Autopsy report · NCT00410566

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

“Terminated by sponsor - Single dose safety objective achieved.”

The sponsor’s own words in the registry.

Life of this trial

• Ran for about 2 years 4 months before it stopped.
• 11 participants enrolled.
• No results are posted on the registry.

Vital statistics

Status

Terminated

Phase

Phase 1

Sponsor

Genzyme, a Sanofi Company

Sponsor type

Industry

Conditions

Acid Sphingomyelinase Deficiency; Niemann-Pick Disease

Interventions

rhASM; rhASM; rhASM; rhASM; rhASM

Participants

11

Sites

1

Countries

United States

Trials that died the same way

• A Study of THE-630 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)
  “The Sponsor terminated the study due to early dose-limiting toxicities.” (77% match in meaning)
• A Phase II Study of Belinostat in Combination With Bortezomib in Patients With Relapsed, Refractory Multiple Myeloma
  “Terminated due to dose limiting toxicity” (72% match in meaning)
• CIVO Intratumoural Microdosing of Anti-Cancer Therapies in Australia
  “Termination by pharmaceutical sponsor” (66% match in meaning)
• Evaluation of Revumenib in Participants With Colorectal Cancer and Other Solid Tumors
  “Study was terminated by sponsor after Phase 1b as pre-defined signals of efficacy were not met in the Phase 1b dose expansion cohort.” (66% match in meaning)
• A Dose Finding Study to Investigate the Safety and Effectiveness of GSK1070806 in Adult Participants With Moderate to Severe Atopic Dermatitis
  “Sponsor decision to early terminate the study based on the pre-defined futility criterion for efficacy. No safety concerns were identified.” (65% 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.