03802201
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
A Phase 2 study testing PTG-300 in people with β-thalassemia and Ineffective Erythropoiesis, run by Protagonist Therapeutics, Inc. at 33 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- What is being tested?
- PTG-300
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 33 sitesin 9 countries
- Study dates
- 19 Dec 2018to 31 Jul 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Main Inclusion Criteria:
- Male and female subjects aged 18 to 65 years, inclusive (Cohorts 1-4b).
- Male and female subjects aged 12-<18 years, with a minimum weight of 30 kg (Cohorts 5 and 6).
- Documented diagnosis of β-thalassemia with no other Hgb abnormality.
Inclusion criteria applicable only for NTD β-thalassemia subjects:
- 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).
- 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:
- Transfusion requirement of at least 6 units of RBC in the 24 weeks prior to screening with no transfusion free period > 45 days.
- Last RBC transfusion 5-10 days prior to dosing.
Main Exclusion Criteria:
- Subjects with Sickle Cell disease, Hgb H, Hb Bart's hydrops foetalis or hemoglobin S
- 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).
- 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.
- Known primary or secondary immunodeficiency.
- 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).
- Pregnant or lactating females.
Trial locations and contacts
33 sites in 9 countries.
33 sites in 9 countries
Turkey (Türkiye)6 sites
Acibadem Adana Hospital
Adana, 01130
Hacettepe University Medical Faculty
Ankara, 06100
Akdeniz Universitesi Hastanesi
Antalya, 07059
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34093
Ege Universitesi Tip Fakultesi
Izmir, 35100
Mersin University Medical Facult
Mersin, 33343
Malaysia5 sites
Hospital Ampang
Ampang, 68000
Hospital Pulau Pinang
George Town, 10990
Hospital Sultanah Aminah
Johor Bahru, 80100
Queen Elizabeth Hospital
Kota Kinabalu, 88586
Hospital Umum Sarawak
Kuching, 93586
Thailand5 sites
Chulalongkorn University
Bangkok, 10330
Siriraj Hospital Mahidol University
Bangkok, 10700
Maharaj Nakorn Chiang Mai Chiang Mai University
Chiang Mai, 50200
Khon Kaen University
Khon Kaen, 40002
Naresuan University
Phitsanulok, 65000
Greece4 sites
Laiko General Hospital of Athens
Athens, Attica, 11562
Athens General Hospital 'G Gennimatas'
Athens, Attica
University General Hospital of Patras
Pátrai, Achaia
Hippokration Hospital
Thessaloniki
Tunisia4 sites
University Hospital Farhat Hached
Sousse, 4000
Bone Marrow Transplant Center
Tunis, 1006
Aziza Othmana Hospital
Tunis, 1008
Principal Military Hospital
Tunis, 1089
United States3 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
UCSF Benioff Children's Hospital
Oakland, California, 94609
Hospital of The University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Italy3 sites
Presidio Ospedaliero Antonio Perrino
Brindisi, 72100
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, 20122
AOU dell'Università degli Studi della Campania Luigi Vanvitelli
Naples, 80138
United Kingdom2 sites
Barts Health NHS Trust
London, E1 1BB
Barts Health NHS Trust
London
About this study
Official title: A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia
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.
Read the 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
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Being tested
- PTG-300
- Keywords
- chronic anemia, β-thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTG-300Drug
Subcutaneous (SC)
| Study group | Role | What they receive |
|---|---|---|
| PTG-300 Active | Experimental | Drug: PTG-300 Subcutaneous Receives: Drug: PTG-300 |
What is being measured
The primary outcome is the main question the study is designed to answer.
NTD: Proportion of responders at each dose
NTD subjects who achieve an increase in Hgb without transfusion
Measured: 4 week period
TD: Proportion of clinical responders at each dose
TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period
Measured: 8 week period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2019
- Study start19 Dec 2018
- Primary completion31 Jul 2020
- Study completion31 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β-thalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- 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)
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protagonist Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03802201
- Sponsor's protocol ID
- PTG-300-02
- Acronym
- TRANSCEND
- Status last verified
- Jul 2021
- First submitted
- 9 Jan 2019
- Last updated
- 16 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects With Chronic Anemia
- ClinicalTrials.gov ID
- NCT03802201
- Acronym / sponsor ID
- TRANSCEND / PTG-300-02
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Keywords
- chronic anemia, β-thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Protagonist Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Dec 2018 (actual)
- Primary completion
- 31 Jul 2020 (actual)
- Study completion
- 31 Jul 2020 (actual)
- First posted
- 14 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| PTG-300 Active | Experimental | Drug: PTG-300Drug: PTG-300 Subcutaneous |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PTG-300Subcutaneous (SC) | Drug | PTG-300 Active |
Primary outcome
NTD: Proportion of responders at each dose
Time frame: 4 week period
NTD subjects who achieve an increase in Hgb without transfusion
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
33 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTG-300 in people with β-thalassemia and Ineffective Erythropoiesis, run by Protagonist Therapeutics, Inc. at 33 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 33 sites in Turkey (Türkiye), Malaysia, Thailand, Greece, Tunisia and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
NTD: Proportion of responders at each dose (time frame: 4 week period). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2021.