Sickle Cell DiseasePhase 2NCT01601340
Effects 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 18 sites in 5 countries.
Trial summary
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F 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?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 18 sitesin 5 countries
- Study dates
- Jul 2012to Dec 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 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males and females between 12 and 60 years of age
- Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
- At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
- Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
- If subject has been transfused in the 3 months prior to screening, then Hb A level < 20% at screening
- Baseline Hb F level obtained within 14 days prior to randomization
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- Able to swallow tablets
- Able and willing to give informed consent and/or assent
- If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine pregnancy test prior to dosing on Day 1
- If a subject is a WCBP, she must agree to use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation
- Sexually active male subjects who have not had a vasectomy must agree to use latex condoms with WCBP partners or ensure that their partner(s) use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation.
Who cannot join
- Assigned to a regular transfusion program
- Use of erythropoiesis stimulating agents within 90 days prior to screening
- An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
- More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
- Pulmonary hypertension requiring therapy
- ALT or AST > 3x ULN
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- Serum creatinine > 1.5x ULN
- Serum amylase levels > 1.5x ULN
- Serum lipase level > 1.5x ULN
- A serious, concurrent illness that would limit ability to complete or comply with the study requirements
- 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 due to SCD-related complication
- Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
- History of pancreatitis
- Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
- Current abuse of alcohol or drugs
- Use of 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 subjects are currently on anti-viral therapy or will be placed on therapy
Trial locations and contacts
18 sites in 5 countries.
18 sites in 5 countries
United States11 sites
Georgia Health Sciences University
Augusta, Georgia, 30912
Tufts Medical Center
Boston, Massachusetts, 02111
New York Methodist Hospital
Brooklyn, New York, 11215
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Illinois at Chicago
Chicago, Illinois, 60612
University of South Alabama
Mobile, Alabama, 36617-2238
Children's Hospital and Research Center - Oakland
Oakland, California, 94609
Virginia Commonwealth Univeristy - Center on Health Disparities
Richmond, Virginia, 23298
The Children's Hospital at Montefiore Medical Center
The Bronx, New York, 10467
Children's National Hospital
Washington D.C., District of Columbia, 20010
Howard University Hospital
Washington D.C., District of Columbia, 20060
Lebanon3 sites
American University of Beirut Medical Center
Beirut
Chronic Care Center
Beirut
Rafik Hariri University Hospital
Beirut
Egypt2 sites
Abu El Reesh Pediatric University Hospital
Cairo
Ain Sham University Hospital
Cairo
Canada1 site
University Health Network Toronto General Hospital
Toronto, Ontario, M5G2C4
About this study
Official title: A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F 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, Placebo
- 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, twice daily for 48 weeks
PlaceboDrug
Placebo tablets, twice daily for 48 weeks
| Study group | Role | What they receive |
|---|---|---|
| HQK-1001 | Active comparator | 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.
Change from baseline in % fetal hemoglobin
Measured: Day 1 through Week 48
Secondary outcomes (5)
Incidence and number of SCD pain crises and SCD-related complications
Measured: Day 1 through Week 52
Subject reported daily pain scale scores and analgesic use
Measured: 7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
Change in FACIT Fatigue Scale results
Measured: Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments
Measured: Day 1 through Week 52
HQK-1001 pharmacokinetic parameters
A subset of subjects (7) will undergo sampling for detailed analysis of pharmacokinetic parameters (AUC, Cmax) with samples taken pre-dose, and 1, 2, 4, 8, and 10 hours after the morning dose at Week 4.
Measured: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2012 (estimated)
- Study startJul 2012
- Primary completionNov 2013
- Study completionDec 2013
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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 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
- NCT01601340
- Sponsor's protocol ID
- HQP 1001-SCD-007
- Status last verified
- Mar 2015
- First submitted
- 12 May 2012
- Last updated
- 18 Mar 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Reid ME, El Beshlawy A, Inati A, Kutlar A, Abboud MR, Haynes J Jr, Ward R, Sharon B, Taher AT, Smith W, Manwani D, Ghalie RG. A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2014 Jul;89(7):709-13. doi: 10.1002/ajh.23725. Epub 2014 Apr 15.
PubMed 24677033
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01601340
- Acronym / sponsor ID
- HQP 1001-SCD-007
- 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
- Terminated
- Enrolment
- 77 participants (actual)
- Lead sponsor
- HemaQuest Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2012
- Primary completion
- Nov 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 18 May 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.
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.
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 |
|---|---|---|
| HQK-1001 | Active comparator | 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 (11 listed)
- Males and females between 12 and 60 years of age
- Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
- At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
- Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
- If subject has been transfused in the 3 months prior to screening, then Hb A level < 20% at screening
Key exclusion criteria (20 listed)
- Assigned to a regular transfusion program
- Use of erythropoiesis stimulating agents within 90 days prior to screening
- An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
- More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
- Pulmonary hypertension requiring therapy
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, twice daily for 48 weeks | Drug | HQK-1001 |
| PlaceboPlacebo tablets, twice daily for 48 weeks | Drug | Placebo |
Primary outcome
Change from baseline in % fetal hemoglobin
Time frame: Day 1 through Week 48
5 secondary outcomes
Incidence and number of SCD pain crises and SCD-related complications
Time frame: Day 1 through Week 52
Subject reported daily pain scale scores and analgesic use
Time frame: 7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
Change in FACIT Fatigue Scale results
Time frame: Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments
Time frame: Day 1 through Week 52
HQK-1001 pharmacokinetic parameters
Time frame: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2015.
- Derived from this study: PubMed 24677033Reid ME, El Beshlawy A, Inati A, Kutlar A, Abboud MR, Haynes J Jr, Ward R, Sharon B, Taher AT, Smith W, Manwani D, Ghalie RG. A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2014 Jul;89(7):709-13. doi: 10.1002/ajh.23725. Epub 2014 Apr 15.
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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.
18 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 18 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 11 inclusion and 20 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: stopped early, as of the sponsor's last update on 18 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Lebanon, Egypt, Canada, 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 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 was stopped early. Results are not available in the source record.
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?
Change from baseline in % fetal hemoglobin (time frame: Day 1 through Week 48). 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 NCT01601340, 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 18 Mar 2015.