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Enzyme Replacement Therapy

GZ402665 for Niemann-Pick Disease (ASCEND Trial)

Phase 2 & 3

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

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 52

Awards & highlights

ASCEND Trial Summary

This trial is testing a new drug for adults with a certain enzyme deficiency. The primary objective is to see if the new drug is effective in reducing spleen size and improving lung function. The secondary objectives are to confirm the safety of the new drug and to see if it improves the participant's perception of spleen size, fatigue, pain, and shortness of breath.

Eligible Conditions

Niemann-Pick Disease

ASCEND Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Combination Spleen Endpoint (Primary for US Only): Component 2: Change From Baseline in Splenomegaly-Related Score (SRS) at Week 52

Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline

Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52

+3 more

Secondary outcome measures

Change From Baseline in Dyspnea Severity as Measured by Functional Assessment of Chronic Illness Therapy (FACIT) Dyspnea Scale at Week 52

Change From Baseline in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI)-Item 3 Scale Score at Week 52

Change From Baseline in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score at Week 52

+2 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

Cellulitis

Hepatic Haemorrhage

Lower Limb Fracture

Transient Ischaemic Attack

Gastritis Viral

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Olipudase Alfa

ASCEND Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: GZ402665Experimental Treatment1 Intervention

Olipudase alfa dose (3 mg/kg body weight) in saline administered intravenously once every 2 weeks during the 52 weeks of the primary analysis period for patients randomized to olipudase alfa, and during the extension treatment period for all patients.

Group II: PlaceboPlacebo Group1 Intervention

Placebo (saline) administered intravenously once every 2 weeks during the 52 weeks of the primary analysis period for patients randomized to placebo.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

GZ402665

2015

Completed Phase 3

~70

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,594 Total Patients Enrolled

Clinical Sciences & OperationsStudy DirectorSanofi

857 Previous Clinical Trials

2,019,263 Total Patients Enrolled

Media Library

Olipudase Alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02004691 — Phase 2 & 3

Niemann-Pick Disease Research Study Groups: Placebo, GZ402665

Niemann-Pick Disease Clinical Trial 2023: Olipudase Alfa Highlights & Side Effects. Trial Name: NCT02004691 — Phase 2 & 3

Olipudase Alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02004691 — Phase 2 & 3

Frequently Asked Questions

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

What other drugs has GZ402665 been tested against in previous studies?

"GZ402665 was first looked into in 2013 at location 840001. So far, 5 clinical trials have concluded with 2 more currently underway. Bronx, New york is where many of these studies are being conducted."

Answered by AI

What makes this research unique in the medical field?

"5 clinical studies have been completed for GZ402665 since 2013, with the most recent trials occurring across 9 cities in 17 different countries. The first trial, sponsored by Genzyme, involved 25 patients and successfully completed Phase 2 drug approval."

Answered by AI

Are patients being accepted for enrollment in this research project at this time?

"This particular trial has already stopped recruiting patients. It was initially posted on December 18th, 2015 but was last updated on April 27th, 2022. There are 11 other trials seeking patients with sphingomyelin lipidosis and 2 trials for GZ402665 that are still looking for participants."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

2015. "Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02004691.