Rare Disease Trial, Not Yet Recruiting (NCT04270058)

Rare Disease Trial, Not Yet Recruiting NCT04270058 Sponsor: Akcea Therapeutics Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT04270058 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

Group 1 – Patients exposed to TEGSEDI (inotersen):

You had at least one dose of the medication TEGSEDI within about 6 months before becoming pregnant, or at any point during your pregnancy.
You are willing and able to give your consent to take part in the study.

Group 2 – Patients not exposed to TEGSEDI:

You have been diagnosed with a hereditary nerve disease called hATTR-PN during your pregnancy.
You have not received TEGSEDI within about 6 months before becoming pregnant, or during your pregnancy.
You are willing and able to give your consent to take part in the study.

Who may not be able to join:

There are no listed exclusion criteria for this trial — anyone who meets the above criteria may be eligible (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 23 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Phone: (844) 962-4787

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT04270058

Sponsor: Akcea Therapeutics

Phase: Not Applicable

Status: Not Yet Recruiting

Condition: Rare Disease

Trial contact: (844) 962-4787

More information: https://clinicaltrials.gov/study/NCT04270058

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Group 1 – Patients exposed to TEGSEDI (inotersen):

You had at least one dose of the medication TEGSEDI within about 6 months before becoming pregnant, or at any point during your pregnancy.
You are willing and able to give your consent to take part in the study.

Group 2 – Patients not exposed to TEGSEDI:

You have been diagnosed with a hereditary nerve disease called hATTR-PN during your pregnancy.
You have not received TEGSEDI within about 6 months before becoming pregnant, or during your pregnancy.
You are willing and able to give your consent to take part in the study.

Who may not be able to join:

There are no listed exclusion criteria for this trial — anyone who meets the above criteria may be eligible (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 23 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Not Yet Recruiting

Phase

Not Applicable

Sponsor

Akcea Therapeutics

Registry

ClinicalTrials.gov

Start date

1 December 2025

Est. completion

15 November 2030

Primary endpoints

Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes; Frequency of Selected Pregnancy Complications

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov