Rare Disease Trial, Recruiting (NCT02543996)

Rare Disease Trial, Recruiting NCT02543996 Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT02543996 — 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:

You must be older than 1 month of age
Pregnant women who have been diagnosed with or are suspected to have the condition being studied, or who become pregnant during the study
Pregnant women who are related to someone with the condition (including spouses or partners), but only to donate cord blood and tissue at the time of delivery
People with cognitive impairment (difficulties with thinking or understanding) who have the condition being studied
People with cognitive impairment who are related to someone with the condition being studied
People who are willing and able to give their informed consent to participate

Who may not be able to join:

Healthy volunteers who are not able to give their own informed consent
People with cognitive impairment who do not have the condition being studied
People with cognitive impairment who are not related to someone with the condition being studied

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 20 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

Principal Investigator: Manfred Boehm, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Phone: (301) 402-9841

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: NCT02543996

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Phase: Not Applicable

Status: Recruiting

Condition: Rare Disease

Trial contact: (301) 402-9841

More information: https://clinicaltrials.gov/study/NCT02543996

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:

You must be older than 1 month of age
Pregnant women who have been diagnosed with or are suspected to have the condition being studied, or who become pregnant during the study
Pregnant women who are related to someone with the condition (including spouses or partners), but only to donate cord blood and tissue at the time of delivery
People with cognitive impairment (difficulties with thinking or understanding) who have the condition being studied
People with cognitive impairment who are related to someone with the condition being studied
People who are willing and able to give their informed consent to participate

Who may not be able to join:

Healthy volunteers who are not able to give their own informed consent
People with cognitive impairment who do not have the condition being studied
People with cognitive impairment who are not related to someone with the condition being studied

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 20 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Registry

ClinicalTrials.gov

Start date

17 September 2015

Est. completion

4 June 2040

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

To procure human biospecimens from affected and unaffected cohorts for on-going and future basic and translational research studies to identify underlying disease mechanism(s)

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: 20 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