Birth DefectNPERNCT02090088
Nplate® Pregnancy Exposure Registry
An observational study of Birth Defect and Spontaneous Abortions, run by Amgen.
Trial summary
US study to estimate the prevalence at birth of major birth defects (ie, those that cause significant functional or cosmetic impairment, require surgery, or are life-limiting) in children born to mothers who have received Nplate® therapy at any time during the pregnancy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Birth Defect, Spontaneous Abortions, Low Birth Weight
- What is being tested?
- Not applicable- observational study
- How many people
- 4 participants
- Where
- No sites listed yet
- Study dates
- May 2009to Jan 2014
- How long will I take part?
- 21 Months
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population consists of two cohorts of women and their offspring who reside in the U.S. The exposed cohort is defined as pregnant women who have received at least one dose of Nplate® during pregnancy and their offspring up to one year of age. The unexposed cohort is defined as women unexposed to Nplate® during pregnancy matched on disease status (cITP) and age (childbearing) to the exposed. In order to provide context for any possible Nplate® exposed women without cITP, comparators from published literature will be used.
Who may be able to join
- Eligible subjects will be currently pregnant women residing in the US who: Have had any exposure to Nplate® at any time during the pregnancy, which is defined as the period between first day of the last menstrual period (LMP) (ie, within two weeks of conception) up to any date before the date of delivery or end of the pregnancy. The date of conception will be defined as fourteen days prior to the next expected menstrual period for women who report a regular menstrual cycle. If the date of the first day of the LMP is unclear, or if the estimated date of conception as determined by a first-trimester ultrasound differs by more than one week from the date as determined by the first day of LMP, the first-trimester ultrasound-derived date will be used to estimate the timing of exposure to Nplate®, as well as timing of enrollment. If neither a clear date of the first day of the LMP nor a first-trimester ultrasound is available, the estimated date of conception for purposes of enrollment will be determined by best estimate of the woman's prenatal care provider.
- Agree to enroll at any time from the estimated date of conception up to any date before the date of delivery or end of the pregnancy and who have not already had prenatal diagnosis of any major structural birth defect in the current pregnancy prior to enrollment.
- Agree to provide consent for participation in the registry including follow-up interviews.
Who cannot join
- Subjects will be ineligible for study participation if any of the following apply:
- Exposure to Nplate® did not occur during pregnancy.
- Prior knowledge of prenatal diagnosis of a birth defect. Patients who fall under this category will be encouraged to call the Amgen PSP toll free number for enrollment and follow-up.
- Cases reported to the NPER after completion of the exposed pregnancy. Patients who fall under this category will be encouraged to call the Amgen PSP toll free number for enrollment and follow-up.
About this study
Official title: Nplate® Pregnancy Exposure Registry (NPER)
US study to estimate the prevalence at birth of major birth defects (ie, those that cause significant functional or cosmetic impairment, require surgery, or are life-limiting) in children born to mothers who have received Nplate® therapy at any time during the pregnancy.
Read the detailed description
The purpose of the NPER is to monitor pregnancies exposed to Nplate® and to detect and record serious adverse events in infants up to one year after birth. The lack of adequate human fetal safety data for Nplate® makes the NPER an important component of the pharmacovigilance program on the safety of this drug. With respect to fetal outcome, it is important to evaluate the spectrum of outcomes that may be relevant to a medication exposure during pregnancy, and these include both easily recognizable defects that are visible at birth, as well as more subtle or delayed defects that may not be readily identifiable without special expertise and observation beyond the newborn period.
- Conditions
- Birth Defect, Spontaneous Abortions, Low Birth Weight
- Being tested
- Not applicable- observational study
- Medical subject headings
- Congenital Abnormalities, Abortion, Spontaneous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Not applicable- observational studyDrug
| Study group | Role | What they receive |
|---|---|---|
| All Subjects | All Subjects Receives: Drug: Not applicable- observational study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Children Born With Major Birth Defects
An external, independent Congenital Malformation Adjudication Panel (CMAP) comprised of two clinical dysmorphologists and/or teratologists organized malformations based upon organ system and embryology, and determined whether structural defects were major or minor according to a modification of the Metropolitan Atlanta Congenital Defects Program (MACDP) birth defect classification system. A major structural defect is defined as a defect which has either cosmetic or functional significance to the child (eg, a cleft lip), require surgery, or are life-limiting).
Measured: At birth
Secondary outcomes (7)
Number of Children Born With Any 3 or More Minor Birth Defects
An external, independent Congenital Malformation Adjudication Panel (CMAP) comprised of two clinical dysmorphologists and/or teratologists organized malformations based upon organ system and embryology, and determined whether structural defects were major or minor according to a modification of the Metropolitan Atlanta Congenital Defects Program (MACDP) birth defect classification system. A minor structural defect is defined as a defect which occurs in less than 4% of the population but which has neither cosmetic nor functional significance to the child (eg, complete 2,3 syndactyly of the toes).
Measured: At birth
Number of Children Born With a Specific Pattern of Minor Birth Defects
Only those infants who have received medical evaluation and who have three or more minor defects will be considered "affected" for purposes of the evaluation of a pattern of minor defects.
Measured: At birth
Number of Participants With Spontaneous and Elective Abortions or Stillbirths
Number of each of spontaneous abortions, elective abortions, and stillbirths among mothers who received Nplate® therapy at any time during the pregnancy.
Measured: 9 months (during pregnancy)
Number of Children With Preterm Birth or Low Birth Weight
Number of children with preterm birth (<37 weeks gestation) or low birth weight (<2,500 grams) among children born to mothers who have received Nplate® therapy at any time during the pregnancy.
Measured: At birth
Number of Children Born With Intrauterine Growth Restriction
Number of children born with intrauterine growth restriction (weight, length or head circumference less than tenth percentile for sex and gestational age) among mothers who have received Nplate® therapy at any time during the pregnancy.
Measured: At birth
Number of Infants With Adverse Events
In the first year of life, the incidence of all serious adverse events, as well as of nevi (birthmarks) and angiomata (benign tumors with blood vessels or lymph vessels), among infants whose mothers received Nplate® therapy at any time during the pregnancy.
Measured: 12 months from birth
Number of Participants With Adverse Events
Measured: Throughout pregnancy and for up to 6 weeks after delivery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2014 (estimated)
- Study startMay 2009
- Primary completionJan 2014
- Study completionJan 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Birth DefectStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 4 participants (actual)
- Target duration
- 21 Months
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090088
- Sponsor's protocol ID
- 20080046
- Acronym
- NPER
- Status last verified
- Dec 2014
- First submitted
- 14 Mar 2014
- Last updated
- 6 Jan 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nplate® Pregnancy Exposure Registry (NPER)
- ClinicalTrials.gov ID
- NCT02090088
- Acronym / sponsor ID
- NPER / 20080046
- Conditions
- Birth Defect, Spontaneous Abortions, Low Birth Weight
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 4 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- May 2009
- Primary completion
- Jan 2014 (actual)
- Study completion
- Jan 2014 (actual)
- First posted
- 18 Mar 2014 (estimated)
- Results
- Posted 6 Jan 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
US study to estimate the prevalence at birth of major birth defects (ie, those that cause significant functional or cosmetic impairment, require surgery, or are life-limiting) in children born to mothers who have received Nplate® therapy at any time during the pregnancy.
Detailed description
The purpose of the NPER is to monitor pregnancies exposed to Nplate® and to detect and record serious adverse events in infants up to one year after birth. The lack of adequate human fetal safety data for Nplate® makes the NPER an important component of the pharmacovigilance program on the safety of this drug. With respect to fetal outcome, it is important to evaluate the spectrum of outcomes that may be relevant to a medication exposure during pregnancy, and these include both easily recognizable defects that are visible at birth, as well as more subtle or delayed defects that may not be readily identifiable without special expertise and observation beyond the newborn period.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 21 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Subjects | – | Drug: Not applicable- observational studyAll Subjects |
Who may qualify
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population consists of two cohorts of women and their offspring who reside in the U.S. The exposed cohort is defined as pregnant women who have received at least one dose of Nplate® during pregnancy and their offspring up to one year of age. The unexposed cohort is defined as women unexposed to Nplate® during pregnancy matched on disease status (cITP) and age (childbearing) to the exposed. In order to provide context for any possible Nplate® exposed women without cITP, comparators from published literature will be used.
Key inclusion criteria (3 listed)
- Eligible subjects will be currently pregnant women residing in the US who: Have had any exposure to Nplate® at any time during the pregnancy, which is defined as the period between first day of the last menstrual period (LMP) (ie, within two weeks of conception) up to any date before the date of delivery or end of the pregnancy. The date of conception will be defined as fourteen days prior to the next expected menstrual period for women who report a regular menstrual cycle. If the date of the first day of the LMP is unclear, or if the estimated date of conception as determined by a first-trimester ultrasound differs by more than one week from the date as determined by the first day of LMP, the first-trimester ultrasound-derived date will be used to estimate the timing of exposure to Nplate®, as well as timing of enrollment. If neither a clear date of the first day of the LMP nor a first-trimester ultrasound is available, the estimated date of conception for purposes of enrollment will be determined by best estimate of the woman's prenatal care provider.
- Agree to enroll at any time from the estimated date of conception up to any date before the date of delivery or end of the pregnancy and who have not already had prenatal diagnosis of any major structural birth defect in the current pregnancy prior to enrollment.
- Agree to provide consent for participation in the registry including follow-up interviews.
Key exclusion criteria (4 listed)
- Subjects will be ineligible for study participation if any of the following apply:
- Exposure to Nplate® did not occur during pregnancy.
- Prior knowledge of prenatal diagnosis of a birth defect. Patients who fall under this category will be encouraged to call the Amgen PSP toll free number for enrollment and follow-up.
- Cases reported to the NPER after completion of the exposed pregnancy. Patients who fall under this category will be encouraged to call the Amgen PSP toll free number for enrollment and follow-up.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Not applicable- observational study | Drug | All Subjects |
Primary outcome
Number of Children Born With Major Birth Defects
Time frame: At birth
An external, independent Congenital Malformation Adjudication Panel (CMAP) comprised of two clinical dysmorphologists and/or teratologists organized malformations based upon organ system and embryology, and determined whether structural defects were major or minor according to a modification of the Metropolitan Atlanta Congenital Defects Program (MACDP) birth defect classification system. A major structural defect is defined as a defect which has either cosmetic or functional significance to the child (eg, a cleft lip), require surgery, or are life-limiting).
7 secondary outcomes
Number of Children Born With Any 3 or More Minor Birth Defects
Time frame: At birth
Number of Children Born With a Specific Pattern of Minor Birth Defects
Time frame: At birth
Number of Participants With Spontaneous and Elective Abortions or Stillbirths
Time frame: 9 months (during pregnancy)
Number of Children With Preterm Birth or Low Birth Weight
Time frame: At birth
Number of Children Born With Intrauterine Growth Restriction
Time frame: At birth
Number of Infants With Adverse Events
Time frame: 12 months from birth
Number of Participants With Adverse Events
Time frame: Throughout pregnancy and for up to 6 weeks after delivery
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
Questions about this trial
What is this clinical trial studying?
An observational study of Birth Defect and Spontaneous Abortions, run by Amgen.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Jan 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jan 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Children Born With Major Birth Defects (time frame: At birth). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2015.