GZ402665 for Sphingomyelin Lipidosis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Investigational Site Number 250002, Bron Cedex, France
Sphingomyelin Lipidosis+4 More
GZ402665 - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether olipudase alfa is safe for people with acid sphingomyelinase deficiency.

See full description

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 8 secondary outcomes in patients with Sphingomyelin Lipidosis. Measurement will happen over the course of Baseline to after at least 3 years in the study.

Baseline to 5 years
Liver ultrasound/Doppler (patients previously enrolled in DFI13803)
Year 3
Liver biopsy (patients previously enrolled in DFI13412)
Year 9
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
Hand X ray for bone age and bone maturation (pediatric patients)
Health outcome questionnaires (adults and pediatric)
Hematology
Immune response assessments
Linear patient growth by height Z -score (pediatric patients)
Lipid profile
Pulmonary function test
Pulmonary imaging
Safety biomarkers
Spleen and liver volume
Vital signs, electrocardiograms and echocardiograms with Doppler

Trial Safety

Safety Progress

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

Other trials for Sphingomyelin Lipidosis

Trial Design

1 Treatment Group

GZ402665
1 of 1
Experimental Treatment

This trial requires 25 total participants across 1 different treatment group

This trial involves a single treatment. GZ402665 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

GZ402665
Drug
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.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to up to 9 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline to up to 9 years for reporting.

Closest Location

Investigational Site Number 840001 - New York, NY

Eligibility Criteria

This trial is for patients born any sex of any age. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Female patients of childbearing potential and sexually mature male patients must be willing to abstain from sexual activity or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug. show original
A woman of childbearing potential must have a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG) in order to be a candidate for this procedure. show original
The patient completed the treatment period of a previous study of olipudase alfa, and the investigator and sponsor considered the safety profile to be acceptable. show original
The patient or the patient's parent or legal guardian is willing and able to provide written consent that they have been informed of the risks and benefits of the procedure. show original

Patient Q&A Section

Can lipidoses be cured?

"There are no cases of total recovery in our series. In all patients, medical and surgical treatment with a long-term follow-up proved to be effective and safe." - Anonymous Online Contributor

Unverified Answer

What are common treatments for lipidoses?

"The aim of treatment is to keep as many of the symptoms of lipodystrophy in remission. Treatments are varied and differ between individuals and include diet and regular exercise, medications, insulin regimen and the use of insulin for the people who are not able to control their blood sugar level which the doctors will prescribe insulin depending on the severity of symptoms.\n" - Anonymous Online Contributor

Unverified Answer

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

"About 100,000 to 140,000 people per year are diagnosed with liposes in the Unites States. These amounts are probably more frequent than estimated because many patients are asymptomatic during their lifetime and may be under-diagnosed." - Anonymous Online Contributor

Unverified Answer

What is lipidoses?

"Focal lipidoses may cause local edema and atrophy of the fat-filled foci in lean tissues. In cases of systemic or disseminated lipidoses, localized edema and atrophy may lead to secondary fluid retention, peripheral edema, and generalized fluid overload, potentially mimicking lipoid myopathy and cardiac lipohypertrophy (see figure)." - Anonymous Online Contributor

Unverified Answer

What are the signs of lipidoses?

"Signs of lipidoses may include weight loss, abdominal pain, enlarged lymph nodes, enlarged spleen, or rashes. In certain rare conditions, symptoms such as an enlarged liver may indicate a lipidosis, while in others, the presence of lipiduria may indicate a disorder related to lipid metabolism." - Anonymous Online Contributor

Unverified Answer

What causes lipidoses?

"There are many possible causes of lipidoses, which can be further divided by the type of lesion, whether it be infectious or genetic, and even by type of organism. Most commonly, lipidoses are associated with endocrine imbalance. Other possible causes include exposure to radiation, as for hyperlipoproteinemia, metabolic imbalance, and the accumulation of lipids in the body due to lipodystrophy, with or without lipomatosis. As of yet, no causal relationship exists." - Anonymous Online Contributor

Unverified Answer

Does lipidoses run in families?

"In a recent study, findings suggests the existence of LPO2 mutations in families and the need to set up a more detailed survey to ascertain whether LPO2 mutations are a prerequisite of LPO1 or LPO3 in the families studied here." - Anonymous Online Contributor

Unverified Answer

Is gz402665 safe for people?

"Overall, gz402665 had a low overall adverse event rate. Mild but clinically relevant (as per protocol) adverse event were reported for 25% of subjects. The frequency of adverse events was similar (approximately 1/10 subjects) regardless of age at onset or gender. No significant differences were observed between people with lipomyeloedema and people without lipodystrophy receiving gz402665 or placebo. Adverse events did not appear to be dose-dependent. However, the safety of gz402665 had not been investigated in people with lipodystrophies receiving concomitant orlistat therapy. Data from a recent study support the safety of gz402665 in lipodystrophies." - Anonymous Online Contributor

Unverified Answer

What is gz402665?

"Gz402665 was well tolerated in the study population, showing no evidence of toxicity in either the short-term, Phase 1a, or long-term, Phase 1b, studies. It has been granted Orphan Drug Status by the Food and Drug Administration (FDA) for use as a potential treatment for lipidoses. Results from a Phase 1c clinical trial were announced in October 2010. A larger Phase 2a study is planned and will evaluate safety and tolerability in patients with lysosomal storage diseases. A new study was initiated in 2013. Gz402665 has yet to complete clinical development for lysosomal storage diseases, but Phase 1b studies have proven promising results." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of lipidoses?

"Lipidoses are a disease of aging of both whites and persons of African descent. If these two groups can make adjustments in lifestyle, a low rate of lipidoses would likely be achieved." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating lipidoses?

"Most of the recent developments have focused on new treatments for patients with Gaucher disease (GD), Morquio syndrome or the Tay–Sachs disease. There has also been an increase of research related to other lipidoses. Patients suffering from the three main forms of lipidoses can be treated with current treatments, [hepatitis C] (https://www.med.umich.edu/news/ncbi/2013/11/ncbi." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in gz402665 for therapeutic use?

"[The FDA-approved (2007) treatment of gz402665 for inflammatory eye disease has been followed up in clinical trials with an expanded treatment schedule and more frequent follow-up visits (24 weeks rather than 4 weeks for the placebo group). The FDA label also highlights the need for long-term trials of gz402665 as, at the current rate of progression of disease (nausea and dizziness experienced in 1/3 of patients at this dose), gz402665 could be in effect for many years to come." - 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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