NCT07530796 is a Phase 1, PHASE2 clinical trial of scAAV9/AGA in Aspartylglucosaminuria, sponsored by Rare Trait Hope.

Who is sponsoring NCT07530796?

NCT07530796 is sponsored by Rare Trait Hope.

What drugs are being tested in NCT07530796?

Interventions in NCT07530796: scAAV9/AGA.

What condition does NCT07530796 study?

NCT07530796 studies Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency.

Is NCT07530796 still recruiting?

NCT07530796 is currently not yet recruiting. Last updated 15 April 2026.

Last reviewed 2026-04-15 · How we verify

NCT07530796

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

Not yet recruiting Phase 1, PHASE2 Last updated 15 April 2026

What this trial tests

Phase 1, PHASE2 trial testing scAAV9/AGA in Aspartylglucosaminuria in 9 participants. Not yet recruiting.

Timeline

1 May 2026

Primary endpoint
1 May 2029

1 May 2032

Quick facts

Lead sponsor	Rare Trait Hope
Phase	Phase 1, PHASE2
Status	Not yet recruiting
Study type	INTERVENTIONAL
Allocation	na
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	9
Start date	1 May 2026
Primary completion	1 May 2029
Estimated completion	1 May 2032

Drugs / interventions tested

scAAV9/AGA

Conditions studied

Aspartylglucosaminuria — all drugs for Aspartylglucosaminuria →
Aspartylglucosamidase (AGA) Deficiency — all drugs for Aspartylglucosamidase (AGA) Deficiency →

Sponsor

Rare Trait Hope

Who can join

Adults 4 to 45, any sex, with Aspartylglucosaminuria or Aspartylglucosamidase (AGA) Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

Verify or expand the search:

Other recruiting trials for Aspartylglucosaminuria

Currently open trials in the same condition.

NCT02171104 — MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis · Phase 2 · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT07530796 (US National Library of Medicine, public domain)
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Rare Trait Hope
Last refreshed: 15 April 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07530796.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing