Niemann-Pick Type C DiseasePhase 3NCT07054515
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
A Phase 3 study testing AZ-3102 in people with Niemann-Pick Type C Disease and GM1 Gangliosidosis, run by Azafaros B.V. at 39 sites in 17 countries.
Trial summary
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- What is being tested?
- AZ-3102
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 147 participants
- Where
- 39 sitesin 17 countries
- Study dates
- 30 Jun 2025to 14 Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
- Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).
Who cannot join
- Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
- Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
Trial locations and contacts
39 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
39 sites in 17 countries
United States5 sites, 5 recruiting
- Recruiting
Children's Medical Center Dallas
Dallas, Texas, 75235
Charina Ramirez, MD, 214-456-8000, charina.ramirez@utsouthwestern.edu
- Recruiting
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-7404
Lauren Noll, 571-732-4655, lnoll@ldrtc.org
- Recruiting
University of Minnesota Medical School
Minneapolis, Minnesota, 55455
Melissa Fetterly, 612-672-5151, melissa.fetterley@fairview.org
- Recruiting
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, 94609
Veronica Cheung, CRC, 510-428-3885 ext. 2752, Veronica.Cheung@ucsf.edu
Caroline Hastings, MD
- Recruiting
Mayo Clinic Childrens Center - PIN
Rochester, Minnesota, 55905
Brad Jorde, 507-293-3609, RSTCGRESEARCH@mayo.edu
India4 sites, 4 recruiting
- Recruiting
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, 682041
Sheela Nampoothiri, +91 9447978222, sheelanampoothiri@aims.amrita.edu
- Recruiting
JK Lone Hospital
Jaipur, Rajasthan, 302004
Priyanshu Mathur, +91 9982451490, Priyanshujkl@gmail.com
- Recruiting
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, 110029
Faijan Beg, +91 78283 96432, faijan.crds@outlook.com
- Recruiting
Christian Medical College and Hospital
Vellore, Tamil Nadu, 632004
Sangeetha Yoganathan, Dr., +91 9442611622, doc_ys@yahoo.co.in
Australia3 sites, 3 recruiting
- Recruiting
Women's and Children's Hospital
North Adelaide, South Australia, 5006
Louise Jaensch, +61 881 616 610, louise.jaensch@sa.gov.au
- Recruiting
Royal Melbourne Hospital
Parkville, Victoria, 3050
Karen Chidgey, NeuropsychiatryResearch@mh.org.au
- Recruiting
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, 3052
Heidi Peters, +61 03 9345 6205, metabolictrials@mcri.edu.au
Brazil3 sites, 3 recruiting
- Not yet recruiting
Hospital Pequeno Príncipe
Curitiba, 80250-060
Daniel Almeida do Valle, +55 41 9825-0325, daniel.valle@hpp.org.br
- Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, 90560-030
Paula de Castro Pereira, +55 513 359 6340, paulapereira@hcpa.edu.br
Roberto Giugliani
- Recruiting
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, 22250-020
DAFNE D. G. HOROVITZ, +55 21 2554-1709, dafne.horovitz@fiocruz.br
Canada3 sites, 3 recruiting
- Recruiting
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, T3B 6A8
Olugbenga Olunaike, +1 587 885 3158, olugbenga.olunaike@magiccalgany.ca
- Not yet recruiting
University of Alberta Medical Genetics Clinic
Edmonton, Alberta, T6G 2B7
Casey Brodie, +1 780-492-9277, casey.brodey@albertahealthservices.ca
- Not yet recruiting
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
Montreal, Quebec, H2X 0C1
Martine Comeau, +1 514-890-8000 ext. 30153, martine.comeau.chum@ssss.gouv.qc.ca
Turkey (Türkiye)3 sites, 3 recruiting
- Recruiting
Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, 1250
Fatma Derya Bulut, +90 532 743 27 18, deryaozduran@yahoo.com
- Recruiting
Ege Universitesi Tip Fakultesi
Bornova, İzmir, 35100
Sema Kalkhan Ucar, +90 505 779 73 12, sema.kalkan.ucar@ege.edu.tr
- Recruiting
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, 6500
Fatih Suheyl Ezgu, +90 532 685 36 97, fezgu@gazi.edu.tr
United Kingdom3 sites, 3 recruiting
- Recruiting
Great Ormond Street Hospital
London, London, WC1N 3JH
Christopher Jackson, +44 0207 762 6897, christopher.jackson@gosh.nhs.uk
- Not yet recruiting
University College London Hospitals (UCLH)
London, Middlesex, WC1N 3BG
- Recruiting
Royal Manchester Children's Hospital
Manchester, M13 9WL
Richard Curnock, +44 0161 701 2137, richard.curnock@mft.nhs.uk
Argentina2 sites, 1 recruiting
- Active, not recruiting
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ
- Recruiting
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, X5004 ASL
France2 sites, 1 recruiting
- Recruiting
AP-HP - Hôpital Armand Trousseau
Paris, 75012
Aurelie Phelep, +33 01-71-73-82-16, a.phelep@institut-myologie.org
- Active, not recruiting
AP-HP - Hôpital de la Pitié Salpétrière
Paris
Mexico2 sites, 2 recruiting
- Not yet recruiting
Centenario Hospital Miguel Hidalgo
Aguascalientes, 20000
- Not yet recruiting
Hospital Universitario Dr. Jose Eleuterio González
Monterrey, Nuevo León, 64460
Portugal2 sites, 2 recruiting
- Recruiting
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
Lisbon, 1649-035
Tiago Proenca dos Santos, +351 210405814, cic@ulssm.min.saude.pt
- Recruiting
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, 4050-651
Anabela Bandeira, +35 915676520, anabelabandeira.dca@chporto.min-saude.pt
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, 8035
Nuria Leal, +34677171367, nuria.leal@vhir.org
- Recruiting
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, 28009
Beatriz Bernardino Cuesta, neurologia.hnjs@salud.madrid.org
Italy1 site, 1 recruiting
- Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Anna Ardissone, +39 0223942210, anna.ardissone@istituto-besta.it
Pakistan1 site, 1 recruiting
- Not yet recruiting
The Children's Hospital & the University of Child Health Lahore
Lahore, Punjab Province
Sweden1 site
- Active, not recruiting
Sahlgrenska universitetssjukhuset Östra
Gothenburg, Västra Götaland County, 416 50
Switzerland1 site, 1 recruiting
- Recruiting
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, 3010
Sandrina Schneider, +41 31 63 2 07 41, sandrina.schneider@insel.ch
About this study
Official title: 18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
Read the detailed description
This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.
Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.
For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .
- Being tested
- AZ-3102, Placebo
- Keywords
- Nizubaglustat
- Medical subject headings
- Niemann-Pick Disease, Type C, Gangliosidosis, GM1, Gangliosidoses, GM2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZ-3102Drug
Also called nizubaglustat
Oral dispersible tablets
PlaceboDrug
A matching placebo will be administered in the same regimen as the intervention
| Study group | Role | What they receive |
|---|---|---|
| Subprotocol NCT07082725: Intervention Group NPC | Experimental | Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Recruiting) Receives: Drug: AZ-3102; Drug: Placebo |
| Subprotocol NCT07082543: Intervention Group GM1/GM2 | Experimental | Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Active, not recruiting - No longer looking for participants) Receives: Drug: AZ-3102; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants allocated to subprotocol AZA-001-301-NPC
Measured: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Measured: Baseline to month 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2025
- Study start30 Jun 2025
- Primary completion14 May 2028 (estimated)
- Study completion14 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Niemann-Pick Type C DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 147 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Azafaros B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07054515
- Sponsor's protocol ID
- AZA-001-301
- Other IDs
- 2024-515778-28-00
- Status last verified
- Jun 2026
- First submitted
- 27 Jun 2025
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
- ClinicalTrials.gov ID
- NCT07054515
- Acronym / sponsor ID
- AZA-001-301
- Keywords
- Nizubaglustat
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 147 participants (estimated)
- Lead sponsor
- Azafaros B.V.Industry (pharma, biotech, devices)
- Study start
- 30 Jun 2025 (actual)
- Primary completion
- 14 May 2028 (estimated)
- Study completion
- 14 Jun 2028 (estimated)
- First posted
- 8 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
Detailed description
This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.
Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.
For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Subprotocol NCT07082725: Intervention Group NPC | Experimental | Drug: AZ-3102, Drug: PlaceboParticipants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo). (Recruiting) |
| Subprotocol NCT07082543: Intervention Group GM1/GM2 | Experimental | Drug: AZ-3102, Drug: PlaceboParticipants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo). (Active, not recruiting - No longer looking for participants) |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
- Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).
Key exclusion criteria (2 listed)
- Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
- Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AZ-3102Also: nizubaglustatOral dispersible tablets | Drug | Subprotocol NCT07082543: Intervention Group GM1/GM2, Subprotocol NCT07082725: Intervention Group NPC |
| PlaceboA matching placebo will be administered in the same regimen as the intervention | Drug | Subprotocol NCT07082543: Intervention Group GM1/GM2, Subprotocol NCT07082725: Intervention Group NPC |
Primary outcome
Number of participants allocated to subprotocol AZA-001-301-NPC
Time frame: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Time frame: Baseline to month 18
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Niemann-Pick Type C Disease
RecruitingPhase 3
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
Niemann-Pick Type C Disease
RecruitingObservational study
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C
Active, not recruitingPhase 2
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
GM2 Gangliosidosis, Niemann-Pick Type C Disease
Not yet recruitingPhase 2/3
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
Niemann-Pick Disease Type C, GM2 Gangliosidosis
RecruitingObservational study
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
39 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AZ-3102 in people with Niemann-Pick Type C Disease and GM1 Gangliosidosis, run by Azafaros B.V. at 39 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, India, Australia, Brazil, Canada and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patient Advocacy Representative (Please reach out by email, info@azafaros.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants allocated to subprotocol AZA-001-301-NPC (time frame: Baseline to month 18). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.