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| ID | Type | Description | Link |
|---|---|---|---|
| 153072 | Registry Identifier | JAPIC CTI |
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| Name | Class |
|---|---|
| Daiichi Sankyo | INDUSTRY |
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This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DS-5141b | Experimental | DS-5141b, Subcutaneous injection Part 1: DS-5141b will be injected subcutaneously once a week for 2 weeks at the following dose levels. Dose escalation will be performed. DS-5141b will be administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2.
Part 2: Two doses of DS-5141b will be selected based on the results obtained in Part 1. Each selected dose will be administered subcutaneously once a week for 12 weeks. Part 2-Extension-2: Two doses, 2.0 mg/kg or 6.0 mg/kg, of DS-5141b will be administered subcutaneously once a week for 48 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DS-5141b | Drug | DS-5141b, Subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy | A treatment-emergent adverse event (TEAE) is defined as an adverse event that emerges during treatment having been absent prior to treatment or reemerges during treatment or worsens in severity during treatment. | 48 Weeks of Part 2-Extension-2 |
| Pharmacokinetic Parameter Maximum Concentration (Cmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Week 48 of Part 2-Extension-2 |
| Pharmacokinetic Parameter Area Under the Curve (AUC) Tau of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Week 48 of Part 2-Extension-2 |
| Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Week 48 of Part 2-Extension-2 |
| Pharmacokinetic Parameter Half-life (T1/2) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Week 48 of Part 2-Extension-2 |
| Mean Dystrophin Protein Expression in Muscle Tissue | Week 48 of Part 2-Extension-2 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Exon 45-skipped Dystrophin mRNA Expression in Muscle Tissue Posttreatment | Week 48 of Part 2-Extension-2 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Global Clinical Leader | Daiichi Sankyo | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kobe University Hospital | Hyōgo | Kobe-shi | 650-0017 | Japan | ||
| National Center of Neurology and Psychiatry |
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A total of 12 participants were screened; 8 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study at 2 clinic centers in Japan.
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| ID | Title | Description |
|---|---|---|
| FG000 | DS-5141b 2.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 2.0 mg/kg once a week. |
| FG001 | DS-5141b 6.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 6.0 mg/kg once a week. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Demographic and baseline characteristics were assessed in the Safety Analysis Set.
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| ID | Title | Description |
|---|---|---|
| BG000 | DS-5141b 2.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 2.0 mg/kg once a week. |
| BG001 | DS-5141b 6.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 6.0 mg/kg once a week. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy | A treatment-emergent adverse event (TEAE) is defined as an adverse event that emerges during treatment having been absent prior to treatment or reemerges during treatment or worsens in severity during treatment. | Safety events were assessed in the Safety Analysis Set. | Posted | Count of Participants | Participants | 48 Weeks of Part 2-Extension-2 |
|
Adverse events were collected from baseline up to 48 weeks post extension period.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DS-5141b 2.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 2.0 mg/kg once a week. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Contact for Clinical Trial Information | Daiichi Sankyo | 908-992-6400 | CTRinfo@dsi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 11, 2020 | Jul 28, 2023 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| Tokyo |
| Kodaira-shi |
| 187-8551 |
| Japan |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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Participants who received a subcutaneous injection of DS-5141b 6.0 mg/kg once a week.
|
|
| Primary | Pharmacokinetic Parameter Maximum Concentration (Cmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set. | Posted | Mean | Standard Deviation | ng/mL | Week 48 of Part 2-Extension-2 |
|
|
|
| Primary | Pharmacokinetic Parameter Area Under the Curve (AUC) Tau of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Pharmacokinetic parameters were assessed in participants with available data in the Pharmacokinetic Analysis Set. | Posted | Mean | Standard Deviation | ng*h/mL | Week 48 of Part 2-Extension-2 |
|
|
|
| Primary | Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set. | Posted | Median | Full Range | hours | Week 48 of Part 2-Extension-2 |
|
|
|
| Primary | Pharmacokinetic Parameter Half-life (T1/2) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Dystrophy | Pharmacokinetic parameters were assessed using non-compartmental methods. | Pharmacokinetic parameters were assessed in participants with available data in the Pharmacokinetic Analysis Set. | Posted | Mean | Standard Deviation | hours | Week 48 of Part 2-Extension-2 |
|
|
|
| Primary | Mean Dystrophin Protein Expression in Muscle Tissue | Dystrophin protein expression was assessed in the Efficacy Analysis Set. | Posted | Mean | Standard Deviation | percent of normal protein expression | Week 48 of Part 2-Extension-2 |
|
|
|
| Secondary | Number of Participants With Exon 45-skipped Dystrophin mRNA Expression in Muscle Tissue Posttreatment | Dystrophin mRNA expression was assessed in the Efficacy Analysis Set. | Posted | Count of Participants | Participants | Week 48 of Part 2-Extension-2 |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 3 |
| 3 |
| EG001 | DS-5141b 6.0 mg/kg | All participants who received a subcutaneous injection of DS-5141b 6.0 mg/kg once a week. | 0 | 5 | 1 | 5 | 5 | 5 |
| Chronic sinusitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Molluscum contagiosum | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Enterocolitis viral | Infections and infestations | MedDRA 23.1 | Systematic Assessment |
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| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.1 | Systematic Assessment |
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| Enuresis | Psychiatric disorders | MedDRA 23.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypoaesthesia | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Tension headache | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Intercostal neuralgia | Nervous system disorders | MedDRA 23.1 | Systematic Assessment |
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| Chalazion | Eye disorders | MedDRA 23.1 | Systematic Assessment |
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| Conjunctivitis allergic | Eye disorders | MedDRA 23.1 | Systematic Assessment |
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| Hypermetropia | Eye disorders | MedDRA 23.1 | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | MedDRA 23.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Anal fissure | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Stomatitis | Gastrointestinal disorders | MedDRA 23.1 | Systematic Assessment |
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| Eczema asteatotic | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Hyperkeratosis | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Nail disorder | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Papule | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Growing pains | Musculoskeletal and connective tissue disorders | MedDRA 23.1 | Systematic Assessment |
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| Balanoposthitis | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
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| Spontaneous penile erection | Reproductive system and breast disorders | MedDRA 23.1 | Systematic Assessment |
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| Injection site bruising | General disorders | MedDRA 23.1 | Systematic Assessment |
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| Injection site reaction | General disorders | MedDRA 23.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 23.1 | Systematic Assessment |
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| Injection site discolouration | General disorders | MedDRA 23.1 | Systematic Assessment |
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| Activated partial thrombroplastin time prolonged | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Albumin urine present | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Beta 2 microglobulin urine increased | Investigations | MedDRA 23.1 | Systematic Assessment |
|
| Beta-N-acetyl-D-glucosaminidase increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Blood creatinine increased | Investigations | MedDRA 23.1 | Systematic Assessment |
|
| Blood iron decreased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Blood pressure diastolic increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Blood uric acid increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Glucose urine | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Glucose urine present | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Lymphocyte count decreased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Monocyte count decreased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Serum ferritin decreased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Urinary casts | Investigations | MedDRA 23.1 | Systematic Assessment |
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| White blood cell count increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Urinary sediment present | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Protein urine present | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Urine albumin/creatinine ratio increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Urine ketone body present | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Cystatin C increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Alpha 1 microglobulin increased | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Magnetic resonance imaging brain abnormal | Investigations | MedDRA 23.1 | Systematic Assessment |
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| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 23.1 | Systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |