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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) (NEXUS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04528706
Recruitment Status : Recruiting
First Posted : August 27, 2020
Last Update Posted : January 10, 2023
Information provided by (Responsible Party):
Minoryx Therapeutics, S.L.

Brief 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)

Condition or disease Intervention/treatment Phase
Cerebral Adrenoleukodystrophy Drug: MIN-102 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : September 13, 2019
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : December 31, 2023

Arm Intervention/treatment
Experimental: MIN-102 Drug: MIN-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.

Primary Outcome Measures :
  1. 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 ]
  2. "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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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:

  • Previous HSCT
  • Too far progressed inflammatory brain lesions

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04528706

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Contact: Arun Mistry +34 93 544 14 66

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Hernan Amartino Recruiting
Bueno Aires, Buenos Aires, Argentina, B16641NZ
Contact: Hernan Amartino         
CHU Kremlin Bicêtre Recruiting
Paris, France
Contact: Dr. Carolin Sevine         
Universitätsmedizin Göttingen Georg-August-Universität Recruiting
Göttingen, Germany
Contact: Dr. Hendrik Rosewich         
Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin Terminated
Hamburg, Germany
Hospital Sant Joan de Déu Recruiting
Barcelona, Spain
Contact: Dr Maria Angeles García Cazorla         
Sponsors and Collaborators
Minoryx Therapeutics, S.L.
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Responsible Party: Minoryx Therapeutics, S.L. Identifier: NCT04528706    
Other Study ID Numbers: MT-2-02
First Posted: August 27, 2020    Key Record Dates
Last Update Posted: January 10, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Disease Progression
Disease Attributes
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases