Active — Not RecruitingPhase 2

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

Brazil13 participantsStarted 2023-04-24

Plain-language summary

This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.

Who can participate

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male and female patients aged above 12 years old at informed consent signature. * GM2 patients : Genetically and biochemically confirmed diagnosis of Tay-Sachs or Sandhoff disease. * NP-C patients : Genetically confirmed diagnosis of NP-C. * NP-C patients : Miglustat-naïve patients unwilling or unable to take miglustat, OR, patients who have discontinued miglustat because of confirmed safety/tolerability issues. Miglustat must have been discontinued at least 1 month prior to Baseline visit. * Total SARA score ≥ 1 at Baseline. * A male participant with a female partner of childbearing potential is eligible if he agrees to follow the contraceptive guidance. * If a female participant is a WOCBP and is having a male partner, she must agree to follow the contraceptive guidance. * Willing and able to complete protocol assessments. * Parent and/or legal guardian is able to read, understand, and sign the informed consent. Where appropriate, assent will also be sought for patients who have not reached the age of majority or who are not able to sign the consent form. Exclusion Criteria: * Any abnormal conditions at baseline visit which, in the opinion of the PI; could interfere with study assessments (e.g., severe infection). * History of medical conditions other than GM2 gangliosidosis/NP-C that, in the opinion of the PI; would confound scientific rigor or interpretation of results. * Presence of another inherited neurologic disease. * The dose of anti-epilept…

What they're measuring

Safety/tolerability: Incidence and severity of treatment emergent adverse events

Timeframe: Through study completion, up to Week 12

Assessment of pharmacokinetic (PK) parameters in plasma: Cmax

Timeframe: Through study completion, up to Week 12

Assessment of PK parameters in plasma: Tmax

Timeframe: Through study completion, up to Week 12

Assessment of PK parameters in plasma: AUC0-24h

Timeframe: Concentration versus time curve calculated from time 0 to 24 hours (AUC0-24h)

Trial details

NCT IDNCT05758922

SponsorAzafaros A.G.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-04-19

View on ClinicalTrials.gov More GM2 Gangliosidosis trials

Plain-language summary

Who can participate

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Safety/tolerability: Incidence and severity of treatment emergent adverse events

Timeframe: Through study completion, up to Week 12

Assessment of pharmacokinetic (PK) parameters in plasma: Cmax

Timeframe: Through study completion, up to Week 12

Assessment of PK parameters in plasma: Tmax

Timeframe: Through study completion, up to Week 12

Assessment of PK parameters in plasma: AUC0-24h

Timeframe: Concentration versus time curve calculated from time 0 to 24 hours (AUC0-24h)