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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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EUCTR |
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Last refreshed on:
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24 July 2012 |
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Main ID: |
EUCTR2008-002302-18-DE |
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Date of registration:
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26/09/2008 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Scientific title:
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An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease -
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Date of first enrolment:
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16/02/2009 |
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Target sample size:
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18 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-002302-18 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: yes
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
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Phase:
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Countries of recruitment
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Germany
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United Kingdom
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Contacts
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion criteria:
1. Male or female, 18 to 74 years of age at time of consent
2. Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory GAA genotyping will be performed on all subjects who are screened for the study.
3. Naïve to ERT or has not received ERT in the 3 months prior to screening
4. Willing not to initiate ERT or other prohibited treatment during study participation
5. Functional grade for arms and/or legs =2 (See Appendix 2) OR sitting FVC = 30% and < 80% of predicted value, with maximum FVC (L) value reproducible (± 15 % ) between Visits 1 and 2
6. Subjects of reproductive potential agree to use reliable methods of contraception during the study
7. Subject is willing and able to provide written informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
1. Any intercurrent condition that may preclude accurate interpretation of study data
2. Obstructive pulmonary disease
3. Invasive ventilatory support
4. Use of noninvasive ventilatory support > 8 hours a day while awake
5. History of QTc prolongation > 450 msec for males and > 470 msec for females
6. History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (e.g., miglustat, miglitol)
7. Pregnancy or breast-feeding
8. Current or recent drug or alcohol abuse
9. Treatment with another investigational drug within 30 days of study start
10. Use of prohibited medications < 3 months prior to screening
11. Otherwise unsuitable for the study in the opinion of investigator (e.g., a subject with poor reproducibility of assessments between days -28 and -27 may be excluded at the investigator’s discretion)
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Pompe Disease
MedDRA version: 9.1
Level: LLT
Classification code 10036143
Term: Pompe's disease
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Intervention(s)
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Product Code: AT2220
Pharmaceutical Form: Powder for oral solution
CAS Number: 73285-50-4
Current Sponsor code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 2.5-
Product Code: AT2220
Pharmaceutical Form: Powder for oral solution
CAS Number: 73285-50-4
Current Sponsor code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 5.0-
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Primary Outcome(s)
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Secondary Objective: • To evaluate the effect of AT2220 on functional parameters of Pompe disease
• To evaluate the effect of AT2220 on pharmacodynamic parameters of Pompe disease
• To evaluate pharmacokinetics of AT2220
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Primary end point(s): • Treatment-emergent physical exam changes up to end of study (EOS) visit
• Treatment-emergent vital signs (blood pressure, heart rate, respiratory rate) changes up to EOS
• Treatment-emergent safety laboratory test (hematology, chemistry, urinalysis) abnormalities up to EOS
• Treatment-emergent ECG abnormalities up to EOS
• Treatment-emergent adverse events (AEs) up to 24 hours after EOS
• Treatment-emergent changes in concomitant medications up to EOS
• Adverse events leading to permanent discontinuation of study medication
• Serious adverse events (SAEs) up to 28 days after study medication discontinuation
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Main Objective: To evaluate the safety and tolerability of AT2220 in patients with Pompe disease
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Secondary ID(s)
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POM-CL-201
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Source(s) of Monetary Support
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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