Trial Outcomes & Findings for A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously (NCT NCT02618512)
NCT ID: NCT02618512
Last Updated: 2018-04-17
Results Overview
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
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.
Results posted on
2018-04-17
Participant Flow
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
Baseline characteristics by cohort
| Measure |
SBC-103
n=3 Participants
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.
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|---|---|
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Age, Categorical
<=18 years
|
3 Participants
n=39 Participants
|
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Age, Categorical
Between 18 and 65 years
|
0 Participants
n=39 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Region of Enrollment
United Kingdom
|
3 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.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
Outcome measures
| Measure |
SBC-103
n=3 Participants
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.
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|---|---|
|
Safety and Tolerability of SBC-103
|
3 Participants
|
Adverse Events
SBC-103
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SBC-103
n=3 participants at risk
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.
|
|---|---|
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Infections and infestations
Device Related Infection
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Erythema Multiforme
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Surgical and medical procedures
Catheter placement
|
33.3%
1/3 • Number of events 1 • 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.
|
Other adverse events
| Measure |
SBC-103
n=3 participants at risk
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.
|
|---|---|
|
Injury, poisoning and procedural complications
Fall
|
100.0%
3/3 • Number of events 8 • 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.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
100.0%
3/3 • Number of events 4 • 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.
|
|
Injury, poisoning and procedural complications
Contusion
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Injury, poisoning and procedural complications
Scratch
|
33.3%
1/3 • Number of events 1 • 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.
|
|
General disorders
Pyrexia
|
66.7%
2/3 • Number of events 5 • 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.
|
|
General disorders
Gait disturbance
|
33.3%
1/3 • Number of events 2 • 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.
|
|
General disorders
Pain
|
33.3%
1/3 • Number of events 2 • 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.
|
|
General disorders
Complication associated with device
|
33.3%
1/3 • Number of events 1 • 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.
|
|
General disorders
Excessive granulation tissue
|
33.3%
1/3 • Number of events 1 • 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.
|
|
General disorders
Fatigue
|
33.3%
1/3 • Number of events 1 • 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.
|
|
General disorders
Peripheral swelling
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
100.0%
3/3 • Number of events 6 • 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.
|
|
Gastrointestinal disorders
Diarrhoea
|
66.7%
2/3 • Number of events 8 • 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.
|
|
Gastrointestinal disorders
Toothache
|
66.7%
2/3 • Number of events 4 • 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.
|
|
Gastrointestinal disorders
Constipation
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Dysphagia
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Vomiting projectile
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Infections and infestations
Nasopharyngitis
|
100.0%
3/3 • Number of events 11 • 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.
|
|
Infections and infestations
Staphylococcal infection
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Infections and infestations
Candida infection
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Infections and infestations
Ear infection
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Infections and infestations
Urinary tract infection
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
33.3%
1/3 • Number of events 1 • 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.
|
|
General disorders
Catheter site pain
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Psychiatric disorders
Restlessness
|
66.7%
2/3 • Number of events 4 • 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.
|
|
Psychiatric disorders
Anxiety
|
66.7%
2/3 • Number of events 3 • 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.
|
|
Psychiatric disorders
Sleep disorder
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Psychiatric disorders
Abnormal behaviour
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Psychiatric disorders
Breath holding
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Psychiatric disorders
Emotional distress
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Nervous system disorders
Headache
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Nervous system disorders
Dyskinesia
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Nervous system disorders
Seizure
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Nervous system disorders
Motor dysfunction
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Nervous system disorders
Petit mal epilepsy
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Skin warm
|
66.7%
2/3 • Number of events 2 • 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.
|
|
Skin and subcutaneous tissue disorders
Application site erythema
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Excessive granulation tissue
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Hair disorder
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
33.3%
1/3 • Number of events 2 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Choking
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Reproductive system and breast disorders
Premenstrual pain
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Cardiac disorders
Bradycardia
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Endocrine disorders
Precocious puberty
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Immune system disorders
Seasonal allergy
|
33.3%
1/3 • Number of events 1 • 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.
|
|
Investigations
Body temperature increased
|
33.3%
1/3 • Number of events 1 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER