Clinical trial NCT06312644: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Official title: Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Ultomiris-exposed Pregnant/ Postpartum
Pregnancy
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Atypical Hemolytic Uremic Syndrome (aHUS)
Generalized Myasthenia Gravis (gMG)
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Keywords: Ultomiris
pregnancy
PNH
aHUS
gMG
NMOSD
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2024-03-15
Enrollment: 75
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants exposed to Ultomiris at any point up to 40 weeks prior to conception (Last Menstrual Period +14 days) through pregnancy and/or breastfeeding through the first 52 weeks of the infant's life.

Brief summary: The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.

Detailed description: This observational study will collect prospective and retrospective data in participants exposed to Ultomiris during pregnancy and/or breastfeeding to assess risk of maternal complications, adverse effects on the developing fetus, and adverse effects on the infant.

Eligibility criteria: Inclusion Criteria:

* Female participant must have a medically confirmed qualifying pregnancy (prospectively or retrospectively identified).
* Participant informed consent (written or e-consent per local regulations or ethics committee requirements) must be obtained prior to the participant's enrollment. If the participant is a minor, consent must be obtained from the parent or legal guardian, with assent from the minor (as locally appropriate).
* Willing to provide contact information for the participant.
* Willing to authorize HCP(s) to release maternal and infant medical information to the study, upon request, if applicable to current local regulations.
* Diagnosed with an indication for which Ultomiris is approved, based on HCP or medical records.
* Exposed to Ultomiris at any point during the defined exposure window based on HCP or medical record documentation. (If exact exposure dates are unknown, the reporter must be able to specify or estimate trimester or timing of exposure \[prior to conception as LMP+14 days, or during breastfeeding\].)
* Use of Ultomiris per local product information (i.e., United States Prescribing Information \[USPI\] or summary of product characteristics \[SmPC\])

Exclusion Criteria:

* Participants who are unable to provide consent or assent (as locally appropriate) (e.g., diagnosed with severe psychiatric conditions or severe intellectual disabilities) will be excluded from this study

Primary outcomes:
measure: Pregnancy Complications
timeFrame: Up to 4 weeks post-delivery
measure: Maternal Complications
timeFrame: Up to 4 weeks post-delivery
measure: Fetal/Infant Outcomes
timeFrame: In utero through 52 weeks of age after exposure to Ultomiris (in utero or via breastmilk)
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: North American call center (NACC)
state: Massachusetts
Study officials:
affiliation: North American Coordinating Center (NACC)
name: Sydney Williams
role: PRINCIPAL_INVESTIGATOR
References: