Cerebral AdrenoleukodystrophyPhase 2NEXUSNCT04528706
A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
A Phase 2 study testing MIN-102 in people with Cerebral Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 5 sites in 4 countries.
Trial summary
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT) Read more about this study
- Who is it for?
- Ages 2 to 12 years, men only
- Conditions the study is for
- Cerebral Adrenoleukodystrophy
- What is being tested?
- MIN-102
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 13 Sep 2019to 31 Dec 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 2 to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD
- White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- Major Functional Disabilities (MFD) score of 0.
- Baseline Loes score >0 and ≤10
- Gadolinium Intensity Score >2
Who cannot join
- Previous HSCT
- Too far progressed inflammatory brain lesions
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Germany2 sites
Universitätsmedizin Göttingen Georg-August-Universität
Göttingen
Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
Hamburg
About this study
Official title: An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
- Conditions
- Cerebral Adrenoleukodystrophy
- Being tested
- MIN-102
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MIN-102Drug
Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.
| Study group | Role | What they receive |
|---|---|---|
| MIN-102 | Experimental | Not described Receives: Drug: MIN-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.
Measured: 6 months- 2 years
"arrested disease" will be assessed at 24 weeks and at 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Measured: at 24 weeks and 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2020
- Study start13 Sep 2019
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral AdrenoleukodystrophyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04528706
- Sponsor's protocol ID
- MT-2-02
- Acronym
- NEXUS
- Status last verified
- Jan 2023
- First submitted
- 31 Jul 2020
- Last updated
- 18 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
- ClinicalTrials.gov ID
- NCT04528706
- Acronym / sponsor ID
- NEXUS / MT-2-02
- Conditions
- Cerebral Adrenoleukodystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 13 participants (estimated)
- Lead sponsor
- Minoryx Therapeutics, S.L.Industry (pharma, biotech, devices)
- Study start
- 13 Sep 2019 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 27 Aug 2020 (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 Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MIN-102 | Experimental | Drug: MIN-102 |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD
- White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- Major Functional Disabilities (MFD) score of 0.
- Baseline Loes score >0 and ≤10
- Gadolinium Intensity Score >2
Key exclusion criteria (2 listed)
- Previous HSCT
- Too far progressed inflammatory brain lesions
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 |
|---|---|---|
| MIN-102Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml. | Drug | MIN-102 |
Primary outcome
evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.
Time frame: 6 months- 2 years
"arrested disease" will be assessed at 24 weeks and at 96 weeks
Time frame: at 24 weeks and 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2023.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MIN-102 in people with Cerebral Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 5 sites in 4 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 5 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: status not confirmed, as of the sponsor's last update on 18 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Germany, Argentina, France, Spain. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects. (time frame: 6 months- 2 years). 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 18 Jul 2023.