Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00532883
Recruitment Status : Terminated (Enrollment has been terminated due to a slow rate of enrollment.)
First Posted : September 21, 2007
Results First Posted : May 11, 2010
Last Update Posted : January 18, 2013
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Hemoglobin SC Disease
Interventions Drug: Hydroxyurea
Drug: Magnesium Pidolate
Other: Placebo Pills and Placebo Liquid
Enrollment 44
Recruitment Details Subjects were recruited from October 2006 through June 2008 at 10 sites across the United States. Subjects were recruited from sickle cell specific clinics.
Pre-assignment Details Subjects were screened to ensure specific laboratory measurement levels after enrollment but prior to randomization.
Arm/Group Title Hydroxyurea/Magnesium Hydroxyurea/Mg Placebo HU Placebo/Magnesium HU Placebo/Mg Placebo
Hide Arm/Group Description Hydroxyurea (20 mg/kg/day) combined with liquid magnesium pidolate (0.6 mEq/kg/day). Hydroxyurea (20 mg/kg/day) combined with liquid Mg placebo. HU placebo combined with liquid magnesium pidolate (0.6 mEq/kg/day). Placebo for both hydroxyurea (20 mg/kg/day) and liquid magnesium pidolate (0.6 mEq/kg/day).
Period Title: Overall Study
Started 11 12 10 11
Completed 5 [1] 5 [1] 6 [1] 6 [1]
Not Completed 6 7 4 5
Reason Not Completed
Adverse Event             1             0             0             1
Lost to Follow-up             1             1             1             0
Withdrawal by Subject             0             1             1             0
Subject was Nonadherent             0             1             1             0
Early Study Termination             4             4             1             4
[1]
Study terminated early.
Arm/Group Title Hydroxyurea/Magnesium Hydroxyurea/Mg Placebo HU Placebo/Magnesium HU Placebo/Mg Placebo Total
Hide Arm/Group Description Hydroxyurea (20 mg/kg/day) combined with liquid magnesium pidolate (0.6 mEq/kg/day). Hydroxyurea (20 mg/kg/day) combined with liquid Mg placebo. HU placebo combined with liquid magnesium pidolate (0.6 mEq/kg/day). Placebo for both hydroxyurea (20 mg/kg/day) and liquid magnesium pidolate (0.6 mEq/kg/day). Total of all reporting groups
Overall Number of Baseline Participants 11 12 10 11 44
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 12 participants 10 participants 11 participants 44 participants
<=18 years
10
  90.9%
9
  75.0%
9
  90.0%
9
  81.8%
37
  84.1%
Between 18 and 65 years
1
   9.1%
3
  25.0%
1
  10.0%
2
  18.2%
7
  15.9%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 12 participants 10 participants 11 participants 44 participants
12.5  (4.41) 16.8  (12.6) 14.8  (8.45) 17.3  (12.41) 16.7  (9.81)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 12 participants 10 participants 11 participants 44 participants
Female
4
  36.4%
8
  66.7%
3
  30.0%
4
  36.4%
19
  43.2%
Male
7
  63.6%
4
  33.3%
7
  70.0%
7
  63.6%
25
  56.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 11 participants 12 participants 10 participants 11 participants 44 participants
11 12 10 11 44
1.Primary Outcome
Title Distribution of the Density of Hemoglobin SC Red Cells
Hide Description An individuals' percentage of red blood cells with density greater than 41 g/dL as measured by Advia.
Time Frame measured 2 months after initiation of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
ITT: All randomized subjects who receive any clinical trial material. Subjects in the ITT population will be classified according to the treatment group to which they were randomized, regardless of what study drug they received.
Arm/Group Title Hydroxyurea/Magnesium Hydroxyurea/Mg Placebo HU Placebo/Magnesium HU Placebo/Mg Placebo
Hide Arm/Group Description:
Hydroxyurea (20 mg/kg/day) combined with liquid magnesium pidolate (0.6 mEq/kg/day).
