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A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

ConditionsGM2 GangliosidosisNiemann-Pick Type C Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorAzafaros B.V.

About this trial

This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Have been randomized into Phase 2 Study AZA-001-5A2-01.

Be male or female aged ≥12 years

Have a genetically-confirmed diagnosis of NPC disease

Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.

Disqualifiers

A positive serum pregnancy test (only tested for women of childbearing potential).

Female planning to breastfeed during the study.

Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.

Participation in another interventional or non-interventional study or early access program.

Trial design

Design model

Single group

Treatments tested in this trial

  • AZ-3102

    Drug

    Daily oral intake of AZ-3102 dispersible tablets

Treatment groups

21 Participants
are divided into 1 treatment group
Group A: All patientsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in treatment-emergent adverse events (TEAEs)

Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death

Time frame
Through study completion, an average of 4 years
2

Change from baseline in electrocardiogram (ECG)

ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done.

Time frame
Through study completion, an average of 4 years
3

Change from baseline in seizures

Seizure duration (minutes) as per the seizure diary.

Time frame
Through study completion, an average of 4 years
4

Change from baseline in seizures

Seizure frequency (number) as per seizure diary.

Time frame
Through study completion, an average of 4 years

Secondary outcomes

1

Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0

Time frame
Baseline, Month 1 (Cohort 2 only) and Month 6
2

Change from Baseline in the concentrations of Neurofilament light chain (NfL)

Time frame
Through study completion, an average of 4 years
3

For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Monosialoganglioside GM2 (GM2)

Time frame
Through study completion, an average of 4 years
4

For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Lyso-monosialoganglioside GM2

Time frame
Through study completion, an average of 4 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Azafaros B.V.

Lead sponsor

Source ClinicalTrials.gov
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