Clinical Trial Results:
Follow-Up Study in Patients with Acromegaly Previously Participating in Chiasma Study CH-ACM-01
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Summary
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EudraCT number |
2015-001292-51 |
Trial protocol |
DE HU SI PL IT |
Global end of trial date |
12 Jan 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
23 Dec 2018
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First version publication date |
23 Dec 2018
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
CH-ACM-01-FU
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Chiasma, Inc.
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Sponsor organisation address |
460 Totten Pond Road, Suite 530, Waltham, Massachusetts, United States, 02451
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Public contact |
William H. Ludlam, Sr. VP Clinical Development and Medical
Affairs, Chiasma, Inc., William H. Ludlam, Sr. VP Clinical Development and Medical
Affairs, Chiasma, Inc., 1 6179285294, william.ludlam@chiasmapharma.com
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Scientific contact |
Asi Haviv, VP Clinical Development, Chiasma, Inc., Asi Haviv, VP Clinical Development, Chiasma, Inc., 972 89393888, Asi@chiasmapharma.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
12 Jan 2016
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
12 Jan 2016
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Global end of trial reached? |
Yes
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Global end of trial date |
12 Jan 2016
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
This was a non-interventional, observational study. The objective was to assess reinitiation of standard of care medical treatment for acromegaly, following the completion of study CH-ACM-01, where patient were treated with Octreotide Capsules (previously called oral octreolin).
Group 1 were patients who did not reinitiate standard of care acromegaly treatment within the 2 weeks follow up period, following completion of CH-ACM-01. For this group the objective was to investigate if standard of care treatment for acromegaly was reinitiated following the completion of the study.
Group 2 were patients who reinitiated standard of care acromegaly treatment within the 2 weeks follow up period and were responders to octreotide capsules at the end of study CH-ACM-01. For this group the objective was to assess the rationale for reinitiation of standard of care medical treatment for acromegaly.
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Protection of trial subjects |
Not applicable
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Background therapy |
In both groups, patients were treated/could reinitiate treatment with standard of care treatment for acromegaly per best clinical practice. | ||
Evidence for comparator |
Not applicable | ||
Actual start date of recruitment |
29 May 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Poland: 8
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Country: Number of subjects enrolled |
Romania: 9
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Country: Number of subjects enrolled |
Slovenia: 2
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Country: Number of subjects enrolled |
Germany: 10
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Country: Number of subjects enrolled |
Hungary: 11
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Country: Number of subjects enrolled |
Italy: 4
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Country: Number of subjects enrolled |
Lithuania: 3
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Country: Number of subjects enrolled |
Netherlands: 10
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Country: Number of subjects enrolled |
Serbia: 7
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Country: Number of subjects enrolled |
Slovakia: 4
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Country: Number of subjects enrolled |
United Kingdom: 10
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Country: Number of subjects enrolled |
Israel: 9
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Worldwide total number of subjects |
87
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EEA total number of subjects |
71
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
67
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From 65 to 84 years |
20
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85 years and over |
0
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Recruitment
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Recruitment details |
The study was a follow-up study of trial CH-ACM-01 and was performed in centers that previously participated in that study. Patients who completed study CH-ACM-01 were invited to participate. Information was collected retrospectively from eligible patients and documented. No study drug was administered. | |||||||||
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Pre-assignment
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Screening details |
Acromegaly patients who previously participated in and completed study CH-ACM-01. | |||||||||
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Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | |||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | |||||||||
Blinding implementation details |
Not applicable
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Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Group 1 (no new acromegaly treatment) | |||||||||
Arm description |
Patients who had completed trial CH-ACM-01 and did not initiate new acromegaly therapy, or patients for whom data on new acromegaly therapy is missing. | |||||||||
Arm type |
Standard of care | |||||||||
Investigational medicinal product name |
Standard of care
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection/infusion, Tablet
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Routes of administration |
Oral use, Subcutaneous use, Intramuscular use
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Dosage and administration details |
Patients received standard of care, including injectable somatostatin receptor ligands, dopamine agonists, growth Hormone Antagonist, or no treatment
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Arm title
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Group 2 (new acromegaly therapy) | |||||||||
Arm description |
Patients who responded to MYCAPSSA Treatment (oral octreotide) in study CH-ACM-01 and who initiated new acromegaly Treatment after completion of Trial CH-ACM-01 | |||||||||
Arm type |
Standard of care | |||||||||
Investigational medicinal product name |
Standard of care
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection/infusion, Tablet
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Routes of administration |
Intravenous use, Oral use, Subcutaneous use
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Dosage and administration details |
Patients received standard of care, including injectable somatostatin receptor ligands, dopamine agonists, growth Hormone Antagonist, or no treatment
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Investigational medicinal product name |
Standard of care
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Tablet, Solution for injection/infusion
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Routes of administration |
Oral use, Subcutaneous use, Intramuscular use
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Dosage and administration details |
Patients received standard of care, including injectable somatostatin receptor ligands, dopamine agonists, growth Hormone Antagonist, or no treatment
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Baseline characteristics reporting groups
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Reporting group title |
Group 1 (no new acromegaly treatment)
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Reporting group description |
Patients who had completed trial CH-ACM-01 and did not initiate new acromegaly therapy, or patients for whom data on new acromegaly therapy is missing. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Group 2 (new acromegaly therapy)
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Reporting group description |
Patients who responded to MYCAPSSA Treatment (oral octreotide) in study CH-ACM-01 and who initiated new acromegaly Treatment after completion of Trial CH-ACM-01 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Group 1 (no new acromegaly treatment)
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Reporting group description |
Patients who had completed trial CH-ACM-01 and did not initiate new acromegaly therapy, or patients for whom data on new acromegaly therapy is missing. | ||
Reporting group title |
Group 2 (new acromegaly therapy)
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Reporting group description |
Patients who responded to MYCAPSSA Treatment (oral octreotide) in study CH-ACM-01 and who initiated new acromegaly Treatment after completion of Trial CH-ACM-01 | ||
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End point title |
Proportion of patients initiating acromegaly treatment [1] [2] | ||||||
End point description |
Proportion of patients initiating acromegaly treatment. This endpoint was analysed for Group 1 only.
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End point type |
Primary
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End point timeframe |
This was a follow-up, retrospective, non-interventional study of patients with acromegaly who previously participated in study CH-ACM-01 where they were treated with MYCAPSSA. Follow up occured between 1.5-2.5 years after completion of study CH-ACM-01.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: This study was descriptive in nature, with no formal primary hypothesis testing. [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: In line with the objectives of the trial, this primary endpoint was only analysed for Group 1 of the Trial Population. |
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| No statistical analyses for this end point | |||||||
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End point title |
Reasons for reinitiating acromegaly treatment [3] [4] | ||||||||||||||||
End point description |
Reasons for patients to reinitiate acromegaly treatment. This endpoint was analysed for Group 2 only.
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End point type |
Primary
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End point timeframe |
This was a follow-up, retrospective, non-interventional study of patients with acromegaly who previously participated in study CH-ACM-01 where they were treated with MYCAPSSA. Follow up occured between 1.5-2.5 years after completion of study CH-ACM-01.
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| Notes [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: This study was descriptive in nature, with no formal primary hypothesis testing. [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period. Justification: In line with the objectives of the trial, this primary endpoint was only analysed for Group 2 of the Trial Population. |
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| No statistical analyses for this end point | |||||||||||||||||
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Adverse events information [1]
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Timeframe for reporting adverse events |
Four-week follow-up period (The study duration for each eligible patient did not exceed 4 weeks from the time of signing ICF to complete capture of the requested information).
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Assessment type |
Systematic | |||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||
Dictionary version |
14
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Reporting groups
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Reporting group title |
Group 1 (no new acromegaly treatment)
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Reporting group description |
Patients who had completed trial CH-ACM-01 and did not initiate new acromegaly therapy, or patients for whom data on new acromegaly therapy is missing. | |||||||||||||||
Reporting group title |
Group 2 (new acromegaly therapy)
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Reporting group description |
Patients who responded to MYCAPSSA Treatment (oral octreotide) in study CH-ACM-01 and who initiated new acromegaly Treatment after completion of Trial CH-ACM-01 | |||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 1% | ||||||||||||||||
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| Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: This was a non-interventional study. Data on medical treatment for acromegaly were collected from patient files. Adverse events were not collected. |
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| Not applicable | |||
