E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
This is a prospective, follow-up, designed to provide an assessment of the development of children born to mothers enrolled in the trial of the effectiveness of 17P |
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E.1.1.1 | Medical condition in easily understood language |
This is a prospective, follow-up, designed to provide an assessment of the development of children born to mothers enrolled in the trial of the effectiveness of 17P |
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E.1.1.2 | Therapeutic area | Health Care [N] - Health Care Quality, Access, and Evaluation [N05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036594 |
E.1.2 | Term | Premature birth |
E.1.2 | System Organ Class | 10036585 - Pregnancy, puerperium and perinatal conditions |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to determine whether there is a difference in developmental status between children, aged 23 to 25 months after adjustment for gestational age, whose mothers received 17P and those who received vehicle in the 17P Efficacy Trial. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Each subject must meet the following criteria to be enrolled in this study:
1.Maternal enrollment which resulted in a live birth in the 17P Efficacy Trial: A Multi-center, Randomized, Double-blind Study of Hydroxyprogesterone Caproate Injection, 250 mg/mL, Versus Vehicle for the Prevention of Preterm Birth in Women With a Previous Singleton Spontaneous Preterm Delivery.
2.During their involvement in the above protocol, mothers must have received at least one dose of study drug (Safety population).
3.Children between 22 and 25 months of age adjusted for gestational age.
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E.4 | Principal exclusion criteria |
Subjects meeting any of the following criteria will be excluded from the study:
1.There is no parent/legal guardian available to sign an informed consent.
2.Born to women who are unblinded to study group assignment.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome will be the proportion of children who fall below the specified cut-off (see section 7.2) for at least one of the developmental areas of the ASQ version 3. For each area, a score will be determined as the sum of the score for each question in that area. Each question will be scored as YES=10, SOMETIMES=5 and NOT YET=0. If a question has not been answered then a value equal to the mean of the scores for the questions answered within that developmental area will be used. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
When subjects reach the age of 23 months after adjusting for gestational age, were subjected to analysis for developmental delay using the 24-month ASQ, Version 3. |
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E.5.2 | Secondary end point(s) |
•The proportion of children who have a qualitative description of borderline or worse (score <80) on at least one scale of the Bayley-III.
•The proportion of children who have the same developmental delay identified by both the ASQ and the Bayley-III (see section 7.3.1).
•The proportion of children who have a positive neurological examination (i.e., have at least one abnormality identified on neurological examination).
•ASQ score by developmental area – the proportion of children who fall below the specified cut-off for each of the developmental areas: communication, gross motor, fine motor, problem solving, and personal/social.
•Bayley-III by scale – the proportion of children who have a qualitative description of borderline or worse (score <80) on each of the scales: cognitive, language, motor, social emotional, and adaptive behavior.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
This will be a prospective follow-up study designed to provide a developmental assessment of children born to mothers who participated in the 17P Efficacy Trial. When subjects reach an age of 23 months after adjustment for gestational age, they will be screened for developmental delay using the 24 month ASQ version 3. Subjects who score positive (fall below the specified cut-off) for developmental delay in 1 or more domains will be referred for the 24 month Bayley Scale of Infant and Toddler Development (3rd edition, Bayley-III) and a neurological examination. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
A prospective follow-up study |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
A prospective follow-up study |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Czech Republic |
Hungary |
Italy |
Mexico |
Russian Federation |
Spain |
Ukraine |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 6 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |