The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS

• ClinicalTrials.gov Identifier: NCT04391361
• Recruitment Status: Not yet recruiting
• First Posted: May 18, 2020
• Last Update Posted: October 14, 2020
• Sponsor: Ruijin Hospital
• Information provided by (Responsible Party): Ruijin Hospital

Tracking Information

• First Submitted Date: May 13, 2020
• First Posted Date: May 18, 2020
• Last Update Posted Date: October 14, 2020
• Estimated Study Start Date: November 1, 2020
• Estimated Primary Completion Date: September 30, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 9, 2020)

• Effective [ Time Frame: 48 weeks ]
  Forced expiratory volume in one second (FVC%) or ALS functional rating scale score (ALSFR, minimum score: 0, maximum score: 48, higher score is related to a better outcome) was elevated.
• Ineffective [ Time Frame: 48 weeks ]
  Forced expiratory volume in one second (FVC%) or ALS functional rating scale score (ALSFR, minimum score: 0, maximum score: 48, higher score is related to a better outcome) was decreased or remained unchanged.

Original Primary Outcome Measures (submitted: May 13, 2020)

• Effective [ Time Frame: 24 weeks ]
  ALS-FR score was elevated.
• Ineffective [ Time Frame: 24 weeks ]
  ALS-FR score was decreased or unchanged.

Current Secondary Outcome Measures (submitted: October 9, 2020)

• Effective [ Time Frame: 48 weeks ]
  Partial pressure of oxygen (PO2), maximum displacement of the hyoid or the modified Norris scale score (minimum score: 0, maximum score: 100, higher score is related to a better outcome) was elevated or partial pressure of carbon dioxide (PCO2) was decreased or remained unchanged.
• Ineffective [ Time Frame: 48 weeks ]
  Partial pressure of carbon dioxide (PCO2) was elevated, or partial pressure of oxygen (PO2), maximum displacement of the hyoid or the modified Norris scale score (minimum score: 0, maximum score: 100, higher score is related to a better outcome) was decreased or remained unchanged.

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS
• Official Title: The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of Amyotrophic Lateral Sclerosis
• Brief Summary: Thirty cases of amyotrophic lateral sclerosis patients were recruited from the neurology department of Ruijin Hospital, the pain department and the encephalopathy center of Luwan Branch of Ruijin Hospital. After the informed consent was signed, they were divided into a trial group and a control group. Each group contains 15 cases. The patients in the control group was treated with edaravone dissolved in saline during hospitalization, while the patients in the trial group was treated with edaravone, scopolamine, atropine and dexmedetomidine. Both groups of subjects were treated for 7 days within 3 weeks, followed by a buffer period of 3 weeks for observation, which was one treatment course. The total treatment protocol contains 3 treatment courses (or 18 weeks). Patients with amyotrophic lateral sclerosis were evaluated before treatment and 6, 12, 18, 24, 36, 48 weeks after treatment. The observations include whether the functional scores of patients with amyotrophic lateral sclerosis, Norris amyotrophic lateral sclerosis score, amyotrophic lateral sclerosis self-score, forced expiratory volume in one second, partial pressure of oxygen and maximum displacement of the hyoid were superior to those before treatment, and whether the partial pressure of carbon dioxide was inferior to those before treatment. Study hypothesis: Cholinergic receptor blocking therapy for amyotrophic lateral sclerosis is safe and effective in improving motor function and delaying disease progression in patients with amyotrophic lateral sclerosis.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Participant); Primary Purpose: Treatment

Condition

• Amyotrophic Lateral Sclerosis
• Respiratory Function
• Scopolamine

Intervention

• Drug: Scopolamine, atropine, edaravone and dexmedetomidine
  The patients were treated with scopolamine, atropine, edaravone and dexmedetomidine dissolved in saline.
• Drug: Edaravone
  The patients was treated with edaravone dissolved in the same volume of saline as the intervention applied in the experimental group.

Study Arms

• Experimental: trial group
  Intervention: Drug: Scopolamine, atropine, edaravone and dexmedetomidine
• Placebo Comparator: control group
  Intervention: Drug: Edaravone

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: May 13, 2020): 30
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: October 31, 2023
• Estimated Primary Completion Date: September 30, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Patients should voluntarily participate in the experiment by signing an informed consent form.
2. Patients should comply with the research process and cooperate with the interventions applied throughout the experiment.
3. Patients should be between 30 and 65 years of age, and both sexes are acceptable.
4. Patients should comply with the diagnostic criteria and exclusion criteria for amyotrophic lateral sclerosis in the Chinese Guidelines for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis developed by the Chinese Society of Neurology of the Chinese Medical Association in 2012.
5. Patient's amyotrophic lateral sclerosis history must not exceed 5 years.
6. ALS functional scale score (ALSFRS-R) should be ≥ 2 points for each patients.

Exclusion Criteria:

1. Patients with cardiac conduction block, severe ventricular insufficiency, severe hepatorenal insufficiency and severe, progressive or uncontrolled major organ and systemic disease.
2. High or low blood pressure: systolic blood pressure >150 or <110 mmHg; bradycardia (<60 beats per minute).
3. Patients with ventilator-assisted ventilation.
4. Patients who are allergic to research intervention drugs.
5. Patients with obvious signs of dementia.
6. Female patients who are pregnant or breastfeeding or who have a plan to become pregnant in the near future.
7. Persons with other psychiatric disorders that may affect the assessment of their condition.
8. Severely obese patients (BMI >35kg/m2).

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 30 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Jun Liu, Professor; 64370045 ext +86-021; jly0520@hotmail.com

• Listed Location Countries: China

Administrative Information

• NCT Number: NCT04391361
• Other Study ID Numbers: lj11128
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No
• Plan Description: No individual participant data (IPD) was available to other researchers.

• Current Responsible Party: Ruijin Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: Ruijin Hospital
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Chair: Jun Liu, Professor; Department of Neurology, Rui Jin Hospital, Shanghai Jiao Tong University School of Medicine

• PRS Account: Ruijin Hospital
• Verification Date: October 2020