Nerve Growth Factor Encapsulated With 2-methacryloyloxyethyl Phosphorylcholine Nanocapsules in the Treatment of Amyotrophic Lateral Sclerosis

NCT06391645 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: HX-A-2023027
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

Amyotrophic lateral sclerosis (ALS) is one of the most lethal neurodegenerative diseases, with most patients dying from respiratory failure 3-5 years after the onset. The purpose of this study is to explore the efficacy and safety of nerve growth factor (NGF) encapsulated with 2-methacryloyloxyethyl phosphorylcholine (MPC) nanocapsules in the treatment of ALS patients.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

nerve growth factor; amyotrophic lateral sclerosis; 2-methacryloyloxyethyl phosphorylcholine

Outcome Measures

Primary Outcomes (1)

• Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

  Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score (ALSFRS-R scores from 0 to 48, score decline indicates worse outcome of ALS patients, or disease progression and disability).

  84±7 days

Secondary Outcomes (7)

• Endpoint events

  28±4 days, 84±7 days, 120±7 days, 180±14 days, 1 year ±14 days

• Tolerance

  28±4 days, 84±7 days

• Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

  28±4 days, 120±7 days, 180±14 days, 1 year ±14 days

• Lung function: forced vital capacity

  28±4 days, 84±7 days, 120±7 days

• Lung function: slow vital capacity

  28±4 days, 84±7 days, 120±7 days

Other Outcomes (18)

• Medullary function

  28±4 days, 84±7 days, 120±7 days

• Cognitive function

  120±7 days

• Wexner continence grading scale

  84±7 days, 120±7 days

Study Arms (3)

MPC-NGF T1

EXPERIMENTAL

19.2mg MPC wrapped NGF per week for 13 times

Drug: Nerve Growth Factor

MPC-NGF T2

EXPERIMENTAL

11.84mg MPC wrapped NGF per week for 13 times

Drug: Nerve Growth Factor

X-NGF

ACTIVE COMPARATOR

19.2mg NGF per week for 13 times

Drug: Nerve Growth Factor

Interventions

Nerve Growth Factor DRUG

intravenously injection

MPC-NGF T1; MPC-NGF T2; X-NGF

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years old;
• Confirmed or possible familial or sporadic ALS diagnosed according to the revised El Escorial criteria;
• months ≤disease duration ≤ 3 years, (onset time is defined as the time of the first occurrence of myasthenia);
• Forced vital capacity (FVC) ≥ 85% of predicted value (based on gender, height and age);
• Informed consent signed.

You may not qualify if:

• Patients undergoing endotracheal intubation, non-invasive or mechanical ventilation;
• Patients with diaphragmatic pacemakers;
• Allergy to any component of the investigational medication, or any other allergic history that researchers deem necessary to be vigilant about;
• Local skin infection or other suspicious signs of infection at the injection site;
• Known hemorrhagic tendency (including but not limited to: platelet count \<100×109/ L; on therapy of heparin, activated partial thromboplastin time (APTT) ≥35s; on therapy of warfarin, international normalized ratio (INR) \>1.7; on therapy of novel oral anticoagulants; with direct thrombin or factor Xa inhibitor; accompanied with coagulopathy such as hemophilia);
• Severe cardiac insufficiency before randomization (comply with New York College of Cardiology (NYHA) Cardiac Function Class III, IV);
• Suffering from infectious diseases: hepatitis, tuberculosis, acquired immunodeficiency syndrome, etc;
• Psychiatric disorders diagnosed according to Diagnostic and Statistical Manual of Mental Disorders (DSM-V) diagnostic criteria; or with suicidal intentions;
• Women/men with desire to conceive during the experiment, and patients with pregnancy and lactation;
• Difficulty in verbal communication, inability to communicate, understand or follow instructions, inability to cooperate with treatment and evaluation;
• Combining with history of alcohol and drug abuse;
• Unable to cooperate in follow-up due to geographical or other reasons;
• Patients participated in other clinical trials or used other biologics, drugs, or devices under study.

Sponsors & Collaborators

• Beijing Tiantan Hospital: lead
• Beijing University of Chemical Technology: collaborator
• Beijing Healthunion Cardio-Cerebrovascular Disease Prevention and Treatment Foundation: collaborator

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Hereditary Sensory and Autonomic Neuropathies

Interventions

Nerve Growth Factor

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Polyneuropathies; Peripheral Nervous System Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Nerve Growth Factors; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Nerve Tissue Proteins; Biological Factors

Central Study Contacts

Xinru Liu

CONTACT

13521588395; liuxinru0826@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Executive Vice-President

Study Record Dates

• First Submitted: March 8, 2024
• First Posted: April 30, 2024
• Study Start: May 1, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 30, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share