Effects of HQK-1001 in Patients With Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01601340
• Other study ID #: HQP 1001-SCD-007
• Status: Terminated
• Phase: Phase 2
• First received: May 12, 2012
• Last updated: March 17, 2015
• Start date: July 2012
• Est. completion date: December 2013

Study information

• Verified date: March 2015
• Source: HemaQuest Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review BoardJamaica: Ministry of HealthEgypt: Ministry of Health and PopulationEgypt: Institutional Review BoardLebanon: Institutional Review BoardCanada: Health CanadaCanada: Ethics Review Committee
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 77
• Est. completion date: December 2013
• Est. primary completion date: November 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 60 Years

Eligibility

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

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Disease
• Hemoglobin S Disease
• Sickle Cell Anemia
• Sickle Cell Disease
• Sickle Cell Disorders
• Sickling Disorder Due to Hemoglobin S

Intervention

Drug:
• HQK-1001
HQK-1001 tablets, twice daily for 48 weeks
• Placebo
Placebo tablets, twice daily for 48 weeks

Locations

Country	Name	City	State
Canada	University Health Network Toronto General Hospital	Toronto	Ontario
Egypt	Abu El Reesh Pediatric University Hospital	Cairo
Egypt	Ain Sham University Hospital	Cairo
Jamaica	University of the West Indies	Mona, Kingston 7
Lebanon	American University of Beirut Medical Center	Beirut
Lebanon	Chronic Care Center	Beirut
Lebanon	Rafik Hariri University Hospital	Beirut
United States	Georgia Health Sciences University	Augusta	Georgia
United States	Tufts Medical Center	Boston	Massachusetts
United States	The Children's Hospital at Montefiore Medical Center	Bronx	New York
United States	New York Methodist Hospital	Brooklyn	New York
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	University of Illinois at Chicago	Chicago	Illinois
United States	University of South Alabama	Mobile	Alabama
United States	Children's Hospital and Research Center - Oakland	Oakland	California
United States	Virginia Commonwealth Univeristy - Center on Health Disparities	Richmond	Virginia
United States	Children's National Hospital	Washington	District of Columbia
United States	Howard University Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
HemaQuest Pharmaceuticals Inc.

Countries where clinical trial is conducted

United States,  Canada,  Egypt,  Jamaica,  Lebanon, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in % fetal hemoglobin		Day 1 through Week 48	No
Secondary	Incidence and number of SCD pain crises and SCD-related complications		Day 1 through Week 52	No
Secondary	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	No
Secondary	Change in FACIT Fatigue Scale results		Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48	No
Secondary	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	Yes
Secondary	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.	1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48	No