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AdrenomyeloneuropathyPhase 2NCT01495260

A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

A Phase 2 study testing N-acetylcysteine, lipoic acid and vitamin E in people with Adrenomyeloneuropathy, run by Pujol, Aurora, M.D. at 1 site in Spain.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

X-linked adrenoleukodystrophy is a rare, demyelinating and neurodegenerative disorder, due to a loss of function of a fatty acid transporter, the peroxisomal ABCD1protein. Its more frequent phenotype, the adrenomyeloneuropathy in adults, is characterized by axonal degeneration in spinal cord, spastic paraparesis and a… Read more about this study

Who is it for?
Ages 18 to 64 years
Conditions the study is for
Adrenomyeloneuropathy
What is being tested?
N-acetylcysteine, lipoic acid and vitamin E
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteSpain
Study dates
Sep 2011to Nov 2013
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 18 to 64 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Symptomatic AMN patients,
  • 18-64 years old,
  • male and female,
  • clinically and biochemically diagnosed;
  • females must be obligated heterozygotes or must have gene mutation identified.

Who cannot join

  • Pregnant and lactation in females,
  • Cerebral inflammatory disease with cognitive disorder, and/or
  • need the help of two walking sticks,
  • epilepsy,
  • hypersensibility to cysteine related compounds,
  • transaminases 2 fold up normal values.

Trial locations and contacts

1 site in 1 country.

Spain1 site

  • Hospital Universitari de Bellvitge

    L'Hospitalet de Llobregat, Barcelona, 08907

Other trials in Spain

About this study

Official title: A Clinical Trial for Adrenomyeloneuropathy (AMN): Validation of Biomarkers of Oxidative Stress, and Efficacy, Tolerance and Safety of a Mixture of the Antioxidants N-acetylcysteine, Lipoic Acid and Vitamin E

X-linked adrenoleukodystrophy is a rare, demyelinating and neurodegenerative disorder, due to a loss of function of a fatty acid transporter, the peroxisomal ABCD1protein. Its more frequent phenotype, the adrenomyeloneuropathy in adults, is characterized by axonal degeneration in spinal cord, spastic paraparesis and a disabling peripheral neuropathy. Actually, there is no efficient treatment for the disease. Our work in the last twelve years dissecting the physiopathological basis of the disorder has uncovered an involvement of the oxidative stress early in the neurodegenerative cascade. In a preclinical trial we have identified an antioxidant cocktail that efficiently reverse the clinical symptoms and the axonal degeneration in the mouse model for the disease. We propose the translation of the results to an open trial to test the tolerance and effectiveness of these drugs in the correction of the previously identified oxidative lesion biomarkers, as a first step to a randomized versus placebo, multicentric and international trial. You will be clinically explored and assessed in the Hospital Universitari of Bellvitge (HUB) using clinical scales for spasticity, disability, electroneurogram and cranial and spinal Nuclear Magnetic resonance (NMR). The information will be collected in a data base that will be of great value to improve the present attention and the future follow-up to facilitate your inclusion in therapeutic randomized, double blind, against placebo clinical trials.

Being tested
N-acetylcysteine, lipoic acid, vitamin E
Keywords
XALD, AMN
Medical subject headings
Adrenoleukodystrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • N-acetylcysteineDrug

    N-acetylcysteine, 800-2400 mg daily for 2 months

  • lipoic acidDrug

    lipoic acid, 300-600 mg daily for 2 months

  • vitamin EDrug

    vitamin E, 150-300 mg daily for 2 months

Study groups in NCT01495260
Study groupRoleWhat they receive
N-acetylcysteine, lipoic acid and vitamin EExperimentalTwo Dose titration design
Receives: Drug: N-acetylcysteine; Drug: lipoic acid; Drug: vitamin E

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. oxidative lesion biomarkers

    oxidative lesion biomarkers: protein, DNA and peroxidation biomarkers

    Measured: 12 months

Secondary outcomes (1)

  1. clinical parameters

    spasticity, disability,electroneurograms and evocated potentials. Cranial and spinal NMR will be done at the beginning and the end of the trial.

    Measured: 2, 6 and 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Dec 2011 (estimated)
  2. Study startSep 2011
  3. Primary completionNov 2013
  4. Study completionNov 2013

Resources

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 (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01495260
Sponsor's protocol ID
XAMNANTIOXAP2010
Status last verified
Sep 2016
First submitted
28 Nov 2011
Last updated
11 Mar 2019
Results posted
No
Data monitoring committee
No

Publications

  • Lopez-Erauskin J, Fourcade S, Galino J, Ruiz M, Schluter A, Naudi A, Jove M, Portero-Otin M, Pamplona R, Ferrer I, Pujol A. Antioxidants halt axonal degeneration in a mouse model of X-adrenoleukodystrophy. Ann Neurol. 2011 Jul;70(1):84-92. doi: 10.1002/ana.22363.

    PubMed 21786300

  • Galino J, Ruiz M, Fourcade S, Schluter A, Lopez-Erauskin J, Guilera C, Jove M, Naudi A, Garcia-Arumi E, Andreu AL, Starkov AA, Pamplona R, Ferrer I, Portero-Otin M, Pujol A. Oxidative damage compromises energy metabolism in the axonal degeneration mouse model of X-adrenoleukodystrophy. Antioxid Redox Signal. 2011 Oct 15;15(8):2095-107. doi: 10.1089/ars.2010.3877. Epub 2011 Jun 8.

    PubMed 21453200

  • Parameswaran J, Goicoechea L, Planas-Serra L, Pastor A, Ruiz M, Calingasan NY, Guilera C, Aso E, Boada J, Pamplona R, Portero-Otin M, de la Torre R, Ferrer I, Casasnovas C, Pujol A, Fourcade S. Activating cannabinoid receptor 2 preserves axonal health through GSK-3beta/NRF2 axis in adrenoleukodystrophy. Acta Neuropathol. 2022 Aug;144(2):241-258. doi: 10.1007/s00401-022-02451-2. Epub 2022 Jul 1.

    PubMed 35778568

  • Casasnovas C, Ruiz M, Schluter A, Naudi A, Fourcade S, Veciana M, Castaner S, Alberti A, Bargallo N, Johnson M, Raymond GV, Fatemi A, Moser AB, Villarroya F, Portero-Otin M, Artuch R, Pamplona R, Pujol A. Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot Study. Neurotherapeutics. 2019 Oct;16(4):1167-1182. doi: 10.1007/s13311-019-00735-2.

    PubMed 31077039

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing N-acetylcysteine, lipoic acid and vitamin E in people with Adrenomyeloneuropathy, run by Pujol, Aurora, M.D. at 1 site in Spain.

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 6 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: finished, as of the sponsor's last update on 11 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

oxidative lesion biomarkers (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01495260, which also lists 4 linked publications.

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 11 Mar 2019.

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