NCT00143572

Brief Summary

The purpose of this study is to estimate the MTD of Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Nov 2004

Typical duration for phase_1

Geographic Reach
1 country

1 active site

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

November 1, 2004

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

September 1, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 2, 2005

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2008

Completed
Last Updated

April 26, 2017

Status Verified

February 1, 2010

Enrollment Period

3.2 years

First QC Date

September 1, 2005

Last Update Submit

April 24, 2017

Conditions

Anemia, Sickle Cell

Keywords

Hematologic DiseasesAnemia, Sickle Cell

Outcome Measures

Primary Outcomes (1)

  • To estimate the maximum tolerated dose of magnesium pidolate in combination with hydroxyurea in patients with sickle cell disease who have been on a therapeutic dose of hydroxyurea for at least six months.

    Every 2 weeks for first 8 weeks; then every 4 weeks

Secondary Outcomes (2)

  • To document the toxicity of the combination of hydroxyurea and magnesium pidolate.

    Every 2 weeks for first 8 weeks; then every 4 weeks

  • To investigate the effect of the combination of hydroxyurea and magnesium on hematological parameters and red cell metabolism.

    3 months, 6 months, and 9 months

Study Arms (1)

1

OTHER
Drug: Magnesium Pidolate, Hydroxyurea

Interventions

Magnesium Pidolate, HydroxyureaDRUG

Intervention Description: Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months. Mg pidolate will be given at an initial dose of 0.6 mEq/kg/day divided into 2 daily doses for the first cohort of patients. This dose will be escalated for the subsequent patient cohorts as defined by a classic Phase I design (according to toxicity).

1

Eligibility Criteria

Age3 Years - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Age \> 3 years and \< 15 years at the time of study enrollment
  • Diagnosis of Hb SS or Hb S beta thalassemia
  • Hydroxyurea treatment for at least 6 months prior to study entry at dose of 15 - 30 mg/kg/day
  • Compliance with taking HU treatment of at least 70 % for 6 months prior to study entry

You may not qualify if:

  • Red blood cell transfusion within the last 3 months resulting in a level of Hb A of 10% or more
  • Pregnancy or unwillingness to use effective birth control in sexually active subjects (females who state that they are sexually active)
  • Renal dysfunction defined by a serum creatinine greater than 1.5 times the upper limit of normal for age
  • Liver dysfunction defined by an ALT greater than twice the upper limit of normal for age
  • Concomitant usage of an "antisickling" agent other than hydroxyurea
  • Current use of Mg containing drugs
  • Iron deficiency, defined by serum ferritin ≤ 10 ng/ml
  • Concomitant chronic illness other than sickle cell anemia

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

MeSH Terms

Conditions

Anemia, Sickle CellHematologic Diseases

Interventions

Pyrrolidonecarboxylic AcidHydroxyurea

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

PyrrolidinonesPyrrolidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsGlutamatesAmino Acids, AcidicAmino AcidsAmino Acids, Peptides, and ProteinsImino AcidsAmino Acids, CyclicUreaAmidesOrganic Chemicals

Study Officials

  • Winfred Wang, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 1, 2005

First Posted

September 2, 2005

Study Start

November 1, 2004

Primary Completion

January 1, 2008

Study Completion

January 1, 2008

Last Updated

April 26, 2017

Record last verified: 2010-02

Locations

US(1)