Rare Disease Trial, Recruiting (NCT06795152)

Rare Disease Trial, Recruiting NCT06795152 Sponsor: Duke University Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT06795152 — 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 have been diagnosed with a rare Glycogen Storage Disease (GSD), specifically one of these types: GSD 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, or Danon disease
Your diagnosis has been confirmed through genetic testing showing one or two faulty gene variants linked to your specific type of GSD (the number required depends on how the condition is inherited)
Your diagnosis has been confirmed through enzyme activity testing in liver, muscle, skin, or other body tissue
Your diagnosis has been confirmed by a doctor based on tissue samples (a biopsy) or other clinical evidence
You are able to give your own permission to take part (if you are an adult), or a parent, guardian, or legal representative can give permission on your behalf (for children or adults who need assistance with decision-making)
You are able to give permission for your medical records to be shared with the research team
Pregnant women who have been diagnosed with one of the rare GSDs listed above are welcome to participate

Who may not be able to join:

You are not able to give your own consent to take part, and there is no parent, guardian, or legal representative available to give consent on your behalf

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: Priya Kishnani, M.D., Duke

Phone: 919-681-8823

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: NCT06795152

Sponsor: Duke University

Phase: Not Applicable

Status: Recruiting

Condition: Rare Disease

Trial contact: 919-681-8823

More information: https://clinicaltrials.gov/study/NCT06795152

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 have been diagnosed with a rare Glycogen Storage Disease (GSD), specifically one of these types: GSD 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, or Danon disease
Your diagnosis has been confirmed through genetic testing showing one or two faulty gene variants linked to your specific type of GSD (the number required depends on how the condition is inherited)
Your diagnosis has been confirmed through enzyme activity testing in liver, muscle, skin, or other body tissue
Your diagnosis has been confirmed by a doctor based on tissue samples (a biopsy) or other clinical evidence
You are able to give your own permission to take part (if you are an adult), or a parent, guardian, or legal representative can give permission on your behalf (for children or adults who need assistance with decision-making)
You are able to give permission for your medical records to be shared with the research team
Pregnant women who have been diagnosed with one of the rare GSDs listed above are welcome to participate

Who may not be able to join:

You are not able to give your own consent to take part, and there is no parent, guardian, or legal representative available to give consent on your behalf

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

Duke University

Registry

ClinicalTrials.gov

Start date

23 December 2024

Est. completion

1 December 2034

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Progression of disease confirmed by medical record review

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