- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05758922
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 (RAINBOW)
February 23, 2024 updated by: Azafaros A.G.
Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
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.
If approved by the country health authorities, a double-blind extension period will be proposed to the patients who complete the 12-week study.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
13
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Curitiba, Brazil
- Hospital Pequeno Principe
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Porto Alegre, Brazil
- Hospital de Clínicas de Porto Alegre
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Rio De Janeiro, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Male and female patients aged between 12-20 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-epileptic treatment(s) was not stable and/or a new anti-epileptic treatment (drug or procedure) was prescribed during the last month before baseline.
- Total bilirubin >2 x ULN (isolated bilirubin >2 x ULN is acceptable if bilirubin is fractionated and direct bilirubin is <35%).
- Platelet count < 100 x 10^9/L.
- Presence of moderate or severe renal impairment.
- Prior participation in a clinical study with an investigational drug within 3 months prior to Baseline.
- Patient with a positive serum pregnancy test (tested only for women of childbearing potential) at baseline.
- Breast feeding ongoing at baseline or planned during the study.
- ECG with an average of triplicate QTcF interval > 440 msec.
- Received treatment with enantiomers of N-Acetyl-Leucine, gene therapy, stem cell transplantation, or with any other azasugars (iminosugars) compound with similar mechanism of action within 3 months before baseline (except for miglustat for which it is 1 month).
- Any known allergy to azasugars or any excipients.
- Evidence of suicidal ideation with intent (Type 4-5) on the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening. Only in patients judged by the PI cognitively capable to understand the concept of suicide.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
Participant will receive placebo once daily during the course of the study (12 weeks).
|
Pharmaceutical form: capsule Route of administration: oral |
Experimental: AZ-3102 (Dose 1)
Participant will receive AZ-3102 (Dose 1) once daily during the course of the study (12 weeks) and the study extension (if applicable).
|
Pharmaceutical form: capsule Route of administration: oral |
Experimental: AZ-3102 (Dose 2)
Participant will receive AZ-3102 (Dose 2) once daily during the course of the study (12 weeks) and the study extension (if applicable).
|
Pharmaceutical form: capsule Route of administration: oral |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety/tolerability: Incidence and severity of treatment emergent adverse events
Time Frame: Through study completion, up to Week 12
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Through study completion, up to Week 12
|
Assessment of pharmacokinetic (PK) parameters in plasma: Cmax
Time Frame: Through study completion, up to Week 12
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Through study completion, up to Week 12
|
Assessment of PK parameters in plasma: Tmax
Time Frame: Through study completion, up to Week 12
|
Through study completion, up to Week 12
|
Assessment of PK parameters in plasma: AUC0-24h
Time Frame: Concentration versus time curve calculated from time 0 to 24 hours (AUC0-24h)
|
Concentration versus time curve calculated from time 0 to 24 hours (AUC0-24h)
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 24, 2023
Primary Completion (Estimated)
March 1, 2024
Study Completion (Estimated)
December 1, 2024
Study Registration Dates
First Submitted
February 15, 2023
First Submitted That Met QC Criteria
February 24, 2023
First Posted (Actual)
March 8, 2023
Study Record Updates
Last Update Posted (Estimated)
February 26, 2024
Last Update Submitted That Met QC Criteria
February 23, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Mental Disorders
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Lymphatic Diseases
- Neurocognitive Disorders
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Dementia
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Frontotemporal Lobar Degeneration
- Histiocytosis, Non-Langerhans-Cell
- Histiocytosis
- Frontotemporal Dementia
- Pick Disease of the Brain
- Niemann-Pick Diseases
- Niemann-Pick Disease, Type A
- Niemann-Pick Disease, Type C
- Gangliosidoses
- Gangliosidoses, GM2
- Tay-Sachs Disease
Other Study ID Numbers
- AZA-001-5A2-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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