Beta Thalassemia IntermediaPhase 2LB-04-THALNCT01642758
Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
A Phase 2 study testing Sodium 2,2 dimethylbutyrate in people with Beta Thalassemia Intermedia, run by Boston University at 1 site in Lebanon.
Trial summary
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Read more about this study
- Who is it for?
- Ages 16 to 50 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 siteLebanon
- Study dates
- May 2012to Jan 2013
- 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 16 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of beta thalassemia intermedia
- Ages 16-50 years
- Average total Hgb levels between 6.0 and 9.0 gm/dl within 30 days of initial dose of study drug
- Able to comply with all study procedures
- If female and of childbearing potential, must have a documented negative pregnancy test prior to entry and every 4 weeks
Who cannot join
- Red blood cell transfusions within 3 months prior to administration of study drug
- QT Segment corrected (QTc)> 450 msec
- Use of Erythropoiesis Stimulating Agents(ESAs)within 9 days of first dose
- Hydroxyurea treatment within 6 months of first study drug
- History of significant arrythmias, syncope, or resuscitation
- Alanine Transaminase (ALT)> 4x upper limit of normal
Show all 9Show fewer
- Serum creatinine > 1.5 mg/dl
- Sse of iron chelating agents within 7 days of first dose
- Pulmonary hypertension requiring oxygen therapy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label Phase 2 Study of HQK-1001 in Subjects With Beta Thalassemia Intermedia
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia.
This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.
Read the detailed description
This is a trial of an experimental oral medicine which stimulates production of fetal hemoglobin, an innate type of hemoglobin which is normally made but is suppressed in infancy. Fetal globin (HbF) can perform the function of the missing beta globin and reduce anemia in beta thalassemia, when it is produced in higher amounts than normal.
In this trial, 10 patients with beta thalassemia intermedia in Lebanon will all receive the study drug for 6 months at a dose which has been previously shown to be safe in normal volunteers and in beta thalassemia and sickle cell patients and to stimulate fetal globin production in many, when given for brief periods. The purpose of this trial is the following:
- To determine if total hemoglobin levels increase above baseline in some subjects when the study drug is taken for 26 weeks.
- To determine if fetal globin is increased above baseline levels in a proportion of subjects when the study drug is taken for 26 weeks.
- To determine the number of adverse events which occur with 26 weeks of administration of the study drug in beta thalassemia intermedia subjects.
After a screening period, the subjects will take the study drug at home once a day. They will be seen once every 4 weeks for examinations and laboratory tests during the dosing period and for 4 weeks afterwards.
This trial will provide an important step in evaluating a potential treatment for patients with beta thalassemia intermedia, that can be used around the world, if it is effective and safe.
- Conditions
- Beta Thalassemia Intermedia
- Being tested
- Sodium 2,2 dimethylbutyrate
- Keywords
- Thalassemia intermedia, Fetal hemoglobin, Hemoglobin
- Medical subject headings
- beta-Thalassemia
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 ST20
Oral capsules, dose 20 mg/kg/day, once per day for 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| Sodium 2,2 dimethylbutyrate | Experimental | A single dose (20 mg/kg/day) of study drug will be taken once per day by mouth. Receives: Drug: Sodium 2,2 dimethylbutyrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia.
Baseline hemoglobin levels will be determined in each subject and averaged from levels obtained on a screening visit and on day one of the study, before any drug is taken. Hemoglobin levels will then be analyzed every 4 weeks during 26 weeks of taking the study drug and for 4 weeks after the dosing is completed. Changes from baseline will be determined.
Measured: 6 months
Secondary outcomes (2)
To measure the number of adverse events which occur with HQK-1001 treatment when given over 26 weeks in beta thalassemia intermedia.
Adverse events which occur during HQK-1001 administration for 26 weeks will be recorded every 4 weeks.
Measured: 6 months
To measure changes from baseline in HbF during treatment with HQK-1001 for 26 weeks in beta thalassemia intermedia.
Levels of HbF will be averaged from a screening visit and day 1 of the study, prior to any drug treatment. HbF levels will then be measured every 4 weeks during treatment and for 4 weeks after the treatment, and compared to each subject's baseline value. The number of subjects in which an increase in HbF develops above individuals' average baseline value will be obtained.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2012 (estimated)
- Study startMay 2012
- Primary completionNov 2012
- Study completionJan 2013
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
- HemaQuest Pharmaceuticals Inc.
- Responsible party
- Sponsor
- Investigators
- Susan P Perrine, MD, Study director, Boston UniversityAdlette Inati, MD, Principal investigator, Chronic Care Center and Rafik Hariri University Hospital, Beirut, Lebanon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01642758
- Sponsor's protocol ID
- HQK-P2-THAL
- Acronym
- LB-04-THAL
- Status last verified
- Mar 2013
- First submitted
- 12 Jul 2012
- Last updated
- 14 Mar 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Perrine 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 21422239Perrine 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 20712788
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Phase 2 Study of HQK-1001 in Subjects With Beta Thalassemia Intermedia
- ClinicalTrials.gov ID
- NCT01642758
- Acronym / sponsor ID
- LB-04-THAL / HQK-P2-THAL
- Conditions
- Beta Thalassemia Intermedia
- Keywords
- Thalassemia intermedia, Fetal hemoglobin, Hemoglobin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Boston UniversityUniversity, hospital or other
- Collaborators
- HemaQuest Pharmaceuticals Inc.
- Study start
- May 2012
- Primary completion
- Nov 2012 (actual)
- Study completion
- Jan 2013 (actual)
- First posted
- 17 Jul 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 syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia.
This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.
Detailed description
This is a trial of an experimental oral medicine which stimulates production of fetal hemoglobin, an innate type of hemoglobin which is normally made but is suppressed in infancy. Fetal globin (HbF) can perform the function of the missing beta globin and reduce anemia in beta thalassemia, when it is produced in higher amounts than normal.
In this trial, 10 patients with beta thalassemia intermedia in Lebanon will all receive the study drug for 6 months at a dose which has been previously shown to be safe in normal volunteers and in beta thalassemia and sickle cell patients and to stimulate fetal globin production in many, when given for brief periods. The purpose of this trial is the following:
- To determine if total hemoglobin levels increase above baseline in some subjects when the study drug is taken for 26 weeks.
- To determine if fetal globin is increased above baseline levels in a proportion of subjects when the study drug is taken for 26 weeks.
- To determine the number of adverse events which occur with 26 weeks of administration of the study drug in beta thalassemia intermedia subjects.
After a screening period, the subjects will take the study drug at home once a day. They will be seen once every 4 weeks for examinations and laboratory tests during the dosing period and for 4 weeks afterwards.
This trial will provide an important step in evaluating a potential treatment for patients with beta thalassemia intermedia, that can be used around the world, if it is effective and safe.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sodium 2,2 dimethylbutyrate | Experimental | Drug: Sodium 2,2 dimethylbutyrateA single dose (20 mg/kg/day) of study drug will be taken once per day by mouth. |
Who may qualify
- Ages
- 16 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Diagnosis of beta thalassemia intermedia
- Ages 16-50 years
- Average total Hgb levels between 6.0 and 9.0 gm/dl within 30 days of initial dose of study drug
- Able to comply with all study procedures
- If female and of childbearing potential, must have a documented negative pregnancy test prior to entry and every 4 weeks
Key exclusion criteria (9 listed)
- Red blood cell transfusions within 3 months prior to administration of study drug
- QT Segment corrected (QTc)> 450 msec
- Use of Erythropoiesis Stimulating Agents(ESAs)within 9 days of first dose
- Hydroxyurea treatment within 6 months of first study drug
- History of significant arrythmias, syncope, or resuscitation
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: ST20Oral capsules, dose 20 mg/kg/day, once per day for 26 weeks | Drug | Sodium 2,2 dimethylbutyrate |
Primary outcome
To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia.
Time frame: 6 months
Baseline hemoglobin levels will be determined in each subject and averaged from levels obtained on a screening visit and on day one of the study, before any drug is taken. Hemoglobin levels will then be analyzed every 4 weeks during 26 weeks of taking the study drug and for 4 weeks after the dosing is completed. Changes from baseline will be determined.
2 secondary outcomes
To measure the number of adverse events which occur with HQK-1001 treatment when given over 26 weeks in beta thalassemia intermedia.
Time frame: 6 months
To measure changes from baseline in HbF during treatment with HQK-1001 for 26 weeks in beta thalassemia intermedia.
Time frame: 6 months
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 14 Mar 2013.
- 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.
- 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.
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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.
Susan P Perrine, MD study director
Boston University
Adlette Inati, MD principal investigator
Chronic Care Center and Rafik Hariri University Hospital, Beirut, Lebanon
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 Lebanon.
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 5 inclusion and 9 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 14 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 Lebanon. 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?
To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia. (time frame: 6 months). 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 NCT01642758, 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 14 Mar 2013.