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Trial record 1 of 13 for:    patisiran
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Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05040373
Recruitment Status : Recruiting
First Posted : September 10, 2021
Last Update Posted : November 16, 2022
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

Condition or disease
Hereditary Transthyretin-mediated (hATTR) Amyloidosis Polyneuropathy

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Study Type : Observational
Estimated Enrollment : 10 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Patisiran-LNP Pregnancy Surveillance Program
Actual Study Start Date : August 1, 2020
Estimated Primary Completion Date : October 12, 2030
Estimated Study Completion Date : October 12, 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pregnancy
Drug Information available for: Patisiran

Group/Cohort
Patisiran
Pregnant women exposed to commercial patisiran-LNP (ONPATTRO) during the 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.



Primary Outcome Measures :
  1. Prevalence of Major Congenital Malformations [ Time Frame: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) ]
    Major congenital malformations will be classified according to the European Concerted Action on Congenital Anomalies and Twins (EUROCAT) and Metropolitan Atlanta Congenital Defects Program (MACDP) classification systems.


Secondary Outcome Measures :
  1. Prevalence of Minor Congenital Malformations [ Time Frame: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) ]
    Minor congenital malformations will be classified classified according to the EUROCAT and MACDP classification systems.

  2. Prevalence of Pregnancy Outcomes [ Time Frame: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) ]
    Pregnancy outcomes are defined as live birth, spontaneous abortions, stillbirths, elective abortions, molar or pregnancy, ectopic pregnancy, preterm births, and maternal death.

  3. Prevalence of Other Adverse Fetal/Neonatal/Infant Outcomes [ Time Frame: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) ]
    Other adverse fetal/neonatal/infant outcomes are defined as low birth weight, failure to thrive, small for gestational age, postnatal growth and development, neonatal, and perinatal, or infant death.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Any woman exposed to patisiran-LNP 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.
Criteria

Inclusion Criteria:

  • Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before LMP or at any point during pregnancy

Exclusion Criteria:

  • There are no exclusion criteria for participation in this program.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05040373


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com

Locations
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United States, Iowa
Clinical Trial Site Recruiting
Iowa City, Iowa, United States, 52242
France
Clinical Trial Site Recruiting
Nantes, France
Germany
Clinical Trial Site Recruiting
Münster, Germany
Italy
Clinical Trial Site Recruiting
Pavia, Italy
Netherlands
Clinical Trial Site Recruiting
Groningen, Netherlands
Portugal
Clinical Trial Site Recruiting
Lisboa, Portugal
Spain
Clinical Trial Site Recruiting
Madrid, Spain
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05040373    
Other Study ID Numbers: ALN-TTR02-010
First Posted: September 10, 2021    Key Record Dates
Last Update Posted: November 16, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Product Manufactured in and Exported from the U.S.: No
Keywords provided by Alnylam Pharmaceuticals:
Hereditary neuropathic amyloidosis
Familial amyloidosis
Amyloidosis
Amyloidosis senile
Cardiac amyloidosis
Additional relevant MeSH terms:
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Polyneuropathies
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases