A Clinical Study in Male Pediatric Patients With Cerebral X-linked

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

Cerebral Adrenoleukodystrophy Clinical Trial

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)

See also
Status	Clinical Trial	Phase
Recruiting	`NCT03789721` - Adrenoleukodystrophy National Registry Study
Withdrawn	`NCT02410239` - MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)	Phase 1
Terminated	`NCT03367546` - Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases	Phase 2
Recruiting	`NCT06030648` - MT2022-01: MSCs for ALD	Phase 1

See also

Status

Clinical Trial

Phase

Recruiting

NCT03789721 - Adrenoleukodystrophy National Registry Study

Withdrawn

NCT02410239 - MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)

Phase 1

Terminated

NCT03367546 - Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

Phase 2

Recruiting

NCT06030648 - MT2022-01: MSCs for ALD

Phase 1

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT04528706
Study type	Interventional
Source	Minoryx Therapeutics, S.L.
Contact
Status	Active, not recruiting
Phase	Phase 2
Start date	September 13, 2019
Completion date	December 31, 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) — NEXUS

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms