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

Phase 2
12 Years
60 Years
Open (Enrolling)
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S

Thank you

Trial Information

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

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

- 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

Type of Study:


Study Design:

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

Outcome Measure:

Change from baseline in % fetal hemoglobin

Outcome Time Frame:

Day 1 through Week 48

Safety Issue:


Principal Investigator

Richard Ghalie, MD, MBA

Investigator Role:

Study Director

Investigator Affiliation:

HemaQuest Pharmaceuticals Inc.


United States: Food and Drug Administration

Study ID:

HQP 1001-SCD-007



Start Date:

July 2012

Completion Date:

Related Keywords:

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



Howard University Hospital Washington, District of Columbia  20060
New York Methodist Hospital Brooklyn, New York  11215-3609
University of Illinois at Chicago Chicago, Illinois  60612
Children's Hospital and Research Center - Oakland Oakland, California  94609-1809
Tufts Medical Center Boston, Massachusetts  02111
LSU Health Sciences Center Shreveport, Louisiana  71130-3932
University of North Carolina at Chapel Hill Chapel Hill, North Carolina  27599
University of South Alabama Mobile, Alabama  36693
Georgia Health Sciences University Augusta, Georgia  30912
Phoenix Children's Hospital - Ctr for Cancer and Blood Disorders Phoenix, Arizona  85016
Children's National Hospital Washington, District of Columbia  20010
The Children's Hospital at Montefiore Medical Center Bronx, New York  10467
Virginia Commonwealth Univeristy - Center on Health Disparities Richmond, Virginia  23298