A controlled clinical trial to investigate the safety and effectiveness of Stannsoporfin in newborn children with excess amounts of bilirubin in the blood.
- Conditions
- eonatal hyperbilirubinemiaHiperbilirubinemia neonatalMedDRA version: 14.0Level: PTClassification code 10020580Term: Hyperbilirubinaemia neonatalSystem Organ Class: 10019805 - Hepatobiliary disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2009-017434-45-PL
- Lead Sponsor
- InfaCare Pharmaceutical Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 72
1. Term and late preterm subjects (>=35 and <43 weeks gestational age)
2. Risk factors for hemolytic disease including ABO blood type incompatibility, Rh
incompatibility (anti-C, c, D, E, or e), or G6PD deficiency.
3. A minimum birth weight of 2500 g (5.5 lbs)
4. Coombs positive, or Coombs negative
- If Coombs negative, one or more of the following risk factors must also be
present. These include:
-Jaundice observed in the first 24 hours
-Previous sibling received phototherapy
-Cephalohematoma or significant bruising
-Exclusive breastfeeding
5. Serum bilirubin within 2 mg/dL below the threshold for PT up to 12 hours of age or within 3 mg/dL below the threshold for PT at =12 to 72 hours of age, inclusive
Note: TcB monitoring may be used for screening purposes, however randomization/treatment cannot take place until the TSB is within 2-3 mg/dL of
the PT threshold based on age
6. Willingness for parents/guardians to adhere to recommendations regarding light
precautions
7. Written informed consent obtained from parents/guardians
Are the trial subjects under 18? yes
Number of subjects for this age range: 72
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Clinical suggestion of neonatal thyroid disease or current uncontrolled thyroid disease in the mother (maternal Hashimotos disease is not exclusionary)
2. Treatment or need for treatment in the neonate with medications that may prolong the QT interval (see Appendix C) (erythromycin ointment for eye prophylaxis is permitted), family history of Long QT syndrome or family history of Sudden Infant
Death syndrome
3. Known porphyrias or risk factors for porphyrias, including family history
4. A maternal history of systemic lupus erythematosus
5. Maternal use of phenobarbital 30 days before, or after delivery, if breast-feeding
6. Maternal current drug or alcohol abuse, or maternal history of drug or alcohol abuse that, in the opinion of the Investigator, would not make the subject a suitable
candidate for participation in the clinical trial
7. Apgar score =< 6 at age 5 minutes
8 Significant congenital anomalies or infections
9. Cardiorespiratory distress, defined as a respiratory rate >60 breaths per minute at time of enrollment unless a diagnosis of transient tachypnea of the newborn (TTN) has been made
10. Any abnormal auditory or ophthalmologic findings at Screening
- Subjects who fail initial auditory screen may be rescreened
11. Any excess risk of requiring surgery or exposure to operating room (OR) lights in the foreseeable future
12. Clinically significant abnormalities on screening laboratory evaluation
13. Elevated direct or conjugated bilirubin (>1.0 mg/dL if the TSB is <5.0 mg/dL or
>20% of the TSB if the TSB is >= 5.0 mg/dL)
14. Persistent hypoglycemia (blood glucose <40 mg/dL) despite standard-of-care
treatment
15. Alanine aminotransferase and/or aspartate aminotransferase > 2 times the ULN
16. Abnormal renal function defined as creatinine and/or blood urea nitrogen >2 times the ULN
17. Any blood smear finding of structural red cell abnormalities, such as spherocytosis, not caused by isoimmune hemolysis
18. Temperature instability defined as temperature consistently (3 consecutive times)
<36ºC and/or >37.5ºC axillary
19. Use of photosensitizing drugs or agents (see Appendix D)
20. Use of IVIG or albumins
21. Postdelivery treatment with medications that are known or suspected to displace
bilirubin from albumin (eg, ceftriaxone or sulfa-based antibiotics)
22. Other serious morbid conditions, e.g., pulmonary disease, cardiovascular disease
23. Exposure to any investigational medications or devices after delivery, or participation in another clinical trial while participating in this trial;
24. Any other concurrent medical condition, which in the opinion of the Investigator,
makes the subject unsuitable for the clinical trial
25. Unwillingness for parents/guardians to adhere to recommendations regarding light precautions
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine the safety of 3 ascending doses of stannsoporfin in subjects with hyperbilirubinemia.;Secondary Objective: To determine the efficacy and pharmacokinetics (PK) of 3 ascending doses of stannsoporfin in subjects with hyperbilirubinemia. An exploratory pharmacodynamic analysis will examine the relationship between stannsoporfin exposure and change in TSB from Baseline to 48 hours, post dosing of IMP.;Primary end point(s): The primary efficacy outcome will be based on total serum bilirubin (TSB) measurements adjusted for phototherapy (PT) initiation at both baseline and at 48 hours after treatment. In particular, the change in adjusted TSB levels from baseline to 48 hours after treatment will be considered.<br><br>;Timepoint(s) of evaluation of this end point: Change in adjusted TSB levels from baseline to 48 hours after treatment.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary efficacy endpoints are the following:<br>- Unadjusted change from baseline to 48 hours in TSB measurements<br>- Adjusted and unadjusted change from baseline in TSB measurements at various other timepoints<br>- Proportion of subjects who require phototherapy (PT)/exchange transfusion<br>- Time to PT/exchange transfusion<br>- Duration of PT<br>- Time to hospital discharge<br><br>Safety will be based on the following variables:<br>- Reported adverse events (AEs)<br>- Vital sign measurements<br>- Physical examinations<br>- Electrocardiogram (ECG) recordings<br>- Hearing assessments<br>- Vision assessments<br>- Neurologic assessments<br>- Dermatologic reactions<br>- Clinical laboratory tests;Timepoint(s) of evaluation of this end point: Various.