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A Safety and Efficacy Trial of Stannsoporfin in Neonates With Hyperbilirubinemia

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ClinicalTrials.gov Identifier: NCT00850993
Recruitment Status : Terminated (To redefine study population)
First Posted : February 25, 2009
Results First Posted : August 29, 2014
Last Update Posted : August 29, 2014
Sponsor:
Information provided by (Responsible Party):
InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Hyperbilirubinemia, Neonatal
Interventions Drug: stannsoporfin
Other: Saline
Enrollment 63
Recruitment Details 72 patients were to be enrolled to achieve 24 patients in each cohort. Each was to include 6 patients randomly assigned to a placebo group and 18 patients randomly assigned to a treatment group. The number of patients actually enrolled was 63 patients because the study was closed before enrollment of the full 4.5 mg/kg cohort.
Pre-assignment Details Term and late preterm patients (≥35 weeks and <43 weeks gestational age) up to 48 hours of age with hyperbilirubinemia and risk factors for hemolytic disease, including patients with Coombs positive ABO blood type incompatibility, Rhesus (Rh) incompatibility, or glucose-6-phosphate dehydrogenase deficiency, with a minimum birth weight of 2500 g.
Arm/Group Title Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Hide Arm/Group Description Saline : Normal saline (0.9%) solution 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
Period Title: Overall Study
Started 15 17 18 8
Completed 15 16 18 8
Not Completed 0 1 0 0
Arm/Group Title Placebo Stanate® 1.5 mg/kg Stanate® 3.0 mg/kg Stanate® 4.5 mg/kg Total
Hide Arm/Group Description Saline : Normal saline (0.9%) solution 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. Total of all reporting groups
Overall Number of Baseline Participants 15 17 18 8 58
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 17 participants 18 participants 8 participants 58 participants
<=18 years
15
 100.0%
17
 100.0%
18
 100.0%
8
 100.0%
58
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=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:  Hours
Number Analyzed 15 participants 17 participants 18 participants 8 participants 58 participants
19.1  (6.8) 16.5  (7.8) 23.3  (10.9) 21.8  (19.8) 20  (10.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 17 participants 18 participants 8 participants 58 participants
Female
7
  46.7%
10
  58.8%
6
  33.3%
6
  75.0%
29
  50.0%
Male
8
  53.3%
7
  41.2%
12
  66.7%
2
  25.0%
29
  50.0%
1.Primary Outcome
Title The Primary Efficacy Endpoint Was the Change in Adjusted Total Serum Bilirubin (TSB) From Baseline to 48 Hours After Treatment.
Hide Description

The primary efficacy endpoint was the change in adjusted TSB from baseline to 48 hours after treatment.

The adjusted Total Serum Bilirubin (TSB) was a calculation of the percentage difference of the TSB level from the age-specific threshold for PT initiation per the AAP Guidelines, ie, an indication of the distance below the PT threshold at the time.

Time Frame 48 hours after injection
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat population (ITT): Defined as all patients who were randomly assigned treatment in the clinical study, had received stannsoporfin or placebo, and had at least 1 post baseline TSB measurement during the first 48 hours after treatment. Patients were summarized based on randomized treatment.
Arm/Group Title Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Hide Arm/Group Description:
Saline : Normal saline (0.9%) solution
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
Overall Number of Participants Analyzed 15 17 18 8
Least Squares Mean (95% Confidence Interval)
Unit of Measure: adjusted TSB (% of Threshold)
0
(0 to 0)
-13.45
(-26.27 to -0.62)
-10.02
(-22.61 to 2.58)
-14.93
(-30.31 to 0.44)
2.Secondary Outcome
Title Change From Baseline in Unadjusted Total Serum Bilirubin (TSB) at 48 Hours (ITT Population
Hide Description Change from Baseline in Unadjusted TSB at 48 Hours (ITT Population)
Time Frame 48 hrs
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin3.0 mg/kg Stannsoporfin 4.5 mg/kg
Hide Arm/Group Description:
Saline : Normal saline (0.9%) solution
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
Overall Number of Participants Analyzed 15 17 18 8
Least Squares Mean (95% Confidence Interval)
Unit of Measure: TSB (mg/dL)
0
(0 to 0)
-1.81
(-3.71 to 0.09)
-1.34
(-3.24 to 0.56)
-2.63
(-4.97 to -0.30)
Time Frame 30 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Hide Arm/Group Description Saline : Normal saline (0.9%) solution 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively. 3 sequential cohorts of approximately 24 subjects will be recruited. Subjects in the first cohort who are randomized to receive active drug treatment will receive a single dose of 1.5 mg/kg by IM injection. Subjects in the second and third cohorts will receive 3.0 and 4.5 mg/kg, respectively.
All-Cause Mortality
Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/15 (13.33%)      0/17 (0.00%)      1/18 (5.56%)      1/8 (12.50%)    
Blood and lymphatic system disorders         
Anemia * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Hepatobiliary disorders         
hyperbilirubinaemia * 1  2/15 (13.33%)  2 0/17 (0.00%)  0 0/18 (0.00%)  0 0/8 (0.00%)  0
Infections and infestations         
meningitis * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Stannsoporfin 1.5 mg/kg Stannsoporfin 3.0 mg/kg Stannsoporfin 4.5 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/15 (33.33%)      8/17 (47.06%)      10/18 (55.56%)      3/8 (37.50%)    
Blood and lymphatic system disorders         
Anaemia * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 2/8 (25.00%)  2
Leukocytosis * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 2/18 (11.11%)  2 0/8 (0.00%)  0
Thrombocytopenia * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
Cardiac disorders         
Bradycardia * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
Gastrointestinal disorders         
Umbilical hernia * 1  1/15 (6.67%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0 0/8 (0.00%)  0
Vomiting * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Hyperbilirubinaemia * 1  2/15 (13.33%)  2 2/17 (11.76%)  2 1/18 (5.56%)  1 0/8 (0.00%)  0
Jaundice * 1  1/15 (6.67%)  1 5/17 (29.41%)  5 0/18 (0.00%)  0 0/8 (0.00%)  0
Infections and infestations         
Anal abscess * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Meningitis * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
Oral candidiasis * 1  2/15 (13.33%)  2 0/17 (0.00%)  0 0/18 (0.00%)  0 0/8 (0.00%)  0
Injury, poisoning and procedural complications         
Contusion * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Investigations         
Blood glucose decreased * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Blood sodium increased * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
C-reactive protein increased * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Carbon dioxide decreased * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Haemoglobin increased * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Haemangioma * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Nervous system disorders         
Depressed level of consciousness * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 0/18 (0.00%)  0 1/8 (12.50%)  1
Skin and subcutaneous tissue disorders         
Acne infantile * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Dermatitis contact * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Dermatitis diaper * 1  1/15 (6.67%)  1 2/17 (11.76%)  2 1/18 (5.56%)  1 0/8 (0.00%)  0
Erythema * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 1/8 (12.50%)  1
Erythema toxicum neonatorum * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 3/18 (16.67%)  3 0/8 (0.00%)  0
Rash * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Rash neonatal * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Rash papular * 1  1/15 (6.67%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0 0/8 (0.00%)  0
Seborrhoeic dermatitis * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
Skin exfoliation * 1  0/15 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0 0/8 (0.00%)  0
Vascular disorders         
Flushing * 1  0/15 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1 0/8 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedRA
It should be noted that the study was discontinued before enrollment of the full 4.5 mg/kg cohort,and therefore, the stannsoporfin 4.5 mg/kg treatment group included 8 patients and the placebo group included 15 patients.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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
Name/Title: Dan Isaacman
Organization: InfaCare Pharmaceutical
Phone: (267) 515-5852
Responsible Party: InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
ClinicalTrials.gov Identifier: NCT00850993     History of Changes
Other Study ID Numbers: 64,185-202
2009-017434-45 ( EudraCT Number )
First Submitted: February 24, 2009
First Posted: February 25, 2009
Results First Submitted: December 6, 2013
Results First Posted: August 29, 2014
Last Update Posted: August 29, 2014