NCT00790127

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Mar 2009

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 11, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 13, 2008

Completed
4 months until next milestone

Study Start

First participant enrolled

March 1, 2009

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2010

Completed
Last Updated

May 20, 2011

Status Verified

May 1, 2011

Enrollment Period

1.2 years

First QC Date

November 11, 2008

Last Update Submit

May 18, 2011

Conditions

Beta Thalassemia

Outcome Measures

Primary Outcomes (1)

  • Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam

    168 days

Secondary Outcomes (2)

  • Pharmacokinetics assessed by plasma drug concentration levels

    140 days

  • Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin

    168 days

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

HQK-1001

EXPERIMENTAL

HQK-1001

Drug: HQK-1001

Interventions

HQK-1001DRUG

HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days

HQK-1001
PlaceboDRUG

Matching placebo capsules administered once a day, orally, for 56 days

Placebo

Eligibility Criteria

Age12 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia
  • Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization.
  • Age ≥ 12 and ≤ 60 years
  • Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures
  • If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication

You may not qualify if:

  • Spleen palpable ≥ 2 cm below the left costal margin
  • Pulmonary hypertension requiring oxygen therapy
  • QTc \> 450 msec on screening ECG
  • Infection with hepatitis C, hepatitis B requiring therapy
  • Known infection with HIV
  • Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels
  • Fever greater than 38.5°C in the week prior to administration of study medication
  • ALT \> 4x upper limit of normal (ULN)
  • Baseline elevation of CPK value prior to randomization
  • Treatment with hydroxyurea within 2 months prior to administration of study medication
  • Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies)
  • Serum creatinine \> 1.5 mg/dl
  • Received investigational systemic therapy within 30 days prior to randomization
  • Currently pregnant or breast feeding a child
  • Subject history of clinically significant arrhythmias or syncope
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Chronic Care Center

Beirut, Lebanon

Location

Siriraj Hospital

Bangkok, Bangkoknoin District, 10700, Thailand

Location

Related Publications (1)

  • Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.

    PMID: 36637054DERIVED

MeSH Terms

Conditions

beta-Thalassemia

Interventions

2,2-dimethylbutyric acid

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Noppadol Siritanaratkul, MD

    Siriraj Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

November 11, 2008

First Posted

November 13, 2008

Study Start

March 1, 2009

Primary Completion

May 1, 2010

Study Completion

May 1, 2010

Last Updated

May 20, 2011

Record last verified: 2011-05

Locations

LE(1)TH(1)