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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)
Both
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
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

Type of Study:

Interventional

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:

No

Principal Investigator

Richard Ghalie, MD, MBA

Investigator Role:

Study Director

Investigator Affiliation:

HemaQuest Pharmaceuticals Inc.

Authority:

United States: Food and Drug Administration

Study ID:

HQP 1001-SCD-007

NCT ID:

NCT01601340

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

Name

Location

Howard University HospitalWashington, District of Columbia  20060
New York Methodist HospitalBrooklyn, New York  11215-3609
University of Illinois at ChicagoChicago, Illinois  60612
Children's Hospital and Research Center - OaklandOakland, California  94609-1809
Tufts Medical CenterBoston, Massachusetts  02111
LSU Health Sciences CenterShreveport, Louisiana  71130-3932
University of North Carolina at Chapel HillChapel Hill, North Carolina  27599
University of South AlabamaMobile, Alabama  36693
Georgia Health Sciences UniversityAugusta, Georgia  30912
Phoenix Children's Hospital - Ctr for Cancer and Blood DisordersPhoenix, Arizona  85016
Children's National HospitalWashington, District of Columbia  20010
The Children's Hospital at Montefiore Medical CenterBronx, New York  10467
Virginia Commonwealth Univeristy - Center on Health DisparitiesRichmond, Virginia  23298