β-thalassemiaPhase 2TRANSCENDNCT04054921
Safety Study for Beta Thalassemia Subjects on PTG-300
A Phase 2 study testing PTG-300 in people with β-thalassemia and Ineffective Erythropoiesis, run by Protagonist Therapeutics, Inc. at 16 sites in 8 countries.
Trial summary
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- β-thalassemia, Ineffective Erythropoiesis
- What is being tested?
- PTG-300
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 16 sitesin 8 countries
- Study dates
- 10 May 2019to 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
Who may be able to join
- NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.
Who cannot join
- Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).
Trial locations and contacts
16 sites in 8 countries.
16 sites in 8 countries
Thailand4 sites
Siriraj Hospital Mahidol University
Bangkok, 10700
Khon Kaen University
Khon Kaen, 40002
Chulalongkorn University
Pathum Wan, 10330
Naresuan University
Phitsanulok, 65000
Greece3 sites
Laiko General Hospital of Athens
Athens, 11526
Athens General Hospital 'G Gennimatas'
Athens, 11527
University General Hospital of Patras
Pátrai, 26500
United States2 sites
Boston Children's Hospital
Boston, Massachusetts, 02215
UCSF Benioff Children's Hospital
Oakland, California, 94609
Malaysia2 sites
Hospital Ampang
Ampang, 68000
Hospital Umum Sarawak
Kuching, 93586
Turkey (Türkiye)2 sites
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34093
Ege Universitesi Tip Fakultesi
Izmir, 35100
About this study
Official title: An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
Read the detailed description
The PTG-300-03 study will study beta thalassemia patients on PTG-300 for two years. The dose and dose frequency for each subject enrolling in the PTG-300-03 study will be based on the last dose and frequency received by the subject in the previous study PTG-300-02 and the subject's response at that dose. Patients dose will be increased in a manner similar to that used on PTG-300-02 study. The maximum PTG-300 dose allowed is 80 mg/week.
- Conditions
- β-thalassemia, Ineffective Erythropoiesis
- 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
PTG-300 is a hepcidin Mimetic
| Study group | Role | What they receive |
|---|---|---|
| Interventions | Experimental | PTG-300 Receives: Drug: PTG-300 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with side effects and severity of side effects will be tabulated
the long-term safety and tolerability of PTG-300 in Beta Thalassemia.
Measured: Over two year Period after receiving PTG-300
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2019
- Study start10 May 2019
- 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
- Single group
- Masking
- None (open label)
- Enrolment
- 34 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
- NCT04054921
- Sponsor's protocol ID
- PTG-300-03
- Acronym
- TRANSCEND
- Status last verified
- Feb 2021
- First submitted
- 8 Aug 2019
- Last updated
- 16 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
- An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects
- ClinicalTrials.gov ID
- NCT04054921
- Acronym / sponsor ID
- TRANSCEND / PTG-300-03
- Conditions
- β-thalassemia, Ineffective Erythropoiesis
- Keywords
- Chronic anemia, β-thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Protagonist Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 10 May 2019 (actual)
- Primary completion
- 31 Jul 2020 (actual)
- Study completion
- 31 Jul 2020 (actual)
- First posted
- 13 Aug 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.
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
Detailed description
The PTG-300-03 study will study beta thalassemia patients on PTG-300 for two years. The dose and dose frequency for each subject enrolling in the PTG-300-03 study will be based on the last dose and frequency received by the subject in the previous study PTG-300-02 and the subject's response at that dose. Patients dose will be increased in a manner similar to that used on PTG-300-02 study. The maximum PTG-300 dose allowed is 80 mg/week.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventions | Experimental | Drug: PTG-300PTG-300 |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.
Key exclusion criteria (1 listed)
- Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).
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-300PTG-300 is a hepcidin Mimetic | Drug | Interventions |
Primary outcome
Proportion of subjects with side effects and severity of side effects will be tabulated
Time frame: Over two year Period after receiving PTG-300
the long-term safety and tolerability of PTG-300 in Beta Thalassemia.
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.
- Individual participant dataSharing planned: No
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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.
16 sites in 8 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 16 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. 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 16 sites in Thailand, Greece, United States, Malaysia, Turkey (Türkiye) and 3 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?
Proportion of subjects with side effects and severity of side effects will be tabulated (time frame: Over two year Period after receiving PTG-300). 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.