GZ402665 for Sphingomyelin Lipidosis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Investigational Site Number 840005, San Francisco, CA
Sphingomyelin Lipidosis+4 More
GZ402665 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

This study is evaluating whether a drug called olipudase alfa can improve the symptoms of acid sphingomyelinase deficiency.

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Eligible Conditions

  • Sphingomyelin Lipidosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Sphingomyelin Lipidosis

Study Objectives

This trial is evaluating whether GZ402665 will improve 8 primary outcomes and 12 secondary outcomes in patients with Sphingomyelin Lipidosis. Measurement will happen over the course of Baseline (Day 1).

Baseline (Day 1)
Combination Spleen Endpoint (Primary for US Only): Component 2: Splenomegaly-Related Score (SRS) at Baseline
Combination Spleen Endpoint: Component 1: Spleen Volume (in MN) at Baseline
Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline
Baseline to Week 52
Change in SRS (except US, where it is part of the primary "combination spleen endpoint")
Change in dyspnea severity as measured by the Functional Assessment of Chronic Illness Therapy dyspnea tool
Change in fatigue severity as measured by item 3 of the Brief Fatigue Inventory scale
Change in pain severity as measured by item 3 of the Brief Pain Inventory scale
Percentage change in diffusing capacity of the lung for carbon monoxide
Percentage change in liver volume
Percentage change in platelet count
Percentage change in spleen volume (combined with change in SRS in the US only, and referred to as "combination spleen endpoint")
Year 5
Number of adverse events
Baseline, Week 52
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
Combination Spleen Endpoint (Primary for US Only): Component 2: Change From Baseline in Splenomegaly-Related Score (SRS) at Week 52
Combination Spleen Endpoint: Component 1: Percent Change From Baseline in Spleen Volume (in MN) at Week 52
Percent Change From Baseline in Liver Volume (in MN) at Week 52
Percent Change From Baseline in Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Week 52
Percent Change From Baseline in Platelet Counts at Week 52

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Sphingomyelin Lipidosis

Trial Design

2 Treatment Groups

GZ402665
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 36 total participants across 2 different treatment groups

This trial involves 2 different treatments. GZ402665 is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 & 3 and have had some early promising results.

GZ402665
Drug
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.
Placebo
Drug
Placebo (saline) administered intravenously once every 2 weeks during the 52 weeks of the primary analysis period for patients randomized to placebo.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to approximately 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline to approximately 5 years for reporting.

Closest Location

Investigational Site Number 840005 - San Francisco, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The participant is male or female aged 18 years or older.
You are willing and able to provide informed consent. show original
The participant has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; and a clinical diagnosis consistent with Niemann-Pick disease type B (NPD B).
The participant has diffuse capacity of the lung for carbon monoxide less than or equal to (<=)70% of the predicted normal value.
The participant must have a spleen volume greater than or equal to (>=)6 multiples of normal (MN) measured by MRI. show original
The participant has an SRS >=5.
Female participants of childbearing potential must have a negative serum pregnancy test for beta-human chorionic gonadotropin (β-HCG).
Female participants of childbearing potential and male participants must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception for up to 15 days following their last dose of study drug.

Patient Q&A Section

What is lipidoses?

"Lipidoses is a group of disorders causing fatty deposition in body tissues. These disorders differ from classic lipidoses in that they can be caused by a range of factors both genetic and environmental. It is not uncommon for both conditions to be present at the same time. It is a rare disorder, seen usually only in children, which is characterised by the presence of accumulation of fat in the body on a particular part of the body. The location is most often assessed using MRI.\n\nWhat is an anxiety disorder?\nanswer: anxiety - the term loosely defined here and elsewhere - is a common mental health related problem." - Anonymous Online Contributor

Unverified Answer

What are the signs of lipidoses?

"The signs and symptoms of lipidoses can be divided into five categories: visceral, neurologic and sensorineural, pulmonary, and cutaneous. Many patients present with non-specific symptoms with no definite explanation and may be managed by an internist or a rheumatologist." - Anonymous Online Contributor

Unverified Answer

How many people get lipidoses a year in the United States?

"Estimates of age-standardized and cohort-derived lipoprotein associated mortality from the National Institute of Health/National Heart, Lung and Blood Institute (NHLBI) Lipid Action Plan Working Group Report imply an overall rate of lipidoses a year in the United States of 13.6/100,000 per year." - Anonymous Online Contributor

Unverified Answer

Can lipidoses be cured?

"Lipidoses are not curable. Lipidoses is an autosomal recessive disorder resulting from impaired activity of LIF. When a child inherits a mutated gene (either frameshift or missense) from the parent of an affected parent, they are at increased risk for developing lipidoses. A parent who knows that their child has lipidoses and/or who experiences a history suggestive of lipidoses, but who does not carry any of the known mutations, should seek genetic evaluation at early ages to provide early detection and proper medical management. When a child inherits the defective gene, its severity increases until the child develops lipoprotein lipase deficiency and lipidoses can develop in early childhood." - Anonymous Online Contributor

Unverified Answer

What causes lipidoses?

"Lipidoses is a disorder of carbohydrate metabolism which seems similar to Fabry's disease, although the lipidoses is much less severe. Genetic testing for Fabry disease might be a useful way of identifying individuals at risk." - Anonymous Online Contributor

Unverified Answer

What are common treatments for lipidoses?

"Several different drugs are used to treat lipidoses, but none are absolutely effective. Although most medications have only modest positive effects, CBT may help some patients to improve their quality of life. More rigorous studies in the area are required to develop specific recommendations." - Anonymous Online Contributor

Unverified Answer

What does gz402665 usually treat?

"There is a large variety of diseases in which gz402665 shows potential. However, gz402665 is mainly administered in the treatment of patients with lipidoses, who are generally old and have few treatment options. The common adverse effects are dizziness, diarrhea and dizzy spells. No side effects have been reported so far and there are no reports of gz402665 causing or worsening of cardiovascular diseases. The dose-response relationship (in respect to cholesterol-control) between gz402665 and lipids is generally linear and the drug is not likely to show additive or antagonistic effects in combination with other lipid-modifying or statin drugs." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets lipidoses?

"For some conditions the mean and median age for the first observation is younger than for other diseases. For myocardial infarction, the mean age is closer to the median than for other diseases." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of gz402665?

"Results from a recent clinical trial is based on a small sample. This was due to the fast and inexpensive development of this drug. It cannot be ruled out that side effects are less common than we thought before. It is also possible that gz402665 has different adverse effects in different people. However, we can confirm one main effect of gz402665 is an increase in weight in more than 50% of people. The weight gain is most common in those who have already been overweight before the treatment with gz402665. The weight gain may lead to a slight decrease in performance in some healthy people, so the increase in weight is not necessarily a sign of a side effect." - Anonymous Online Contributor

Unverified Answer

Has gz402665 proven to be more effective than a placebo?

"In this pilot study, we found no advantage of a single dose from gz426665 over placebo regarding primary and secondary end points, but the study is too small to reveal the true effect of gz426665." - Anonymous Online Contributor

Unverified Answer

How serious can lipidoses be?

"The spectrum of lipidoses is extensive. Even for the more widely recognized familial diseases of GLUT1 deficiency, non-familial cases exist, and lipidoses may be mistaken for other disorders that have features of lipidosis as an underlying cause such as leukodystrophy." - Anonymous Online Contributor

Unverified Answer

How does gz402665 work?

"Gz402665 decreases triglycerides and cholesterol by targeting bile acid synthesis through the blockade of HMG-CoA reductase in liver. However, it also reduces insulin resistance through activating AMP kinase (AMPK) with a similar potency to pioglitazone. Thus, gz402665 provides a novel and powerful strategy to fight both central features of lipid disorders associated with T2D." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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