Effects of HQK-1001 in Patients With Sickle Cell Disease

Phase 2

Terminated

• Conditions: Sickle Cell Disease; Sickle Cell Anemia; Sickle Cell Disorders; Hemoglobin S Disease; Sickling Disorder Due to Hemoglobin S
• Interventions: Drug: HQK-1001; Drug: Placebo

• Registration Number: NCT01601340
• Lead Sponsor: HemaQuest Pharmaceuticals Inc.

Brief Summary

The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-05-12
• Study First Submitted QC: 2012-05-17
• Study First Posted: 2012-05-18
• Study Start: 2012-07
• Primary Completion: 2013-11
• Study Completion: 2013-12
• Status Verified: 2015-03
• Last Update Submitted: 2015-03-17
• Last Update Posted: 2015-03-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 77

Inclusion Criteria

• 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
• 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.

Exclusion Criteria

• 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
• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
HQK-1001	HQK-1001	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Change from baseline in % fetal hemoglobin	Day 1 through Week 48

Secondary Outcome Measures

Name	Time	Method
Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments	Day 1 through Week 52
HQK-1001 pharmacokinetic parameters	1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48	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.
Incidence and number of SCD pain crises and SCD-related complications	Day 1 through Week 52
Subject reported daily pain scale scores and analgesic use	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	Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48

Trial Locations

Locations (18)

University Health Network Toronto General Hospital; 🇨🇦 Toronto, Ontario, Canada

Tufts Medical Center; 🇺🇸 Boston, Massachusetts, United States

Georgia Health Sciences University; 🇺🇸 Augusta, Georgia, United States

New York Methodist Hospital; 🇺🇸 Brooklyn, New York, United States

American University of Beirut Medical Center; 🇱🇧 Beirut, Lebanon

Abu El Reesh Pediatric University Hospital; 🇪🇬 Cairo, Egypt

Children's Hospital and Research Center - Oakland; 🇺🇸 Oakland, California, United States

University of South Alabama; 🇺🇸 Mobile, Alabama, United States

The Children's Hospital at Montefiore Medical Center; 🇺🇸 Bronx, New York, United States

Children's National Hospital; 🇺🇸 Washington, District of Columbia, United States

University of Illinois at Chicago; 🇺🇸 Chicago, Illinois, United States

Howard University Hospital; 🇺🇸 Washington, District of Columbia, United States

Ain Sham University Hospital; 🇪🇬 Cairo, Egypt

Chronic Care Center; 🇱🇧 Beirut, Lebanon

University of the West Indies; 🇯🇲 Mona, Kingston 7, Jamaica

Rafik Hariri University Hospital; 🇱🇧 Beirut, Lebanon

University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Virginia Commonwealth Univeristy - Center on Health Disparities; 🇺🇸 Richmond, Virginia, United States