What side effects are associated with this type of intervention?

The most common treatments for Niemann-Pick Disease, such as miglustat and arimoclomol, have several known side effects. Miglustat can cause gastrointestinal issues like diarrhea, weight loss, and flatulence. It may also lead to tremors and peripheral neuropathy. Arimoclomol, which enhances heat shock protein production, is generally well-tolerated but can cause mild to moderate side effects such as headache, fatigue, and gastrointestinal discomfort. It is important to monitor these side effects and discuss them with a healthcare provider to manage them effectively.

What prior FDA approvals exist?

The FDA has approved miglustat (Zavesca) for the treatment of Niemann-Pick Disease Type C (NPC). Miglustat works by inhibiting the synthesis of glycosphingolipids, which accumulate in cells of patients with NPC. While miglustat does not enhance heat shock proteins like arimoclomol, it addresses the underlying issue of lipid accumulation. There are no other FDA-approved treatments specifically for NPC that function similarly to arimoclomol by enhancing heat shock proteins.

What other types of research exist?

As of now, there are no specific novel alternatives to Arimoclomol mentioned for Niemann-Pick Disease Type C in the provided context. However, potential alternatives could include therapies that target similar mechanisms, such as enhancing protein folding, reducing cellular stress, or addressing lysosomal storage issues. Consulting with a healthcare provider for the latest research and clinical trials is recommended.

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Arimoclomol for Niemann-Pick Disease

Phase 2 & 3

Waitlist Available

Led By Karl-Eugen Mengel

Research Sponsored by Orphazyme

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Presenting at least one neurological symptom of the disease

All sexually active female participants of child-bearing potential must use highly effective contraception

Must not have

Neurologically asymptomatic participants

Severe liver insufficiency

Timeline

Screening 3 weeks

Treatment Varies

Follow Up months 6 and 12

Summary

This trial tests arimoclomol as an additional treatment for patients with Niemann-Pick disease type C, including young children. The drug helps cells handle stress, potentially reducing disease damage. The study aims to see if arimoclomol is safe and effective.

Who is the study for?

This trial is for individuals aged 2 to under 19 years with Niemann-Pick disease type C, confirmed by genetic analysis or specific criteria. They must be able to walk (with assistance if needed), comply with the study's procedures, and use effective contraception if sexually active. Those treated with miglustat are eligible if stable. Excluded are those on other investigational drugs within the last month, pregnant or breastfeeding women, neurologically asymptomatic patients, severe NP-C cases affecting protocol compliance, liver transplant recipients or candidates, and those with severe liver/renal issues or uncontrolled seizures.

What is being tested?

The study tests arimoclomol against a placebo in participants who continue their routine care which may include miglustat. It aims to determine arimoclomol's effectiveness and safety as an add-on therapy for NPC. The trial includes a randomized double-blind design where neither participants nor researchers know who receives the actual drug versus placebo.

What are the potential side effects?

While not specified here, potential side effects of arimoclomol could range from mild reactions at administration sites to more serious systemic responses depending on individual tolerance and previous health conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am experiencing symptoms related to my nervous system.

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I am using effective birth control methods.

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My BMI is within the normal range for my age.

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My Niemann-Pick disease type is confirmed by genetic tests.

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I am between 2 and 18 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any symptoms related to my nervous system.

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My liver is not working well.

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I have had or am scheduled for a liver transplant.

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My kidneys do not work properly.

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I haven't had severe uncontrolled seizures in the last 2 months.

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My NP-C disease symptoms are severe and may affect my participation.

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I have not taken any experimental drugs in the last 4 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ months 6 and 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~months 6 and 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and months 6 and 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores

Secondary study objectives

Change From Baseline in 17-domain NPCCSS Apart From Hearing Domains (i.e. Hearing and Auditory Brainstem Response)

Change From Baseline in 5-domain NPCCSS Score

Change From Baseline in the NPC Clinical Database (NPC-CDB) Score (Modified "Stampfer Score")

+10 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Arimoclomol Single PK DoseExperimental Treatment1 Intervention

Participants less than 12 years received a single oral dose of arimoclomol capsule, based on participant's body weight, on Day 1.

Group II: Arimoclomol (12-month Double-blind Phase)Experimental Treatment1 Intervention

Participants received arimoclomol capsules orally three times a day (TID) for 12 months. The dose was 31-124 mg arimoclomol base TID (equivalent to 50-200 mg arimoclomol citrate TID), based on participant's body weight.

Group III: Placebo (12-month Double-blind Phase)Placebo Group1 Intervention

Participants received matching placebo capsules (with regard to weight, appearance, smell, flavor etc.) orally TID for 12 months.

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Niemann-Pick Disease (NPC) often focus on enhancing the production of heat shock proteins (HSPs), which are crucial for proper protein folding and function. Arimoclomol, for instance, increases HSP levels, helping to reduce cellular stress and damage caused by lipid accumulation in NPC. This mechanism is particularly important for NPC patients as it addresses the root cause of cellular dysfunction, potentially improving cell health and slowing disease progression.

Modulation of Plasma Membrane Composition and Microdomain Organization Impairs Heat Shock Protein Expression in B16-F10 Mouse Melanoma Cells.Pharmacological induction of heat shock proteins ameliorates toxicity of mutant PKCγ in spinocerebellar ataxia type 14.The discovery of Hsp70 domain with cell-penetrating activity.