Clinical trial NCT03103815: Trial of Amivita in Amyotrophic Lateral Sclerosis
Official title: Trial of Amivita in Amyotrophic Lateral Sclerosis: a Single-center, Single-blind, Self-controlled Clinical Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic lateral sclerosis, survival, adverse event
Lead sponsor: Wujin People's Hospital
Sponsor class: OTHER
Collaborators: name: Nanjing 1718
First posted: 2017-04-06
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is a self-controlled trial. All participants will receive treatment.
Primary purpose: TREATMENT

Brief summary: The primary objectives of this study are to determine the safety and efficacy of Amivita, a compound of amino acids and vitamines in patients with Amyotrophic lateral sclerosis (ALS)ALS. The secondary objectives are to measure quality of life before and during intervention. This is a self-controlled clinical trial. Twenty patients in our ALS center who are already receiving riluzole or other treatments but the condition is worsening will receive treatment for 1o months. The evaluating investigators will be blinded to treatment assignment. Primary outcome measures will be adverse events, the ALS Functional Rating Scale-Revised (ALSFRS-R), and survival. Subjects will also be assessed at enrollment and at study end for weight loss, forced vital capacity (FVC), quality of life and grip strength.

Detailed description: Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder affecting upper and lower motor neurons. Survival is typically 2 to 5 years from symptom onset; death is usually from respiratory paralysis. Standard therapy is with Riluzole 100 mg/day, a FDA approved treatment for ALS that has a small effect on survival. There is a strong need for more effective therapies in ALS.

In our previous studies, we have shown that Amivita, a compound of amino acids and vitamines, is effective for neuronal injury (unpublished data). We have since then use this regimen to treat ALS patients. Our retrospective analysis (unpublished data) of the treated patient indicates that this regimen can slow down the progression of ALS.

We proposed a self-controlled clinical trial to study the safety and efficacy of Amivita. Secondary outcome measures include weight and quality of life. Twenty subjects in our ALS center who are already receiving riluzole will receive treatment for 12 months. The evaluating investigators will be blinded to treatment assignment. Primary outcome measures will be adverse events, the ALS Functional Rating Scale-Revised (ALSFRS-R), and survival. Secondary outcome measures include body weight, forced vital capacity (FVC), quality of life and grip strength.

The total study length from first enrolled subject will be approximately 6 months.

Participants in this study will be subjects with familial or sporadic ALS diagnosed as probable, or definite, according to the World Federation of Neurology El Escorial criteria. Diagnostic and Inclusionary/Exclusionary criteria will be clearly outlined in the protocol.

Eligibility criteria: Inclusion Criteria

* Patients must be men or women between the ages of 18 and 70 years
* Patient is clinical definite or probable ALS by the hospitals listed in the protocol
* Women who are of child bearing potential must have a negative pregnancy test
* Willing to comply with the study visits
* Will not take riluzole during the study period
* Be able to sign informed consent document

Exclusion Criteria

* Myotonic dystrophy
* Myasthenia gravis
* Post-poliomyelitis syndrome
* Multifocal motor neuropathy with or without conduction block
* Hirayama disease
* Kennedy disease
* Hereditary spastic paraplegia
* Syringomyelia
* Spinal cord and brain stem tumors
* Paraneoplastic syndromes
* Severe liver or kidney disease disease
* Infection, severe diarrhea or vomiting
* Serious heart or lung diseases or malignant tumor history
* HIV infection
* Pregnancy or breastfeeding
* Have no ability to communicate
* Have participated in other clinical trials within 4 weeks
* Any form of substance abuse, psychiatric disorder, or other condition that, in opinion of the investigator, may interfere with the study

Primary outcomes:
measure: ALS Functional Rating Scale-Revised (ALSFRS-R) score
timeFrame: 10 months
measure: Adverse event
timeFrame: 10 months
Secondary outcomes:
measure: Forced vital capacity (FVC)
timeFrame: 10 months
measure: EQ-5D
timeFrame: 10 months
Other outcomes:
measure: Grisp strength
timeFrame: 10 months
measure: body weigh
timeFrame: 10 months
Locations:
city: Changzhou
country: China
facility: Wujing People's Hospital
state: Jiangsu
Study officials:
affiliation: Wujing People's Hospital
name: Maoxin Yue, M.D
role: PRINCIPAL_INVESTIGATOR
References: