A Study of HQK-1001 in Patients With Sickle Cell Disease

NCT ID: NCT01322269

Last Updated: 2013-06-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 52 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-04-30

Brief 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.

Conditions

Sickle Cell Disease; Sickle Cell Anemia; Sickle Cell Disorders; Hemoglobin S Disease; Sickling Disorder Due to Hemoglobin S

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HQK-1001 (30 mg/kg)

Group Type: EXPERIMENTAL

HQK-1001

Intervention Type: DRUG

HQK-1001 tablets, once daily for daily 26 weeks

HQK-1001 (40 mg/kg)

Group Type: EXPERIMENTAL

HQK-1001

Intervention Type: DRUG

HQK-1001 tablets, once daily for daily 26 weeks

HQK-1001 (50 mg/kg)

Group Type: EXPERIMENTAL

HQK-1001

Intervention Type: DRUG

HQK-1001 tablets, once daily for daily 26 weeks

Interventions

HQK-1001

HQK-1001 tablets, once daily for daily 26 weeks

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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
• 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

Exclusion Criteria

• 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)
• 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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

HemaQuest Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Richard Ghalie, MD, MBA

Role: STUDY_DIRECTOR

HemaQuest Pharmaceuticals Inc.

Locations

Children's Hospital and Research Center - Oakland

Oakland, California, United States

University of Miami Miller School of Medicine - Dept of Pediatrics

Miami, Florida, United States

Georgia Health Sciences University - Adult SIckle Cell Center

Augusta, Georgia, United States

University of Illinois at Chicago - Dept of Pediatrics

Chicago, Illinois, United States

LSU Health Sciences Center - Feist Weiller Cancer Center

Shreveport, Louisiana, United States

Tufts Medical Center

Boston, Massachusetts, United States

University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program

Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology

Dallas, Texas, United States

The Hospital for Sick Children

Toronto, Ontario, Canada

University Health Network Toronto General Hospital

Toronto, Ontario, Canada

Abu El Reesh Pediatric University Hospital

Cairo, , Egypt

University of the West Indies - Sickle Cell Unit

Mona, Kingston, Jamaica

American University of Beirut Medical Center

Beirut, , Lebanon

Rafik Hariri University Hospital

Beirut, , Lebanon

Chronic Care Center

Hazmiyeh, , Lebanon

Countries

United States; Canada; Egypt; Jamaica; Lebanon

References

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.

Reference Type: DERIVED

PMID: 23828223 (View on PubMed)

Other Identifiers

HQP 1001-SCD-006

Identifier Type: -

Identifier Source: org_study_id