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Enzyme Replacement Therapy

GZ402665 for Niemann-Pick Disease

Phase 2
Waitlist Available
Research Sponsored by Genzyme, a Sanofi Company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to up to 9 years
Awards & highlights

Study Summary

This trial is testing the safety of a drug for a disease that causes problems with fat metabolism.

Eligible Conditions
  • Niemann-Pick Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to up to 9 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to up to 9 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Adverse events (AEs)/treatment-emergent adverse events (TEAEs), including infusion-associated reactions and adverse events of special interest (AESIs)
Clinical laboratory tests
Complete physical examinations including extended neurologic and abbreviated physical exams
+5 more
Secondary outcome measures
Hand X ray for bone age and bone maturation (pediatric patients)
Health outcome questionnaires (adults and pediatric)
Hematology
+5 more

Side effects data

From 2023 Phase 2 & 3 trial • 36 Patients • NCT02004691
67%
Headache
44%
Nasopharyngitis
33%
Upper Respiratory Tract Infection
28%
Cough
22%
Arthralgia
6%
Cellulitis
6%
Transient Ischaemic Attack
6%
Lower Limb Fracture
6%
Gastritis Viral
6%
Hepatic Haemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Olipudase Alfa

Trial Design

1Treatment groups
Experimental Treatment
Group I: GZ402665Experimental Treatment1 Intervention
GZ402665 administered intravenously once every 2 weeks at the dose each patient was receiving at the end of their previous olipudase alfa study, for 9 years or until olipudase alfa becomes commercially accessible, whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
GZ402665
2015
Completed Phase 3
~70

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,617 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,018,930 Total Patients Enrolled

Media Library

Olipudase Alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02004704 — Phase 2
Niemann-Pick Disease Research Study Groups: GZ402665
Niemann-Pick Disease Clinical Trial 2023: Olipudase Alfa Highlights & Side Effects. Trial Name: NCT02004704 — Phase 2
Olipudase Alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02004704 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What other tests have been conducted with GZ402665 as the primary experimental agent?

"Presently, two trials analyzing the effects of GZ402665 are in progress with one evaluation at Phase 3. Although primarily conducted within New york City, 54 sites across the country are participating in these studies."

Answered by AI

Is enrollment in this clinical trial currently available to participants?

"According to clinicaltrials.gov, applications for this trial are not open as of June 1st 2022; the original posting date was December 4th 2013. Nevertheless, 12 other medical experiments remain active and recruiting participants at present."

Answered by AI

What is the designated status of GZ402665 by the Food and Drug Administration?

"Given its Phase 2 status, GZ402665 received a safety rating of 2 - indicating that prior clinical data exists to support the drug's security but there is no evidence demonstrating efficacy."

Answered by AI

How many participants have been included in this trial?

"At this juncture, recruitment for this specific clinical trial has been suspended. The posting of the study was first noted on December 4th 2013 and last edited June 1st 2022. For those seeking participation in similar studies, 10 trials related to sphingomyelin lipidosis are actively recruiting while there are 2 separate investigations involving GZ402665 that can be joined."

Answered by AI

Is this an unprecedented medical trial?

"Currently, 2 trials of GZ402665 are occurring in 9 cities and 17 nations. The first examination began in 2013 under the auspices of Genzyme, a Sanofi Company. They enrolled 25 patients to progress through Phase 2 drug approval stages. Since then, two more investigations have been conducted."

Answered by AI
Recent research and studies
~2 spots leftby Mar 2025