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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up months 6 and 12

Awards & highlights

Study Summary

This trial studies the effects of a drug (arimoclomol) to treat Niemann-Pick Disease (NPC) in people with confirmed diagnosis, compared with a placebo. It includes a pediatric sub-study with participants 6 months to 24 months old.

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.Check my eligibility

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.See study design

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.

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

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

Primary outcome measures

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

Secondary outcome measures

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")

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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 / 5Eligibility criteria met