Beta-ThalassemiaPhase 1NCT02950857
A Study of EPEG in Beta Thalassemia Patients
A Phase 1 study testing EPEG in people with Beta-Thalassemia, run by Prolong Pharmaceuticals at 1 site in Thailand.
Trial summary
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- EPEG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteThailand
- Study dates
- 28 Jun 2017to 27 Sep 2017
- Phase
- Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female
- Age 18 - 65 years of age
- Confirmed diagnosis of Non-Transfusion Dependent β-thalassemia (β-NTDT)
- Hemoglobin 6.0-10.0 g/dL
- Signed and dated informed written consent by the subject
- Able to receive subcutaneous injections of study drug
- Female patients must be non-lactating
- Female patients of reproductive potential must have a negative serum pregnancy (β-HCG) test at screening.
Who cannot join
- In the judgment of the investigator the patient is not a good candidate for the study
- Blood transfusion within the last 30 days
- Any of the following medical conditions:
- Severe kidney insufficiency, defined as use of hemodialysis or serum creatinine at levels greater than 2.5 mg/dL at the time of screening
- Cardiac disease with adjustment of cardiac medications in the 60 days before study entry
- Symptomatic coronary artery disease, as indicated by a history of chest pain, angina, claudication, or surgery to treat coronary artery disease in the 1 year before study entry
Show all 16Show fewer
- Stroke, defined as a new focal neurological deficit lasting more than 24 hours in the 45 days before study entry
- New diagnosis of pulmonary embolism by ventilation-perfusion scan, angiography, or any other technique in the 90 days before study entry
- History of retinal detachment or retinal hemorrhage in the 180 days before study entry
- Use of nitrate-based vasodilators, prostacyclin (inhaled, subcutaneous, or intravenous)
- Acute asthma exacerbation requiring use of prednisone in the 60 days before study entry
- Initiation or dosage increase of calcium channel blockers in the 30 days before study entry
- Initiation of any other cardiac or pulmonary medication in the 90 days before study entry
- Presence of any other condition, which in the opinion of the investigator, would make the person unsuitable for enrollment or could interfere with compliance in the study, including but not limited to alcohol or drug abuse
- Any prior treatment with Erythropoiesis-stimulating Agents (ESA) within 90 days of study treatment;
- History of hypersensitivity to erythropoietin or any related drug.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
Read the detailed description
Following the provision of informed consent, screening visit procedures to be performed will include: a detailed medical history (including concomitant medications), physical exam, vital signs (non-invasive systolic and diastolic arterial blood pressure, heart rate, and respiratory rate, temperature, and oxygen saturation by pulse oximetry,), laboratory testing of blood samples collections for safety (hematology and chemistry), and urinalysis (dipstick and microscopy, if necessary). A serum pregnancy test for all female patients (of child-bearing potential) will be measured during the Screening Visit. Urine pregnancy test for all female patients (of child-bearing potential) will be measured at each dosing visit prior to dosing.
Eligible patients will receive either subcutaneous injection of EPEG (0.9 µg/kg, 1.2 µg/kg, and 1.5 µg/kg,) for four weeks followed by follow up for 5-6 weeks after 4th dose of IP.
Vital signs will be recorded for study documentation at 1 hour after dosing and at discharge of the day (to occur 2 hours after the time of dosing). All patients will receive standard of care as per investigative site standard practice.
- Conditions
- Beta-Thalassemia
- Being tested
- EPEG
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPEGDrug
(Pegylated erythropoeitin)
| Study group | Role | What they receive |
|---|---|---|
| EPEG (pegylated erythropoietin) - 0.9 µg/kg | Experimental | Four weekly subcutaneous injections of 0.9 µg/kg EPEG Receives: Drug: EPEG |
| EPEG (pegylated erythropoietin) - 1.2 µg/kg | Experimental | Four weekly subcutaneous injections of 1.2 µg/kg EPEG Receives: Drug: EPEG |
| EPEG (pegylated erythropoietin) - 1.5 µg/kg | Experimental | Four weekly subcutaneous injections of 1.5 µg/kg EPEG Receives: Drug: EPEG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment.
Safety of treatment as determined by changes in vital signs, chest X-Ray, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events
Measured: 60 days
Secondary outcomes (4)
Change in the Hemoglobin from Baseline to the Final Visit
Measured: 60 Days
A mean increase in Hematocrit (Hct) from Baseline to the Final Visit
Measured: 60 Days
A mean increase in reticulocyte count from Baseline to the Final Visit
Measured: 60 Days
Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit
Measured: 60 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2016 (estimated)
- Study start28 Jun 2017
- Primary completion27 Sep 2017
- Study completion27 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prolong Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rosa Real, MD, Study director, Prolong Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02950857
- Sponsor's protocol ID
- PETH-001
- Status last verified
- May 2018
- First submitted
- 27 Oct 2016
- Last updated
- 25 May 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib, Open-label, Repeat Dose, Study of EPEG in Beta Thalassemia Patients With Non-transfusion Dependent Thalassemia (β NTDT)
- ClinicalTrials.gov ID
- NCT02950857
- Acronym / sponsor ID
- PETH-001
- Conditions
- Beta-Thalassemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Prolong PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Jun 2017 (actual)
- Primary completion
- 27 Sep 2017 (actual)
- Study completion
- 27 Sep 2017 (actual)
- First posted
- 1 Nov 2016 (estimated)
- 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.
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
Detailed description
Following the provision of informed consent, screening visit procedures to be performed will include: a detailed medical history (including concomitant medications), physical exam, vital signs (non-invasive systolic and diastolic arterial blood pressure, heart rate, and respiratory rate, temperature, and oxygen saturation by pulse oximetry,), laboratory testing of blood samples collections for safety (hematology and chemistry), and urinalysis (dipstick and microscopy, if necessary). A serum pregnancy test for all female patients (of child-bearing potential) will be measured during the Screening Visit. Urine pregnancy test for all female patients (of child-bearing potential) will be measured at each dosing visit prior to dosing.
Eligible patients will receive either subcutaneous injection of EPEG (0.9 µg/kg, 1.2 µg/kg, and 1.5 µg/kg,) for four weeks followed by follow up for 5-6 weeks after 4th dose of IP.
Vital signs will be recorded for study documentation at 1 hour after dosing and at discharge of the day (to occur 2 hours after the time of dosing). All patients will receive standard of care as per investigative site standard practice.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| EPEG (pegylated erythropoietin) - 0.9 µg/kg | Experimental | Drug: EPEGFour weekly subcutaneous injections of 0.9 µg/kg EPEG |
| EPEG (pegylated erythropoietin) - 1.2 µg/kg | Experimental | Drug: EPEGFour weekly subcutaneous injections of 1.2 µg/kg EPEG |
| EPEG (pegylated erythropoietin) - 1.5 µg/kg | Experimental | Drug: EPEGFour weekly subcutaneous injections of 1.5 µg/kg EPEG |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female
- Age 18 - 65 years of age
- Confirmed diagnosis of Non-Transfusion Dependent β-thalassemia (β-NTDT)
- Hemoglobin 6.0-10.0 g/dL
- Signed and dated informed written consent by the subject
Key exclusion criteria (16 listed)
- In the judgment of the investigator the patient is not a good candidate for the study
- Blood transfusion within the last 30 days
- Any of the following medical conditions:
- Presence of any other condition, which in the opinion of the investigator, would make the person unsuitable for enrollment or could interfere with compliance in the study, including but not limited to alcohol or drug abuse
- Any prior treatment with Erythropoiesis-stimulating Agents (ESA) within 90 days of study treatment;
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 |
|---|---|---|
| EPEG(Pegylated erythropoeitin) | Drug | EPEG (pegylated erythropoietin) - 0.9 µg/kg, EPEG (pegylated erythropoietin) - 1.2 µg/kg, EPEG (pegylated erythropoietin) - 1.5 µg/kg |
Primary outcome
Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment.
Time frame: 60 days
Safety of treatment as determined by changes in vital signs, chest X-Ray, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events
4 secondary outcomes
Change in the Hemoglobin from Baseline to the Final Visit
Time frame: 60 Days
A mean increase in Hematocrit (Hct) from Baseline to the Final Visit
Time frame: 60 Days
A mean increase in reticulocyte count from Baseline to the Final Visit
Time frame: 60 Days
Change in clinical signs and symptoms of β-NTDT from Baseline to the Final Visit
Time frame: 60 Days
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 25 May 2018.
- 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.
Rosa Real, MD study director
Prolong Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EPEG in people with Beta-Thalassemia, run by Prolong Pharmaceuticals at 1 site in Thailand.
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 8 inclusion and 16 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 25 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Thailand. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Number of participants in each treatment arm with abnormal laboratory values and/or adverse events that are related to treatment. (time frame: 60 days). 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 25 May 2018.