NCT00661726

Brief Summary

Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels. This study will evaluate the safety and effectiveness of decitabine at increasing hemoglobin levels in people with TI.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
6

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2008

Geographic Reach
2 countries

3 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

Study Start

First participant enrolled

January 1, 2008

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

April 16, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 18, 2008

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2010

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2010

Completed
3.6 years until next milestone

Results Posted

Study results publicly available

April 8, 2014

Completed
Last Updated

April 25, 2014

Status Verified

February 1, 2014

Enrollment Period

2.3 years

First QC Date

April 16, 2008

Results QC Date

May 1, 2012

Last Update Submit

April 9, 2014

Conditions

Thalassemia

Keywords

Thalassemia Intermedia

Outcome Measures

Primary Outcomes (2)

  • Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL

    up to 12 weeks

  • Change in Total Hemoglobin (Hb) From Baseline to Peak (the Follow-up Time Point With the Highest Value)

    up to 12 weeks

Secondary Outcomes (10)

  • Change in Absolute Fetal Hemoglobin (HbF) From Baseline to Peak (the Follow-up Time Point With the Highest Value)

    up to 12 weeks

  • Change in Indirect Bilirubin From Baseline to Nadir (the Follow-up Time Point With the Lowest Value)

    up to 12 weeks

  • Change in Serum Lactate Dehydrogenase (LDH) From Baseline to Nadir (the Follow-up Time Point With the Lowest Value)

    up to 12 weeks

  • Change in Absolute Reticulocyte Count From Baseline to Nadir (the Follow-up Time Point With the Lowest Value)

    up to 12 weeks

  • Change in Erythropoietin Levels From Baseline to Nadir (the Follow-up Time Point With the Lowest Value)

    up to 12 weeks

  • +5 more secondary outcomes

Study Arms (1)

1

EXPERIMENTAL

Participants will receive injected decitabine for 12 weeks.

Drug: Decitabine (USAN, INN)

Interventions

Decitabine (USAN, INN)DRUG

Participants will receive 0.2 mg/kg of decitabine subcutaneously twice a week for 12 weeks. The dose will be reduced for toxicities as needed. The maximum dose of decitabine to be given will be 0.2 mg/kg.

Also known as: 5-aza-2'-deoxycytidine (NCS 127716), DAC, 5-Aza-CdR, Deoxyazacytidine, 1-(2'deoxy-D-ribofuranosy1)-5-azacytosine, Dezocitidine, Dacogen
1

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Beta-thalassemia and beta thalassemia-hemoglobin E (HbE), as confirmed by DNA testing
  • Transfusion independent for at least 120 days before study entry
  • Red blood cell folate levels above the lower limit of normal

You may not qualify if:

  • Absolute neutrophil count (ANC) less than 2000/mm3 in the 8 weeks before study entry or a history of chronic neutropenia, defined as an ANC less than 2000/mm3
  • Platelet count less than 100,000/mm3 or greater than 1,000,000/mm3 in the 8 weeks before study entry
  • Family history of an inherited disease resulting in low ANC or bone marrow failure
  • Serum creatinine level greater than 2 mg/dL in the 8 weeks before study entry
  • Evidence of liver disease, as defined by one or more of the following conditions:
  • Alanine aminotransferase (ALT) level greater than 3 times the upper limit of normal in the 8 weeks before study entry
  • Serum albumin level less than 3 g/dL in the 8 weeks before study entry
  • Evidence of cirrhosis on liver biopsy obtained in the 6 months before study entry
  • Approaching death; has concurrent liver, kidney, cardiac, or metabolic disease; or has any disease of such severity that death within 7 to 10 days of study entry is likely
  • Pregnant, planning to become pregnant, or breastfeeding
  • Sexually active female of childbearing potential who is unwilling to use at least two acceptable methods of contraception, as determined by the investigator
  • Sexually active male whose partner is of child-bearing potential and who is unwilling to use at least two acceptable methods of contraception, as determined by the investigator, during and for 2 months after decitabine treatment
  • Diagnosed with cancer (except non-melanoma skin cancer) in the 5 years before study entry. In particular, suspicion or evidence of myelodysplastic syndrome (MDS) on clinically indicated bone marrow aspirate or a family history of MDS or concurrent leukemia
  • HIV infection
  • Not expected to be able to complete 24 weeks of study follow-up
  • +4 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Children's Hospital and Research Center at Oakland

Oakland, California, 94609, United States

Location

Children's Hospital Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University Health Network

Toronto, M5G 2C4, Canada

Location

Related Publications (1)

  • Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL, Vichinsky EP; Thalassemia Clinical Research Network. A pilot study of subcutaneous decitabine in beta-thalassemia intermedia. Blood. 2011 Sep 8;118(10):2708-11. doi: 10.1182/blood-2011-03-341909. Epub 2011 Jun 23.

    PMID: 21700776RESULT

MeSH Terms

Conditions

Thalassemiabeta-Thalassemia

Interventions

Decitabine

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

AzacitidineAza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Results Point of Contact

Title
Nancy Gee, TCRN Network Manager
Organization
NERI
ngee@neriscience.com
617-972-3295

Study Officials

  • Nancy Olivieri, MD

    University Health Network/Toronto General Hospital

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 16, 2008

First Posted

April 18, 2008

Study Start

January 1, 2008

Primary Completion

May 1, 2010

Study Completion

September 1, 2010

Last Updated

April 25, 2014

Results First Posted

April 8, 2014

Record last verified: 2014-02

Locations

US(2)CA(1)