Beta ThalassemiaPhase 1/2NCT00790127
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
A Phase 1/2 study testing HQK-1001 in people with Beta Thalassemia, run by HemaQuest Pharmaceuticals Inc. at 2 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Beta Thalassemia
- What is being tested?
- HQK-1001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Mar 2009to May 2010
- Phase
- Phase 1/2
- 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 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia
- Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization.
- Age ≥ 12 and ≤ 60 years
- Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures
- If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication
Who cannot join
- Spleen palpable ≥ 2 cm below the left costal margin
- Pulmonary hypertension requiring oxygen therapy
- QTc > 450 msec on screening ECG
- Infection with hepatitis C, hepatitis B requiring therapy
- Known infection with HIV
- Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels
Show all 16Show fewer
- Fever greater than 38.5°C in the week prior to administration of study medication
- ALT > 4x upper limit of normal (ULN)
- Baseline elevation of CPK value prior to randomization
- Treatment with hydroxyurea within 2 months prior to administration of study medication
- Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies)
- Serum creatinine > 1.5 mg/dl
- Received investigational systemic therapy within 30 days prior to randomization
- Currently pregnant or breast feeding a child
- Subject history of clinically significant arrhythmias or syncope
- Known current drug or alcohol abuse
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
- Conditions
- Beta Thalassemia
- Being tested
- HQK-1001, Placebo
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HQK-1001Drug
HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days
PlaceboDrug
Matching placebo capsules administered once a day, orally, for 56 days
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
| HQK-1001 | Experimental | HQK-1001 Receives: Drug: HQK-1001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
Measured: 168 days
Secondary outcomes (2)
Pharmacokinetics assessed by plasma drug concentration levels
Measured: 140 days
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin
Measured: 168 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2008 (estimated)
- Study startMar 2009
- Primary completionMay 2010
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Noppadol Siritanaratkul, MD, Principal investigator, Siriraj Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00790127
- Sponsor's protocol ID
- HQP-2008-003b
- Status last verified
- May 2011
- First submitted
- 11 Nov 2008
- Last updated
- 20 May 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.
PubMed 36637054
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia
- ClinicalTrials.gov ID
- NCT00790127
- Acronym / sponsor ID
- HQP-2008-003b
- Conditions
- Beta Thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2009
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 13 Nov 2008 (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.
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
| HQK-1001 | Experimental | Drug: HQK-1001HQK-1001 |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia
- Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization.
- Age ≥ 12 and ≤ 60 years
- Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures
- If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication
Key exclusion criteria (16 listed)
- Spleen palpable ≥ 2 cm below the left costal margin
- Pulmonary hypertension requiring oxygen therapy
- QTc > 450 msec on screening ECG
- Infection with hepatitis C, hepatitis B requiring therapy
- Known infection with HIV
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 |
|---|---|---|
| HQK-1001HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days | Drug | HQK-1001 |
| PlaceboMatching placebo capsules administered once a day, orally, for 56 days | Drug | Placebo |
Primary outcome
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
Time frame: 168 days
2 secondary outcomes
Pharmacokinetics assessed by plasma drug concentration levels
Time frame: 140 days
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin
Time frame: 168 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 20 May 2011.
- Derived from this study: PubMed 36637054Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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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.
Noppadol Siritanaratkul, MD principal investigator
Siriraj Hospital
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing HQK-1001 in people with Beta Thalassemia, run by HemaQuest Pharmaceuticals Inc. at 2 sites in 2 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 5 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 20 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Lebanon, 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam (time frame: 168 days). 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 NCT00790127, which also lists 1 linked publication.
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 20 May 2011.