Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2015-001983-20 | EudraCT Number |
Not provided
Not provided
The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. The NGLU-CL01 study was a non-interventional study that evaluated structural brain abnormalities and blood brain barrier (BBB) integrity by magnetic resonance imaging (MRI) and cerebrospinal fluid/serum albumin index.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SBC-103 | Experimental | Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SBC-103 | Drug | SBC-103 is a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). Patients were started with low dose of SBC-103 for 12 weeks and then will escalate to a higher dose. |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability of SBC-103 | The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications | Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks. |
Not provided
Not provided
Participants from NGU-CL01 study were enrolled into the NGLU-CL01-T study. Subjects who met all of the inclusion criteria and none of the exclusion criteria were eligible to participate in this study.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | B46NH | United Kingdom |
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | SBC-103 | Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| ICF | No | No | Yes | Informed Consent Form | Jan 25, 2017 | Feb 16, 2018 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | SBC-103 | Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days. |
| 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 | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Count of Participants | 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 | Safety and Tolerability of SBC-103 | The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications | Posted | Count of Participants | Participants | Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks. |
|
|
|
Baseline to 96 Weeks.
Treatment-emergent AEs by System Organ Class (SOC) and Preferred Term are classified according to the Medical Dictionary for Regulatory Activities (MedDRA) v19.0.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | SBC-103 | Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days. | 0 | 3 | 2 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Device Related Infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Wound dehiscence | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Erythema Multiforme | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Catheter placement | Surgical and medical procedures | MedDRA (19.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fall | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Complication associated with device | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Excessive granulation tissue | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Vomiting projectile | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Breath holding | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Emotional distress | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Psychomotor hyperactivity | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Motor dysfunction | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Petit mal epilepsy | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Skin warm | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Application site erythema | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Excessive granulation tissue | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Hair disorder | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Premenstrual pain | Reproductive system and breast disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Precocious puberty | Endocrine disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA (19.0) | Systematic Assessment |
|
Due to early termination of the study only safety data are reported.
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Medical Monitor | Alexion | 617-613-1071 | Bert.Yao@alexion.com |
| ICF_000.pdf |
| Prot | Yes | No | No | Study Protocol | Feb 24, 2016 | Mar 19, 2018 | Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 1, 2017 | Mar 19, 2018 | SAP_002.pdf |
| ID | Term |
|---|---|
| D009084 | Mucopolysaccharidosis III |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|