Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

NCT06312644 | Recruiting FDA Drug

• 1 other identifier: D9289C00007
• Study Type: observational
• Target: 75
• Locations: 7 countries
• Sites: 7

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.

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

Outcome Measures

Primary Outcomes (3)

• Pregnancy Complications

  Pregnancy outcomes

  Up to 4 weeks post-delivery

• 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

  Up to 4 weeks post-delivery

• 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

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

Interventions

Ultomiris DRUG

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.

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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 \[SmPC\])

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (7)

Research Site

Boston, Massachusetts, 02210, United States

RECRUITING

Research Site

Melbourne, Victoria, 3050, Australia

NOT YET RECRUITING

Research Site

Paris, 75475, France

RECRUITING

Research Site

Essen, Norte-Westfalia, D-45147, Germany

RECRUITING

Research Site

Rome, 00168, Italy

RECRUITING

Research Site

Seoul, South Korea

ACTIVE NOT RECRUITING

Research Site

London, SE5 9NU, United Kingdom

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Atypical Hemolytic Uremic Syndrome; Myasthenia Gravis; Neuromyelitis Optica

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Hemolytic-Uremic Syndrome; Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases

Study Officials

• Sydney Williams

  North American Coordinating Center (NACC)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Target Duration: 21 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2024
• First Posted: March 15, 2024
• Study Start: December 16, 2024
• Primary Completion (Estimated): July 11, 2034
• Study Completion (Estimated): July 11, 2034
• Last Updated: April 16, 2026

Record last verified: 2026-04

Locations

GB (1); AU (1); US (1); FR (1); IT (1); KR (1); DE (1)