Extension Study for Patients Who Have Participated in a BMN 701 Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01435772
• Date of registration: 08/09/2011
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: Extension Study for Patients Who Have Participated in a BMN 701 Study
• Scientific title: A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
• Date of first enrolment: August 15, 2011
• Target sample size: 21
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01435772
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, France, Germany, New Zealand, United Kingdom, United States

Contacts

• Name: Medical Monitor
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Inclusion Criteria:

- Have completed 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.

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.



Age minimum: 13 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Biological: BMN 701

Primary Outcome(s)

Number of Participants With a Positive Anti-BMN 701 Antibody [Time Frame: Baseline, Week 144]

Number of Participants With a Positive Anti-BMN 701 Antibody Response [Time Frame: Baseline, Week 144]

Secondary Outcome(s)

Insulin-like Growth Factor Binding Protein 3 (IGFBP3) [Time Frame: Baseline, Week 144]

Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 [Time Frame: Baseline, Week 144]

Percent Predicted Upright Forced Vital Capacity (FVC) [Time Frame: Baseline, Week 144]

6 Minutes Walk Test (Meters) [Time Frame: Baseline, Week 144]

Maximum Voluntary Ventilation (MVV) [Time Frame: Baseline, Week 144]

Plasma IGF-II Concentration [Time Frame: Baseline, Week 144]

Percent Predicted Maximum Expiratory Pressure (MEP) [Time Frame: Baseline, Week 144]

Plasma IGF-I Concentration [Time Frame: Baseline, Week 144]

Percent Predicted Maximal Inspiratory Pressure (MIP) [Time Frame: Baseline, Week 144]

Secondary ID(s)

POM-002

2011-001805-28

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 22/05/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01435772