E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036143 |
E.1.2 | Term | Pompe's disease |
E.1.2 | System Organ Class | 100000004850 |
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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 |
The primary objectives of the study are:
• To provide long-term, ongoing, treatment to patients who have participated in a BMN 701 clinical trial.
• To evaluate the long-term safety and tolerability both during and following BMN 701 administration;
• To determine the anti-BMN 701 antibody response to BMN 701
• To determine the anti-IGF-I and anti-IGF-II antibody response to BMN 701.
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E.2.2 | Secondary objectives of the trial |
Secondary objectives of the study are to measure:
• Mobility and functional exercise capacity, as measured by the six minute walk test (6MWT);
• Pulmonary function, as measured by percent (%) predicted upright and supine forced vital capacity (FVC) and by % predicted upright maximum expiratory pressure (MEP), maximum inspiratory pressure (MIP), and maximal ventilatory volume (MVV);
• Urinary tetrasaccharide;
• IGF levels (IGF-I, IGF-II, and IGFBP3)
• Muscle strength by quantitative muscle testing (QMT) of the arm and leg (optional);
• Maximum cross-sectional muscle dimension (optional);
• Levels of muscle glycogen (optional) and;
• Muscle GAA activity (optional).
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Participated in a prior BMN 701 clinical development study;
• Have provided written informed consent after the nature of the study has been explained prior to performance of any study-related procedures. Minors may participate as long as they provide written assent after the nature of the study has been explained to them and after their parent or legal guardian has provided written informed consent, prior to the performance of any study-related procedures;
• Have been diagnosed with late-onset Pompe disease, based on the entry criteria of a prior BMN 701 study;
• If sexually active, be willing to use 2 known effective methods of contraception from Screening until 4 months after the last dose of study drug;
• If female, and not considered to be of childbearing potential, be at least 2 years post-menopausal, or have had tubal ligation at least 1 year prior to screening, or have had a total hysterectomy;
• If female, and of childbearing potential, have a negative pregnancy test during the Screening Period and at the Baseline visit, and be willing to have additional pregnancy tests during the study;
• Have the ability to comply with the protocol requirements, in the opinion of the Investigator.
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E.4 | Principal exclusion criteria |
• Have received any experimental or approved therapy for Pompe disease, other than BMN 701, subsequent to completion of a BMN 701 study and prior to entry into POM-002;
• Have received, or are anticipated to receive, any investigational medication, other than BMN 701, within 30 days prior to the first dose of study drug;
• Are breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
• Have a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient’s ability to comply with the protocol requirements or compromise the patient’s well being or safety.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary objectives of the study are:
• To evaluate the safety and tolerability of BMN 701;
• To determine the anti-BMN 701 antibody response to BMN 701
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Various assessments are conducted every 2 weeks. |
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E.5.2 | Secondary end point(s) |
Various assessments are conducted every 2 weeks. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Various assessments are conducted every 2 weeks. |
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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 | Yes |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
France |
Germany |
United States |
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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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The study will end after the last patient has completed the Follow-up or Termination visit. |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |