Beta Thalassemia IntermediaPhase 2ST20-P2TNCT01609595
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
A Phase 2 study testing sodium 2,2 dimethylbutyrate in people with Beta Thalassemia Intermedia, run by Boston University at 1 site in Thailand.
Trial summary
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Beta Thalassemia Intermedia
- What is being tested?
- sodium 2,2 dimethylbutyrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteThailand
- Study dates
- Mar 2012to Dec 2012
- 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosis of Beta Thalassemia Intermedia
- Splenectomized
- Average of two Hgb levels between 6.0 and 9.0 g/dl
Who cannot join
- Red blood cell transfusion within 3 months of study drug initiation
- Enlarged spleen
- Use of hydroxyurea within 6 months
- QT Segment corrected (QTc)> 450 msec (men) or 470 msec (women) on screening ECG
- Use of iron chelating agents within 7 days of first dose
- Alanine Transaminase(ALT)> 4 times the upper limit of normal
- Use of erythropoiesis stimulating agents (ESAs) within 90 days of first dose
- serum creatinine > 1.5 mg/dL
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open Label Academic Phase 2 Study of SDMB in Subjects in Thailand With Beta Thalassemia Intermedia
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body. Beta thalassemias cause progressively severe anemia, widespread organ damage, and often require blood transfusions. There is no FDA approved therapeutic to treat the underlying cause of beta thalassemia. Fetal hemoglobin is another type of endogenous hemoglobin which can replace the reduced beta globin protein, reduce the anemia, and even abolish transfusion requirements. This type of hemoglobin is normally suppressed in infancy.
Sodium 2,2 dimethylbutyrate (ST20, or HQK-1001) is a small molecule which stimulates production of fetal hemoglobin in nonhuman primates and in human patients in Phase I/II trials.
This is a Phase 2 open-label trial to evaluate the ability of this oral therapeutic to reduce anemia in patients with beta thalassemia intermedia, when administered once daily for 26 weeks. All participants will receive the study drug.
Read the detailed description
This trial will:
- Determine the proportion of patients in which treatment with the study drug results in an increase in total hemoglobin by 1.5 g/dl above baseline levels when administered for 26 weeks in Thai patients with beta thalassemia intermedia, including Hemoglobin E beta thalassemia.
- Determine the number and proportion of participants in whom treatment with the study drug results in an increase in fetal hemoglobin.
- Determine the number of participants who have adverse events.
- Conditions
- Beta Thalassemia Intermedia
- Being tested
- sodium 2,2 dimethylbutyrate
- Keywords
- Thalassemia, Anemia, Fetal hemoglobin
- Medical subject headings
- beta-Thalassemia, Thalassemia, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodium 2,2 dimethylbutyrateDrug
Also called HQK-1001, ST20
Oral capsule, dose 20 mg/kg/day, daily, for 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Study drug treatment Receives: Drug: sodium 2,2 dimethylbutyrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of participants in which an increase in total hemoglobin of at least 1.5 g/dl above average baseline occurs with study drug treatment.
Baseline hemoglobin levels will be determined by averaging 2 values prior to administration of the study drug.
The number of participants in which an increase in total hemoglobin of at least 1.5 g/dL above baseline occurs will be determined.
Measured: Within 30 weeks, including 26 weeks of dosing with the study drug
Secondary outcomes (1)
The proportion of participants in which an increase in fetal hemoglobin occurs above the subjects' averaged baseline levels.
Tests of fetal hemoglobin will be obtained at two times prior to administration of the study drug and will be averaged. Laboratory tests of fetal hemoglobin will be assessed monthly during the study drug administration for 26 weeks, as a biomarker of drug activity.
Measured: Within 30 weeks, including 26 weeks of study drug administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2012 (estimated)
- Study startMar 2012
- Primary completionNov 2012
- Study completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Beta Thalassemia IntermediaStudies of the same condition, open and under way.
- More trials run by Boston UniversityThe sponsor's other registered studies.
- 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
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston University
- Sponsor type
- University, hospital or other
- Collaborators
- Mahidol University
- Responsible party
- Principal investigator, Susan P. Perrine, Boston University
- Investigators
- Suthat Fuchareon, MD, Principal investigator, Thalassemia Research Centre, Mahidol University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01609595
- Sponsor's protocol ID
- ST20-P2T
- Other IDs
- HQK-1001-Thal P2
- Acronym
- ST20-P2T
- Status last verified
- Mar 2013
- First submitted
- 25 Apr 2012
- Last updated
- 13 Mar 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Perrine SP, Castaneda SA, Chui DH, Faller DV, Berenson RJ, Siritanaratku N, Fucharoen S. Fetal globin gene inducers: novel agents and new potential. Ann N Y Acad Sci. 2010 Aug;1202:158-64. doi: 10.1111/j.1749-6632.2010.05593.x.
PubMed 20712788Perrine SP, Wargin WA, Boosalis MS, Wallis WJ, Case S, Keefer JR, Faller DV, Welch WC, Berenson RJ. Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteers. J Clin Pharmacol. 2011 Aug;51(8):1186-94. doi: 10.1177/0091270010379810. Epub 2011 Mar 21.
PubMed 21422239
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Academic Phase 2 Study of SDMB in Subjects in Thailand With Beta Thalassemia Intermedia
- ClinicalTrials.gov ID
- NCT01609595
- Acronym / sponsor ID
- ST20-P2T / ST20-P2T
- Conditions
- Beta Thalassemia Intermedia
- Keywords
- Thalassemia, Anemia, Fetal hemoglobin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Boston UniversityUniversity, hospital or other
- Collaborators
- Mahidol University
- Study start
- Mar 2012
- Primary completion
- Nov 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 1 Jun 2012 (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.
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body. Beta thalassemias cause progressively severe anemia, widespread organ damage, and often require blood transfusions. There is no FDA approved therapeutic to treat the underlying cause of beta thalassemia. Fetal hemoglobin is another type of endogenous hemoglobin which can replace the reduced beta globin protein, reduce the anemia, and even abolish transfusion requirements. This type of hemoglobin is normally suppressed in infancy.
Sodium 2,2 dimethylbutyrate (ST20, or HQK-1001) is a small molecule which stimulates production of fetal hemoglobin in nonhuman primates and in human patients in Phase I/II trials.
This is a Phase 2 open-label trial to evaluate the ability of this oral therapeutic to reduce anemia in patients with beta thalassemia intermedia, when administered once daily for 26 weeks. All participants will receive the study drug.
Detailed description
This trial will:
- Determine the proportion of patients in which treatment with the study drug results in an increase in total hemoglobin by 1.5 g/dl above baseline levels when administered for 26 weeks in Thai patients with beta thalassemia intermedia, including Hemoglobin E beta thalassemia.
- Determine the number and proportion of participants in whom treatment with the study drug results in an increase in fetal hemoglobin.
- Determine the number of participants who have adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: sodium 2,2 dimethylbutyrateStudy drug treatment |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Diagnosis of Beta Thalassemia Intermedia
- Splenectomized
- Average of two Hgb levels between 6.0 and 9.0 g/dl
Key exclusion criteria (8 listed)
- Red blood cell transfusion within 3 months of study drug initiation
- Enlarged spleen
- Use of hydroxyurea within 6 months
- QT Segment corrected (QTc)> 450 msec (men) or 470 msec (women) on screening ECG
- Use of iron chelating agents within 7 days of first dose
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 |
|---|---|---|
| sodium 2,2 dimethylbutyrateAlso: HQK-1001, ST20Oral capsule, dose 20 mg/kg/day, daily, for 26 weeks | Drug | Treatment |
Primary outcome
The number of participants in which an increase in total hemoglobin of at least 1.5 g/dl above average baseline occurs with study drug treatment.
Time frame: Within 30 weeks, including 26 weeks of dosing with the study drug
Baseline hemoglobin levels will be determined by averaging 2 values prior to administration of the study drug. The number of participants in which an increase in total hemoglobin of at least 1.5 g/dL above baseline occurs will be determined.
1 secondary outcome
The proportion of participants in which an increase in fetal hemoglobin occurs above the subjects' averaged baseline levels.
Time frame: Within 30 weeks, including 26 weeks of study drug administration
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 13 Mar 2013.
- Background: PubMed 20712788Perrine SP, Castaneda SA, Chui DH, Faller DV, Berenson RJ, Siritanaratku N, Fucharoen S. Fetal globin gene inducers: novel agents and new potential. Ann N Y Acad Sci. 2010 Aug;1202:158-64. doi: 10.1111/j.1749-6632.2010.05593.x.
- Background: PubMed 21422239Perrine SP, Wargin WA, Boosalis MS, Wallis WJ, Case S, Keefer JR, Faller DV, Welch WC, Berenson RJ. Evaluation of safety and pharmacokinetics of sodium 2,2 dimethylbutyrate, a novel short chain fatty acid derivative, in a phase 1, double-blind, placebo-controlled, single-dose, and repeat-dose studies in healthy volunteers. J Clin Pharmacol. 2011 Aug;51(8):1186-94. doi: 10.1177/0091270010379810. Epub 2011 Mar 21.
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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.
Suthat Fuchareon, MD principal investigator
Thalassemia Research Centre, Mahidol University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing sodium 2,2 dimethylbutyrate in people with Beta Thalassemia Intermedia, run by Boston University 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 3 inclusion and 8 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 13 Mar 2013. 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 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?
The number of participants in which an increase in total hemoglobin of at least 1.5 g/dl above average baseline occurs with study drug treatment. (time frame: Within 30 weeks, including 26 weeks of dosing with the study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01609595, which also lists 2 linked publications.
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 13 Mar 2013.