Hydroxyurea (20 mg/kg/day) combined with liquid Mg placebo.
HU placebo combined with liquid magnesium pidolate (0.6 mEq/kg/day).
Placebo for both hydroxyurea (20 mg/kg/day) and liquid magnesium pidolate (0.6 mEq/kg/day).
Overall Number of Participants Analyzed 11 12 10 11
Mean (Standard Deviation)
Unit of Measure: percent of cells
12.09  (5.075) 11.16  (7.296) 10.49  (4.572) 12.79  (3.739)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Hydroxyurea/Magnesium, Hydroxyurea/Mg Placebo, HU Placebo/Magnesium, HU Placebo/Mg Placebo
Comments F-test from a longitudinal mixed model (controlling for baseline measurement)testing the hypothesis of no difference in mean percent dense cells between the four treatment groups at Visit 6. The study was originally powered to detect a difference of 20%, but it was stopped early.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.93
Comments This is a global test comparing all four treatment arms.
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hydroxyurea/Magnesium Pidolate Hydroxyurea/Placebo Placebo/Magnesium Pidolate Placebo/Placebo
Hide Arm/Group Description 20 mg/kg/day 0.6 mEq/kg/day 20 mg/kg/day HU + 0.6 mEq/kg/day Mg [Not Specified]
All-Cause Mortality
Hydroxyurea/Magnesium Pidolate Hydroxyurea/Placebo Placebo/Magnesium Pidolate Placebo/Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Hide Serious Adverse Events
Hydroxyurea/Magnesium Pidolate Hydroxyurea/Placebo Placebo/Magnesium Pidolate Placebo/Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/11 (27.27%)      2/12 (16.67%)      3/10 (30.00%)      2/11 (18.18%)    
Blood and lymphatic system disorders         
Acute chest syndrome  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Anaemia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Congenital, familial and genetic disorders         
Sickle cell anaemia with crisis  1  3/11 (27.27%)  5 1/12 (8.33%)  3 3/10 (30.00%)  4 2/11 (18.18%)  3
General disorders         
Pyrexia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Infections and infestations         
Cellulitis  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Pharyngitis streptococcal  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Pneumonia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Surgical and medical procedures         
Skin graft  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 10.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Hydroxyurea/Magnesium Pidolate Hydroxyurea/Placebo Placebo/Magnesium Pidolate Placebo/Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/11 (81.82%)      10/12 (83.33%)      9/10 (90.00%)      10/11 (90.91%)    
Blood and lymphatic system disorders         
Anaemia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Neutropenia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 1/11 (9.09%)  1
Splenomegaly  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Congenital, familial and genetic disorders         
Sickle cell anaemia with crisis  1  6/11 (54.55%)  39 6/12 (50.00%)  18 4/10 (40.00%)  7 8/11 (72.73%)  32
Ear and labyrinth disorders         
Ear pain  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 1/11 (9.09%)  1
Eye disorders         
Conjunctivitis  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Eye pruritus  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Ocular hyperaemia  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Ocular icterus  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Gastrointestinal disorders         
Abdominal pain  1  2/11 (18.18%)  5 1/12 (8.33%)  2 0/10 (0.00%)  0 2/11 (18.18%)  3
Constipation  1  0/11 (0.00%)  0 1/12 (8.33%)  1 2/10 (20.00%)  2 1/11 (9.09%)  1
Diarrhoea  1  2/11 (18.18%)  2 2/12 (16.67%)  2 1/10 (10.00%)  2 2/11 (18.18%)  3
Dyspepsia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Mouth ulceration  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Nausea  1  1/11 (9.09%)  1 2/12 (16.67%)  2 0/10 (0.00%)  0 0/11 (0.00%)  0
Oral soft tissue disorder  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Vomiting  1  1/11 (9.09%)  3 1/12 (8.33%)  2 3/10 (30.00%)  4 3/11 (27.27%)  4
General disorders         
Influenza like illness  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Infusion site erythema  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Injection site pain  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Pain  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Pyrexia  1  2/11 (18.18%)  3 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Immune system disorders         
Hypersensitivity  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Multiple allergies  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Seasonal allergy  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Infections and infestations         
