Status unknownNCT04528706NEXUSUpdated Jul 18, 2023

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 interventional study of MIN-102 in Cerebral Adrenoleukodystrophy, sponsored by Minoryx Therapeutics, S.L.. Status unknown at 5 sites in 4 countries. Open to male participants aged 2 Years to 12 Years. Per ClinicalTrials.gov, last updated 2023-07-18.

Sponsored by Minoryx Therapeutics, S.L. · Phase 2, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2023), so the status shown — last known as Active, not recruiting — may be out of date.

From the registry’s dates

Registered 10 months after the study started (first participant enrolled Sep 2019, registered Jul 2020).

Phase: Phase 2
Study type: Interventional
Enrollment: 13
Allocation: Not applicable
Ages: 2 Years to 12 Years
Sex: Male

Study 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)

Conditions studied

Cerebral Adrenoleukodystrophy

Browse trials for

Adrenoleukodystrophy 68

In context

Adrenoleukodystrophy

68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.

This study's planned enrollment of 13 is below the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.

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Lead sponsor

Minoryx Therapeutics, S.L. is the lead sponsor of 5 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 2 Years to 12 Years
Sexes eligible: Male
Accepts healthy volunteers: No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

Previous
Too far progressed inflammatory brain lesions

Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Allogeneic and autologous transplant: Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 13 participants (estimated)

Study arms

Experimental
MIN-102
Drug: MIN-102

Interventions

DrugMIN-102
Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.

What researchers measure

Primary outcomes

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
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Time frame: at 24 weeks and 96 weeks

Study locations

5 sites

Hernan Amartino
Bueno Aires, Buenos Aires B16641NZ, Argentina
CHU Kremlin Bicêtre
Paris, France
Universitätsmedizin Göttingen Georg-August-Universität
Göttingen, Germany
Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
Hamburg, Germany
Hospital Sant Joan de Déu
Barcelona, Spain

Timeline

Tracking since Sep 25, 2026

ElapsedEstimatedEstimated, not yet reached

Started Sep 2019 (actual). Primary completion is estimated for Dec 2023, 51 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered: Jul 31, 2020
First posted: Aug 27, 2020
StartActual: Sep 13, 2019
Primary completionEst.: Dec 31, 2023
CompletionEst.: Dec 31, 2023
Last update: Jul 18, 2023

What these dates mean

Start.: When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.: When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.: When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.: Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.

No changes since tracking began. The registry record was last updated on Jul 18, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT04528706
Lead sponsor: Minoryx Therapeutics, S.L.
Responsible party: Sponsor
First posted: Aug 27, 2020
Start date: Sep 13, 2019
Primary completion: Dec 31, 2023 (estimated)
Completion: Dec 31, 2023 (estimated)
Last update: Jul 18, 2023

Oversight

Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2023. You cannot join it, but the record below documents what was studied.

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Discussion

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