Sickle Cell DiseasePhase 2NCT01322269
A Study of HQK-1001 in Patients With Sickle Cell Disease
A Phase 2 study testing HQK-1001 in people with Sickle Cell Disease and Sickle Cell Anemia, run by HemaQuest Pharmaceuticals Inc. at 16 sites in 5 countries.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease. Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders
- What is being tested?
- HQK-1001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 16 sitesin 5 countries
- Study dates
- Apr 2011to Mar 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 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Established diagnosis of SCD
- Males and females between 12 and 60 years of age, inclusive
- At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening
- If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening
- If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose
- If transfusion in the 4 months prior to screening, then HbA level < 20% at screening
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- Average of the initial two HbF levels ≥ 2.0 % within ≤ 7 days prior to the initial dose of HQK-1001. The two must be obtained ≥ 24 hours apart
- Ability to swallow tablets
- Able and willing to give informed consent and assent (if applicable)
- If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 7 days of first dose of HQK-1001
- If a subject is a WCBP, she must agree to use an effective form of contraception within 7 days of the initial dose of HQK-1001 and for one month after HQK-1001 discontinuation
- Sexually active male subjects (with WCBP partners) must agree to use latex condoms or ensure that their partner(s) use an effective form of contraception
- In the view of the Investigator, subject is able and willing to comply with necessary study procedures
Who cannot join
- More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening
- Pulmonary hypertension requiring oxygen therapy
- QTc > 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)
- Assigned to a regular transfusion program
- Use of erythropoiesis stimulating agents within 90 days of screening
- ALT > 3x upper limit of normal (ULN)
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- Serum creatinine > 1.2 mg/dL
- A serious, concurrent illness that would limit ability to complete or comply with the study requirements
- An acute vaso-occlusive event within 3 weeks prior to screening
- Creatine phosphokinase (CK) > 20% above the ULN
- An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
- History of syncope, clinically significant dysrhythmias or resuscitation from sudden death
- Chronic opiate use, which, in the view of the Investigator, could confound evaluation of an investigational drug
- Current abuse of alcohol or drugs
- Received another investigational agent within 4 weeks or 5 half-lives, whichever is longer, prior to screening
- Currently pregnant or breast feeding a child
- Known infection with HIV-1
- Infection with hepatitis B or hepatitis C, such that patients are currently on therapy or will be placed on therapy during the trial
Trial locations and contacts
16 sites in 5 countries.
16 sites in 5 countries
United States9 sites
Georgia Health Sciences University - Adult SIckle Cell Center
Augusta, Georgia, 30912
Tufts Medical Center
Boston, Massachusetts, 02111
University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, 27599
University of Illinois at Chicago - Dept of Pediatrics
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology
Dallas, Texas, 75390-9063
University of Miami Miller School of Medicine - Dept of Pediatrics
Miami, Florida, 33101
Children's Hospital and Research Center - Oakland
Oakland, California, 94609
LSU Health Sciences Center - Feist Weiller Cancer Center
Shreveport, Louisiana, 71103
Lebanon3 sites
American University of Beirut Medical Center
Beirut
Rafik Hariri University Hospital
Beirut
Chronic Care Center
Hazmiyeh
Canada2 sites
The Hospital for Sick Children
Toronto, Ontario, MSG 1X8
University Health Network Toronto General Hospital
Toronto, Ontario, MSG 2C4
About this study
Official title: A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
- Being tested
- HQK-1001
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HQK-1001Drug
HQK-1001 tablets, once daily for daily 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| HQK-1001 (30 mg/kg) | Experimental | Not described Receives: Drug: HQK-1001 |
| HQK-1001 (40 mg/kg) | Experimental | Not described Receives: Drug: HQK-1001 |
| HQK-1001 (50 mg/kg) | Experimental | Not described Receives: Drug: HQK-1001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.
Measured: Day 1 through Week 30
Secondary outcomes (2)
Fetal hemoglobin levels
Measured: Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30
Incidence of sickle cell crisis events
Measured: Day 1 through Week 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2011 (estimated)
- Study startApr 2011
- Primary completionMar 2012
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 Sickle Cell DiseaseStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Richard Ghalie, MD, MBA, Study director, HemaQuest Pharmaceuticals Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01322269
- Sponsor's protocol ID
- HQP 1001-SCD-006
- Status last verified
- Jun 2013
- First submitted
- 22 Mar 2011
- Last updated
- 19 Jun 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kutlar A, Reid ME, Inati A, Taher AT, Abboud MR, El-Beshlawy A, Buchanan GR, Smith H, Ataga KI, Perrine SP, Ghalie RG. A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2013 Nov;88(11):E255-60. doi: 10.1002/ajh.23533. Epub 2013 Oct 3.
PubMed 23828223
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01322269
- Acronym / sponsor ID
- HQP 1001-SCD-006
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2011
- Primary completion
- Mar 2012 (actual)
- First posted
- 24 Mar 2011 (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 three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| HQK-1001 (30 mg/kg) | Experimental | Drug: HQK-1001 |
| HQK-1001 (40 mg/kg) | Experimental | Drug: HQK-1001 |
| HQK-1001 (50 mg/kg) | Experimental | Drug: HQK-1001 |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Established diagnosis of SCD
- Males and females between 12 and 60 years of age, inclusive
- At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening
- If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening
- If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose
Key exclusion criteria (18 listed)
- More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening
- Pulmonary hypertension requiring oxygen therapy
- QTc > 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)
- Assigned to a regular transfusion program
- Use of erythropoiesis stimulating agents within 90 days of screening
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 tablets, once daily for daily 26 weeks | Drug | HQK-1001 (30 mg/kg), HQK-1001 (40 mg/kg), HQK-1001 (50 mg/kg) |
Primary outcome
Safety
Time frame: Day 1 through Week 30
Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.
2 secondary outcomes
Fetal hemoglobin levels
Time frame: Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30
Incidence of sickle cell crisis events
Time frame: Day 1 through Week 30
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 19 Jun 2013.
- Derived from this study: PubMed 23828223Kutlar A, Reid ME, Inati A, Taher AT, Abboud MR, El-Beshlawy A, Buchanan GR, Smith H, Ataga KI, Perrine SP, Ghalie RG. A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2013 Nov;88(11):E255-60. doi: 10.1002/ajh.23533. Epub 2013 Oct 3.
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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.
Richard Ghalie, MD, MBA study director
HemaQuest Pharmaceuticals Inc.
16 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HQK-1001 in people with Sickle Cell Disease and Sickle Cell Anemia, run by HemaQuest Pharmaceuticals Inc. at 16 sites in 5 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 13 inclusion and 18 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 19 Jun 2013. 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 United States, Lebanon, Canada, Egypt, Jamaica. 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?
Safety (time frame: Day 1 through Week 30). 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 NCT01322269, 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 19 Jun 2013.