Abscess  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Cellulitis  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Furuncle  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Infection  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Myringitis bullous  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Nasopharyngitis  1  1/11 (9.09%)  1 1/12 (8.33%)  1 1/10 (10.00%)  1 2/11 (18.18%)  2
Osteomyelitis  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Otitis media  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Sinusitis  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Upper respiratory tract infection  1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/10 (10.00%)  2 2/11 (18.18%)  4
Urinary tract infection  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Viral infection  1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/10 (10.00%)  1 0/11 (0.00%)  0
Injury, poisoning and procedural complications         
Arthropod bite  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Head injury  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Joint injury  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Limb injury  1  2/11 (18.18%)  2 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Post procedural swelling  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Scratch  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Skin laceration  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 2/11 (18.18%)  2
Investigations         
Arterial bruit  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Spleen palpable  1  0/11 (0.00%)  0 2/12 (16.67%)  3 0/10 (0.00%)  0 0/11 (0.00%)  0
Metabolism and nutrition disorders         
Decreased appetite  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Dehydration  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 1/11 (9.09%)  1
Back pain  1  2/11 (18.18%)  2 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Joint swelling  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Muscle spasms  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Musculoskeletal chest pain  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Osteonecrosis  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Pain in jaw  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Nervous system disorders         
Dysgeusia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Headache  1  3/11 (27.27%)  10 5/12 (41.67%)  6 1/10 (10.00%)  1 3/11 (27.27%)  4
Migraine  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Psychiatric disorders         
Anxiety  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Depression  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 0/11 (0.00%)  0
Reproductive system and breast disorders         
Breast swelling  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Dysmenorrhoea  1  0/11 (0.00%)  0 2/12 (16.67%)  3 0/10 (0.00%)  0 1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders         
Asthma  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Cough  1  1/11 (9.09%)  1 0/12 (0.00%)  0 1/10 (10.00%)  1 2/11 (18.18%)  5
Dyspnoea exertional  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Epistaxis  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Nasal congestion  1  0/11 (0.00%)  0 0/12 (0.00%)  0 2/10 (20.00%)  2 1/11 (9.09%)  1
Pharyngolaryngeal pain  1  1/11 (9.09%)  1 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Rhinitis allergic  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Rhinorrhoea  1  1/11 (9.09%)  1 0/12 (0.00%)  0 1/10 (10.00%)  1 1/11 (9.09%)  1
Sneezing  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Skin and subcutaneous tissue disorders         
Dermatitis contact  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Nail disorder  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Pigmentation disorder  1  0/11 (0.00%)  0 0/12 (0.00%)  0 0/10 (0.00%)  0 1/11 (9.09%)  1
Rash  1  2/11 (18.18%)  2 0/12 (0.00%)  0 2/10 (20.00%)  2 0/11 (0.00%)  0
Rash macular  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Rash papular  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Skin hyperpigmentation  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/10 (0.00%)  0 0/11 (0.00%)  0
Skin lesion  1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/10 (10.00%)  1 1/11 (9.09%)  1
Swelling face  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/10 (10.00%)  1 0/11 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 10.0
This study was stopped early due to slow enrollment. It should therefore be viewed as a pilot study.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Harvey Luksenburg
Organization: NHLBI
Phone: 301-435-0050
EMail: luksenburgh@mail.nih.gov
Layout table for additonal information
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00532883    
Other Study ID Numbers: CHAMPS-St. Jude
U54HL070587 ( U.S. NIH Grant/Contract )
First Submitted: September 20, 2007
First Posted: September 21, 2007
Results First Submitted: April 1, 2010
Results First Posted: May 11, 2010
Last Update Posted: January 18, 2013