A multicenter, uncontrolled, open-label study to investigate the safety and the efficacy of human (allogeneic) amnion-derived mesenchymal stem cells KA-301 for Duchenne muscular dystrophy

JRCT ID: jRCT2033230295

Registered date:18/08/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Duchenne muscular dystrophy
• Date of first enrollment: 01/09/2023
• Target sample size: 18
• Study type: Interventional
• Intervention(s): KA-301 is administered intravenously at a dose of 1.0 x 10^6 cells/kg or 2.0 x 10^6 cells/kg once every 2 weeks for a total of 6 doses.

Outcome(s)

Primary Outcome

Safety endpoints
(1) Physical findings
(2) Height and weight
(3) Vital signs
(4) Electrocardiogram
(5) Echocardiography
(6) Clinical examination
(7) Immunological examination
(8) Arterial oxygen saturation (SpO2)
(9) Pulmonary function test
(10) Chest x-ray
(11) Abdominal ultrasonography
(12) Adverse events

Secondary Outcome

(1) Motor function assessment: Timed Up & Go Test, quantitative muscle strength measurement, North star ambulatory assessment
(2) Changes in biomarkers: serum myostatin, serum interleukin-6, serum TNF-alfa, creatine kinase

Key inclusion & exclusion criteria

• Age minimum: >= 5age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(1) Patients diagnosed with Duchenne muscular dystrophy based on medical history and genetic diagnosis
(2) Patients who can walk independently without using walking aids
(3) Patients who can complete getting up from the floor in less than 10 seconds
(4) Patients who have been receiving corticosteroids for the treatment of the primary disease at a certain dose for at least 3 months prior to the start of administration of the investigational product and are expected to receive the same dose throughout the study period
(5) Patients aged at least 5 years old at the time of consent
(6) Patients for whom written consent is obtained from a legally acceptable representative (guardian) (Whenever possible, written consent is also obtained from the patient)

Exclude criteria

(1) Patients who cannot assess motor function due to cognitive impairment or behavioral problems
(2) Patients with injuries that may affect the assessment of motor function, except for patients whose previous lower extremity fractures have completely healed and at least 3 months have passed from the date of injury
(3) Patients with complication or history of chronic systemic fungal or viral infection
(4) Patients with acute illness within 4 weeks prior to the first administration of the investigational product
(5) Patients with other musculoskeletal or neurological disorders, physical disabilities, or medical history not related to Duchenne muscular dystrophy, including pulmonary and cardiac disease
(6) Patients with a history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, or allergic disease
(7) Patients who have participated in another clinical trial within 30 days or 5 times the half-life of the other investigational drug or product
(8) Patients treated with androgen or human growth hormone within the past 3 months
(9) Patients with drug hypersensitivity or allergic predisposition
(10) Patients with hypersensitivity to corticosteroids or antihistamines used concomitantly with the investigational product
(11) Patients who have received exon-skipping therapy within 3 months prior to the first dose of the investigational product
(12) Patients who have received gene therapy
(13) Patients who have undergone surgery within 3 months prior to administration of the investigational product or are scheduled to undergo surgery during the study period
(14) Patients who have used other cell products within the past 3 months
(15) Patients with hypersensitivity to the products derived from pigs or humans
(16) Patients with positive results for hepatitis B surface (HBs) antigen, hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) antibody tests at screening
(17) Patients considered ineligible by the investigator or sub investigators

Related Information

• Primary Sponsor: Nakaishi Tomoyuki