Monitoring pregnancy outcomes in women with hereditary transthyretin amyloidosis
Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy
This study is trying to see how pregnancy goes for women with hereditary transthyretin amyloidosis and if the treatment TEGSEDI makes a difference for them and their babies.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Sex | Female |
| Sponsor | Akcea Therapeutics Industry-sponsored |
| Locations | 1 site (Morgantown, West Virginia) |
| Trial ID | NCT04270058 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the safety of pregnancy outcomes in women diagnosed with hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN). It will compare outcomes between women who have been exposed to the treatment TEGSEDI and those who have not. The study will also evaluate pediatric outcomes for children up to one year of age. Participants will be recruited from a specialized pregnancy coordinating center in Morgantown, West Virginia.
Who should consider this trial
Good fit: Ideal candidates include pregnant women diagnosed with hATTR-PN, either exposed or unexposed to TEGSEDI.
Not a fit: Patients who are not pregnant or do not have a diagnosis of hATTR-PN will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety of TEGSEDI during pregnancy, potentially guiding treatment decisions for affected women.
How similar studies have performed: While there may be limited data on similar studies, this approach is relatively novel in the context of hATTR-PN and pregnancy.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: TEGSEDI exposed patients (Cohort 1) meeting the following criteria will be eligible for study entry: 1. Pregnancies exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy. 2. Able and willing to provide informed consent. Unexposed patients (Cohort 2) meeting the following criteria will be eligible for study entry: 1. Have a diagnosis of hATTR-PN during pregnancy. 2. Have not been exposed to TEGSEDI within 25 weeks prior to conception or during pregnancy. 3. Able and willing to provide informed consent. Exclusion Criteria: None
Where this trial is running
Morgantown, West Virginia
- TEGSEDI Pregnancy Coordinating Center — Morgantown, West Virginia, United States (Recruiting)
Study contacts
- Study coordinator: Ionis Pharmaceuticals
- Email: ionisNCT04270058study@clinicaltrialmedia.com
- Phone: (844) 962-4787
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.