Sickle Cell DiseasePhase 1/2NCT00842088
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
A Phase 1/2 study testing HQK-1001 in people with Sickle Cell Disease, run by HemaQuest Pharmaceuticals Inc. at 9 sites in 2 countries.
Trial summary
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) 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
- 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
- 24 participants
- Where
- 9 sitesin 2 countries
- Study dates
- Mar 2009to Jul 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
- Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
- Between 12 and 60 years of age, inclusive
- At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
- Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
- If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
- Able and willing to give informed consent
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- If female, must have a negative serum pregnancy test within 7 days of dosing
- If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
- If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
- In the view of the Investigator, able to comply with necessary study procedures
Who cannot join
- Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
- Participation in a regular blood transfusion program
- More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
- An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
- Pulmonary hypertension requiring oxygen
- QTc > 450 msec on screening
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- Alanine transaminase (ALT) > 3X upper limit of normal (ULN)
- Creatinine phosphokinase (CPK) > 20% above the ULN
- Serum creatinine >1.2 mg/dL
- An acute illness (e.g., febrile, gastrointestinal [GI], respiratory) within 72 hours prior to receiving first dose of study medication
- 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 administration of study medication
- 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
9 sites in 2 countries.
9 sites in 2 countries
United States8 sites
Medical College of Georgia
Augusta, Georgia, 30912
Johns Hopkins School of Medicine
Baltimore, Maryland, 21205
UNC Comprehensive Sickle Cell Program
Chapel Hill, North Carolina, 27599
University of Illinois at Chicago
Chicago, Illinois, 60612
Century Clinical Research, Inc.
Daytona Beach, Florida, 32117
Texas Children's Cancer Center and Hematology Service
Houston, Texas, 77030
Trialogic Research
Madison, Alabama, 35758
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
About this study
Official title: A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- Placebo, 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.
PlaceboDrug
Matching placebo capsule. Administered orally once a day on dosing days.
HQK-1001Drug
HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days.
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | Not described Receives: Drug: HQK-1001 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
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, and (4) physical exam.
Measured: 126 days
Secondary outcomes (2)
Pharmacokinetics assessed by plasma drug concentration levels.
Measured: Days 0, 6, 69 and 97 post first dose
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.
Measured: Every 2 weeks through Day 126 post first dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2009 (estimated)
- Study startMar 2009
- Primary completionJul 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 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Susan Perrine, M.D., Study director, HemaQuest Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00842088
- Sponsor's protocol ID
- HQP 2008-004
- Status last verified
- Jul 2011
- First submitted
- 11 Feb 2009
- Last updated
- 1 Aug 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP. A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.
PubMed 22887019
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 in Subjects With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00842088
- Acronym / sponsor ID
- HQP 2008-004
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2009
- Primary completion
- Jul 2010 (actual)
- First posted
- 12 Feb 2009 (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 assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: HQK-1001 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
- Between 12 and 60 years of age, inclusive
- At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
- Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
- If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
Key exclusion criteria (17 listed)
- Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
- Participation in a regular blood transfusion program
- More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
- An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
- Pulmonary hypertension requiring oxygen
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 |
|---|---|---|
| PlaceboMatching placebo capsule. Administered orally once a day on dosing days. | Drug | Placebo |
| HQK-1001HQK-1001 capsules. 10 mg/kg, 20 mg/kg or 30 mg/kg administered once a day on dosing days. | Drug | Active |
Primary outcome
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
Time frame: 126 days
2 secondary outcomes
Pharmacokinetics assessed by plasma drug concentration levels.
Time frame: Days 0, 6, 69 and 97 post first dose
Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.
Time frame: Every 2 weeks through Day 126 post first dose
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 1 Aug 2011.
- Derived from this study: PubMed 22887019Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP. A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2012 Nov;87(11):1017-21. doi: 10.1002/ajh.23306. Epub 2012 Aug 7.
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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 Perrine, M.D. study director
HemaQuest Pharmaceuticals
9 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 Sickle Cell Disease, run by HemaQuest Pharmaceuticals Inc. at 9 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 10 inclusion and 17 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 1 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, 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. 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, and (4) physical exam. (time frame: 126 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 NCT00842088, 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 1 Aug 2011.