Sickle Cell Disease Trial, Recruiting (NCT03685721)

Sickle Cell Disease Trial, Recruiting NCT03685721 Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Condition: Sickle Cell Disease

Printed from Voxsanity (voxsanity.com.au) — NCT03685721 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Sickle Cell Disease

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 are between 18 and 80 years old.
You are African or of African descent.
You are able to understand and agree to taking part in the trial (give informed consent).

Who may not be able to join:

You have had a blood transfusion in the last 8 weeks.
You have a condition called pyruvate kinase deficiency and are currently taking a medication called AG348 (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 28 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: Swee Lay Thein, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Phone: (301) 480-3765

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: NCT03685721

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

Phase: Not Applicable

Status: Recruiting

Condition: Sickle Cell Disease

Trial contact: (301) 480-3765

More information: https://clinicaltrials.gov/study/NCT03685721

Eligibility summary (AI generated)

Written by an AI model from the 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 are between 18 and 80 years old.
You are African or of African descent.
You are able to understand and agree to taking part in the trial (give informed consent).

Who may not be able to join:

You have had a blood transfusion in the last 8 weeks.
You have a condition called pyruvate kinase deficiency and are currently taking a medication called AG348 (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 28 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

11 October 2018

Est. completion

1 July 2027

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Genotype the 4 PKLR intron-2 variants; Analysis of PK-R transcriptome in red blood cells; Correlation of 2,3-DPG, ATP and pyruvate kinase activities with PKLR intron-2 variants

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: 28 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