NCT00706147

Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis (ALS)

Completed Phase 2/Phase 3 Last updated 9 January 2019

What this trial tests

Phase 2/Phase 3 trial testing Arimoclomol in Amyotrophic Lateral Sclerosis in 38 participants. Completed in 1 November 2016.

Timeline

1 January 2009

Primary endpoint
1 December 2014

1 November 2016

Quick facts

Lead sponsor	University of Miami
Phase	Phase 2/Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	38
Start date	1 January 2009
Primary completion	1 December 2014
Estimated completion	1 November 2016
Sites	2 locations across United States

Drugs / interventions tested

• Arimoclomol (ARIMOCLOMOL) — full drug profile →
• Placebo

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →

Sponsor

University of Miami

Who can join

18 and older, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Time to death, tracheostomy or permanent assisted ventilation will be the primary outcome measure.
  Time frame: 12 months

Sponsor's own description

The purpose of this study will be to demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with SOD1 positive familial Amyotrophic Lateral Sclerosis (ALS). This type of ALS is HEREDITARY (runs in families), and at least one other person in the family must have had ALS.

Study hypotheses: Arimoclomol, taken at a dose of 200 mg three times daily will improve survival as defined by time to death, tracheostomy or permanent assisted ventilation. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS.

Funding Source - FDA-OOPD

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Membrane Lipid Composition: Effect on Membrane and Organelle Structure, Function and Compartmentalization and Therapeutic Avenues.
   Casares D, Escribá PV, Rosselló CA. · · 2019 · cited 532× · PMID 31052427 · DOI 10.3390/ijms20092167
2. Heat shock transcription factor 1 as a therapeutic target in neurodegenerative diseases.
   Neef DW, Jaeger AM, Thiele DJ. · · 2011 · cited 238× · PMID 22129991 · DOI 10.1038/nrd3453
3. Improving clinical trial outcomes in amyotrophic lateral sclerosis.
   Kiernan MC, Vucic S, Talbot K, McDermott CJ, et al · · 2021 · cited 205× · PMID 33340024 · DOI 10.1038/s41582-020-00434-z
4. Superoxide Dismutase 1 in Health and Disease: How a Frontline Antioxidant Becomes Neurotoxic.
   Trist BG, Hilton JB, Hare DJ, Crouch PJ, et al · · 2021 · cited 148× · PMID 32144830 · DOI 10.1002/anie.202000451
5. Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
   Kirkegaard T, Gray J, Priestman DA, Wallom KL, et al · · 2016 · cited 134× · PMID 27605553 · DOI 10.1126/scitranslmed.aad9823
6. Small Heat Shock Proteins, Big Impact on Protein Aggregation in Neurodegenerative Disease.
   Webster JM, Darling AL, Uversky VN, Blair LJ. · · 2019 · cited 115× · PMID 31619995 · DOI 10.3389/fphar.2019.01047
7. Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive <i>SOD1</i> ALS.
   Benatar M, Wuu J, Andersen PM, Atassi N, et al · · 2018 · cited 104× · PMID 29367439 · DOI 10.1212/wnl.0000000000004960
8. Targeting protein homeostasis in sporadic inclusion body myositis.
   Ahmed M, Machado PM, Miller A, Spicer C, et al · · 2016 · cited 96× · PMID 27009270 · DOI 10.1126/scitranslmed.aad4583