Autopsy report · NCT04669535

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

“Sponsor withdrew from the study and there was a lack of funding to complete stage 2 of the designed study.”

The sponsor’s own words in the registry.

Life of this trial

• Registered 2 months before it started.
• Ran for about 3 years 11 months before it stopped.
• 9 participants enrolled.
• Results were posted on the registry 5 months after it ended.

Vital statistics

Status

Terminated

Phase

Phase 1

Sponsor

Terence Flotte

Sponsor type

University, hospital or other

Conditions

Tay-Sachs Disease; Sandhoff Disease

Interventions

AXO-AAV-GM2 Starting Dose; AXO-AAV-GM2 Low Dose; AXO-AAV-GM2 Middle Dose; AXO-AAV-GM2 High Dose

Participants

9

Sites

2

Countries

United States

Trials that died the same way

• Manual Therapy/Exercises vs. Physiotherapy/Back School vs. Waiting List in Patients With Chronic Low Back Pain
  “Sponsor did not want to continue the study.” (80% match in meaning)
• BLOOM: Boldly Living outdOOrs for Mental Health
  “Study was withdrawn because available funding ended and could not be extended. No participants were enrolled.” (80% match in meaning)
• Pasireotide in Hyperinsulinemic Hypoglycemia
  “Funding was withdrawn by Sponsor prior to start of study” (80% match in meaning)
• Navelbine, Taxol, Herceptin and Neupogen in Stage IV Breast Cancer: A Phase I - II Trial
  “Sponsor withdrew the study” (79% match in meaning)
• NRX-1074 in Early Course Schizophrenia
  “The study was withdrawn by the sponsor.” (79% match in meaning)

Look deeper

• The official registry record (the source of truth)
• Record history: every change the sponsor made
• Open it in the database

Everything here comes from the current ClinicalTrials.gov record. The timeline uses the record’s dates; dates marked planned are the sponsor’s plans. The cause of death is an automatic, unverified label. This is research information, not medical advice.