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17-alpha-hydroxyprogesterone caproate injectable

Phase 2

Preterm Birth | Small molecule | Other |Pediatrix Medical Group, Inc.|Last Updated: Apr 11, 2016

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment321
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT0016302017OHP for Reduction of Neonatal Morbidity Due to Preterm Birth (PTB) in Twin and Triplet PregnanciesPHASE2 COMPLETED 321Nov 1, 2004Aug 1, 2009Apr 11, 201618 United States
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Study Endpoints
Primary Endpoints
Newborn Respiratory Distress Syndrome (RDS)
Measured from delivery until 30 days after baby was discharged from the hospital

Newborn RDS in the twin arm is defined as compatible symptoms with radiographically confirmed hyaline membrane disease or with respiratory insufficiency of prematurity requiring ventilator support. Data expressed as mean n(%),Odds ratio, CI, and P-value were determined using repeated measures model wherein each twin/triplet within a given pregnancy is considered a repeated measure. Exceptions are comparison with 0 outcomes in one or both groups, so Fisher's Exact Test was used. Morbidity measures were based on live births with data available for the outcomes.

Use of Oxygen Therapy at 28 Days of Newborn Life
Measured at 28 days after birth.

Supplemental oxygen use by the baby measured at the point that the baby reaches 28 days old (after birth)within the twin group.

Newborn Sepsis
measured during the first week following birth

Newborn Sepsis in the twin group was defined as the presence of positive blood culture obtained in the first week of life in association with clinical findings suggesting illness for which the neonate received antibiotics.

Newborn Pneumonia
measure during the first 28 days after birth.

Newborn Pneumonia in the twin group is described as compatible symptoms with diagnostic radiograph findings and positive results on blood cultures, persistent leukopenia

Newborn Intraventricular Hemorrhage Grade 3 or 4
measured during the first 28 days after birth

Newborn Intraventricular hemorrhage (IVH) Stage III in the twin group is described as - IVH with ventricular dilatation. Neonatal Intraventricular hemorrhage (IVH)Stage IV in the twin group is described as - IVH with parenchymal extension.

Newborn Periventricular Leukomalacia (PVL)
measured in the first 28 days after birth.

Newborn Periventricular leukomalacia (PVL) in the twin group is described as the presence of more than 1 obvious hypo echoic cyst in the periventricular white matter.

Newborn Necrotizing Enterocolitis (NEC)Requiring Surgery
measured in the first 28 days after birth

Newborn NEC in the twin group is described as the presence of any of the following: (1)unequivocal intramural air in abdominal radiograph; (2) perforation abdominal radiograph; (3) clinical evidence of perforation (erythema and induration of the abdominal wall or intrabdominal abscess formation); (4) characteristic findings observed at surgery or autopsy; (5) Stricture formation after an episode of suspected necrotizing enterocolitis.

Newborn Retinopathy of Prematurity (ROP)
measured during the first 28 day after birth

Newborn ROP within the twin group is described as retinopathy confirmed on fundoscopic examination, felt to be due to prematurity and subsequent oxygen therapy.

Newborn Asphyxia With Ischemic Injury of Brain, Heart, Kidneys, or Liver
measured during the first 28 days after delivery

Newborn Asphyxia or Hypoxic-ischemic encephalopathy (HEI) within the twin group is characterized by clinical and laboratory evidence of acute or subacute brain injury due to asphyxia (ie, hypoxia, acidosis).

Perinatal Death
measured from randomization to 28 days after birth.

Perinatal death within the twin group is described as a stillbirth, neonatal death, or miscarriage after randomization.

Secondary Endpoints
Individual Components of Neonatal Morbidity (RDS, IVH-III/IV, Bronchopulmonary Dysplasia(BPD), PVL, Sepsis, NEC, ROP-Stage 3/4, Perinatal Death)
measured as any event noted in the first 28 day following birth.
Twins: Delivery Prior to 28 Weeks (Wks), 32 Wks, 34wks, and 37 Wks
Gestational age noted at time of birth
Triplets: Delivery Prior to 28 Wks, 32 Wks, 35 Wks
noted at delivery
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1 Test Group (170HP)ACTIVE_COMPARATORTest Group will receive weekly doses of 170HP via injection as early as 19weeks until 34.0weeks gestation or delivery which ever comes first.
2 - Control (Normal Saline)PLACEBO_COMPARATORControl Group will receive weekly doses of placebo (NS) via injection as early as 19weeks until 34.0weeks gestation or delivery which ever comes first.
Interventions
NameTypeDescription
17-alpha-hydroxyprogesterone caproate injectableDRUG250mg of 17-alpha-hydroxyprogesterone caproate (+ preservatives) injectable weekly starting as early as 19wks gestation until 34.0wks gestation of delivery which ever comes first.
PlaceboDRUGWeekly doses of placebo (NS + preservatives) via injection as early as 19weeks until 34.0weeks gestation or delivery which ever comes first.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexFEMALE
Healthy VolunteersNo
Study Sites18

Inclusion Criteria: 1. Gestational age (GA) 15-23w0d gestational age at the time of recruitment 2. GA 16w0dk to 23w6d at the time of randomization and initiation of injections 3. Maternal age 18 years or older 4. One of these risk factors for spontaneous preterm birth: 1. Twins in current pregn...

Countries:United States
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