E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056918 |
E.1.2 | Term | Sanfilippo's syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056890 |
E.1.2 | Term | Mucopolysaccharidosis III |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of intravenous administration of SBC-103 in previously studied, SBC-103 treatment naïve subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. |
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E.2.2 | Secondary objectives of the trial |
PK, activity, PD/efficacy of treatment with SBC-103 administered IV |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Previous participation in the NGLU-CL01 study. 2. Subject consents or subject’s parent or legal guardian (if applicable) grants consent for the subject to participate in the study and provides informed consent prior to any study procedures being performed. If the subject is of minor age; he/she is willing to provide assent where required per local regulations, and if deemed able to do so. 3. Female subjects who are of childbearing potential at the time of consent or who become of childbearing potential during participation on study (a) must have a negative urine pregnancy test at Screening, (b) cannot be breast feeding, and (c) must use a highly reliable method of birth control (expected failure rate less than 5% per year) for the duration of the study and for 30 days after last dose of SBC-103. Women may be considered of non-childbearing potential if they have not started their menses or are surgically sterile (i.e., total hysterectomy or bilateral salpingo-oophorectomy). 4. Male subjects must use a highly reliable method of birth control (expected failure rate less than 5% per year) during any sexual contact with females of childbearing potential while participating in the study and for 30 days following discontinuation from this study even if he has undergone a successful vasectomy. 5. Willingness and ability to comply with protocol requirements to the extent that may be expected of a subject with cognitive impairment. |
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E.4 | Principal exclusion criteria |
1. Received treatment with gene therapy at any time, or any investigational drug (including high dose genistein > 150 mg/kg/day) or device intended as a treatment for MPS IIIB within 30 days prior to Screening, or is currently being treated in another study that involves an investigational drug or device. 2. Has any internal or non-removable external metal items 3. Previous hematopoietic stem cell or bone marrow transplant. 4. Known or suspected hypersensitivity to anaesthesia or the use of a sedative is contraindicated for any other reason. 5. History of poorly controlled seizure disorder. 6. A bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy. 7. Known hypersensitivity to eggs. Subjects at high risk for food allergy that may include eggs should be tested according to local guidelines. 8. Other medical conditions or comorbidities
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of SBC-103 in subjects with MPS IIIB. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• PK profile of SBC-103 • Onset and magnitude of changes in levels of total HS in CSF, serum, and urine following dosing with SBC-103 administered IV. • Changes from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) in the following: clinical laboratory tests (haematology, serum chemistry, and urinalysis)and CSF findings (cell counts, glucose, and protein); 12-lead ECGs; vital signs; physical examination and use of concomitant medications/therapies. • Change in neurocognitive and developmental function during the NGLU-CL01-T study (on treatment) • Change in neurocognitive and developmental function from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) • Changes in brain structure • Changes in BBB integrity during the NGLU-CL01-T study (on treatment) • Changes in BBB integrity from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |