CLINICAL TRIALS PROFILE FOR ARIMOCLOMOL

Clinical Trials for Arimoclomol

Trial ID	Title	Status	Sponsor	Phase	Summary
NCT00244244 ↗	A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS	Completed	CytRx	Phase 2	The primary purpose of this study is to evaluate the safety and tolerability of arimoclomol in ALS patients following 90 days of dosing. In addition, the amount of arimoclomol in blood and cerebrospinal fluid will be measured.
NCT00561366 ↗	A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS	Withdrawn	CytRx	Phase 2	Arimoclomol is a small molecule that upregulates "molecular chaperones" in cells under stress. Arimoclomol extends survival by five weeks when given both pre-symptomatically and at disease onset in a mutant superoxide dismutase (SOD1) transgenic mouse model of ALS. Furthermore, it has been demonstrated to have neuroprotective and neuroregenerative effects in other rat models of nerve damage. Molecular chaperone proteins are critical in the cellular response to stress and protein misfolding. Recent data suggest that the SOD1 mutation responsible for ALS in some patients with familial disease reduces the availability of a variety of molecular chaperones, and thus weakens their ability to respond to cellular stress. Protein misfolding and consequent aggregation may play a role in the pathogenesis of both the familial and sporadic forms of ALS. Therapeutic agents such as arimoclomol that improve cellular chaperone response to protein misfolding may be helpful in ALS.
NCT00706147 ↗	Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis	Completed	Massachusetts General Hospital	Phase 2/Phase 3	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
NCT00706147 ↗	Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis	Completed	University of Miami	Phase 2/Phase 3	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
NCT00769860 ↗	Arimoclomol in Sporadic Inclusion Body Myositis	Completed	Richard Barohn, MD	Phase 2/Phase 3	Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.
NCT02612129 ↗	Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C	Active, not recruiting	Orphazyme	Phase 2/Phase 3	A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat. The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including patients aged 6 to <24 months at study enrolment.
NCT02753530 ↗	Study of Arimoclomol in Inclusion Body Myositis (IBM)	Completed	University College, London	Phase 2	Funding Source - FDA OOPD. The purpose of this study is to evaluate the safety and efficacy of the study drug, Arimoclomol in IBM patients.

Clinical Trial Conditions for Arimoclomol

Condition Name

Condition Name for
Intervention	Trials
Amyotrophic Lateral Sclerosis	4
Inclusion Body Myositis	3
Acquired Amyotrophic Lateral Sclerosis	1
Amyotrophic Lateral Sclerosis (ALS)	1

Condition MeSH

Condition MeSH for
Intervention	Trials
Sclerosis	5
Motor Neuron Disease	5
Amyotrophic Lateral Sclerosis	5
Myositis, Inclusion Body	3

Clinical Trial Locations for Arimoclomol

Trials by Country

Trials by Country for
Location	Trials
United States	73
Canada	6
United Kingdom	6
India	5
France	3

Trials by US State

Trials by US State for
Location	Trials
New York	6
California	6
Kansas	6
Virginia	5
Texas	5

Clinical Trial Progress for Arimoclomol

Clinical Trial Phase

Clinical Trial Phase for
Clinical Trial Phase	Trials
Phase 3	3
Phase 2/Phase 3	3
Phase 2	4

Clinical Trial Status

Clinical Trial Status for
Clinical Trial Phase	Trials
Completed	5
Terminated	1
Active, not recruiting	1

Clinical Trial Sponsors for Arimoclomol

Sponsor Name

Sponsor Name for
Sponsor	Trials
Orphazyme	6
University of Kansas Medical Center	3
University College, London	2

Sponsor Type

Sponsor Type for
Sponsor	Trials
Industry	8
Other	8