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RecruitingNCT06312644Updated Apr 16, 2026

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

An observational study in Ultomiris-exposed Pregnant/ Postpartum, Pregnancy and Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Recruiting at 7 sites in 7 countries. Open to female participants. Per ClinicalTrials.gov, last updated 2026-04-16.

Sponsored by Alexion Pharmaceuticals, Inc. · Observational

Study type
Observational
Model
Other
Time perspective
Other
Enrollment
75
Sex
Female
01

Study 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.

Read the 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.

02

Conditions studied

  • 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
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
Female
Accepts healthy volunteers
No
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.

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

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
04

Study design

Observational model
Other
Time perspective
Other
Enrollment
75 participants (estimated)
Target follow-up
21 Months
Patient registry
Yes

Interventions

  • DrugUltomiris

    Participants exposed to Ultomiris at any point during the 40 weeks prior to conception (LMP+14 days) or at any point during pregnancy or breastfeeding up to 52 weeks after birth.

    Also known as: There is no interventional drug. Ultomiris treatment is an inclusion criterion.

05

What researchers measure

Primary outcomes

  1. Pregnancy Complications

    Pregnancy outcomes

    Time frame: Up to 4 weeks post-delivery

  2. Maternal Complications

    Maternal Complications may include pre-eclampsia, eclampsia, deep-vein thrombosis (DVT), pregnancy-induced hypertension, gestational diabetes, preterm labor, placenta previa, and postpartum hemorrhage

    Time frame: Up to 4 weeks post-delivery

  3. Fetal/Infant Outcomes

    Fetal (in Utero)/infant (for Live Born Births) may include major congenital malformation (MCMs), full-term birth, preterm birth, low birth weight, size for gestational age, neonatal death, perinatal death, infant death, serious and severe infections of infants, infant hospitalization, growth delays, and abnormal postnatal growth and development

    Time frame: In utero through 52 weeks of age after exposure to Ultomiris (in utero or via breastmilk)

06

Study locations

4 of 7 sites recruiting
  • Research Site
    Boston, Massachusetts 02210, United States
    Recruiting
  • Research Site
    Melbourne, Victoria 3050, Australia
    • Dr. Jeff Szer · Principal investigator
    Not yet recruiting
  • Research Site
    Paris, 75475, France
    • Dr Flore Sicre de Fontbrune · Principal investigator
    Recruiting
  • Research Site
    Essen, Norte-Westfalia D-45147, Germany
    • Prof. Dr. Alexander Röth · Principal investigator
    Recruiting
  • Research Site
    Rome, 00168, Italy
    • Doctor Simona Sica · Principal investigator
    Recruiting
  • Research Site
    Seoul, South Korea
    Active, not recruiting
  • Research Site
    London, SE5 9NU, United Kingdom
    Active, not recruiting
07

Registry details

Key details

Study ID
NCT06312644
Lead sponsor
Alexion Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Mar 15, 2024
Start date
Dec 16, 2024
Primary completion
Jul 11, 2034 (estimated)
Completion
Jul 11, 2034 (estimated)
Last update
Apr 16, 2026

Study contacts

Alexion Pharmaceuticals, Inc. (Sponsor)
Contact
clinicaltrials@alexion.com
1-855-752-2356
Sydney Williams
principal investigator · North American Coordinating Center (NACC)

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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