Investigation of a New, Oral Growth Hormone Secretagogue, Macimorelin (AEZS-130) as a Growth Hormone Stimulation Test.
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Purpose
The diagnosis of GH deficiency (GHD) in adults is established by laboratory testing in patients with an appropriate clinical history of hypothalamic pituitary disease. Two tests that are considered to be gold standard tests for the diagnosis of GHD are the insulin tolerance test (ITT) and GHRH combined with arginine. However, these tests are either bothersome (given intravenously) to the patient or are linked with side effects. Therefore, an orally available compound like AEZS-130(formerly ARD-07), if demonstrated to be safe and providing adequate sensitivity and specificity could be a welcome alternative and/or complement to the current available tests.
The intent was to recruit 40 AGHD patients and 40 normal control subjects into this trial, but the original sponsor (Ardana Biosciences Ltd.) discontinued the study for financial reasons before this was completed. At the time of withdrawal of GHRH from the market in 2008, 42 AGHD patients and 10 normal controls had completed the study at 9 US sites. This study is now being reactivated to complete the remaining 30 matched control subjects.
Additionally upon agreement with the FDA in a SPA, 10 additional adult grown hormone deficient and their matched control will be enrolled into this trial for a total treated population approximatively 100 subjects.
| Condition | Intervention | Phase |
|---|---|---|
|
Diagnosis of Adult Growth Hormone Deficiency (AGDH) |
Other: macimorelin as a diagnostic |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Diagnostic |
| Official Title: | A Multi-center, Study Investigating a New, Oral Growth Hormone Secretagogue (GHS)(AEZS 130, Formerly ARD-07) as a Growth Hormone (GH) Stimulation Test in Terms of Safety and Efficacy |
- Diagnostic efficacy [ Time Frame: March 2011 ] [ Designated as safety issue: No ]Diagnostic efficacy will be based on the area under the ROC curve (> 0.85) using peak GH levels.
- Sensitivity(ROC) [ Time Frame: March 2011 ] [ Designated as safety issue: No ]
- Specificity (ROC) [ Time Frame: March 2011 ] [ Designated as safety issue: No ]
- Misclassification (CART) analyses [ Time Frame: March 2011 ] [ Designated as safety issue: No ]
- Safety [ Time Frame: March 2011 ] [ Designated as safety issue: No ]
- Incidence of treatment-emergent AEs
- Laboratory findings
- Vital signs
- ECGs
| Estimated Enrollment: | 100 |
| Study Start Date: | June 2007 |
| Study Completion Date: | July 2011 |
| Primary Completion Date: | July 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: AEZS 130 ( formerly ARD-07) |
Other: macimorelin as a diagnostic
A single administration of AEZS-130
Other Name: AEZS-130 (formerly ARD-07)
|
Detailed Description:
Thirty control subjects (i.e., without AGHD) will be matched to the 30 AGHD patients who were not previously matched. Matching will be based upon gender, age, BMI, and estrogen status for females. They will receive one oral dose of AEZS-130 followed by serial blood draws for growth hormone, IGF-1 and PK determinations. There will be no cross over due to the unavailability of GHRH (Geref) in the United States. Under Amendment #4 to this protocol, 10 additional AGHD subjects will be enrolled and matched as described above.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
Inclusion for Matched Control Subjects:
- Undergone normal growth and development
- Normal serum PRL concentrations
- Females should have a history of regular, age-appropriate menses
- Males should have normal serum testosterone concentrations
- Matched GHD subject already enrolled in study; matched in terms of sex, age, BMI and Estrogen status (women only)
Exclusion Criteria for Matched Control Subjects:
- Inability or unwillingness to comply with study medication
- Pregnancy or lactation
- Clinically relevant ECG abnormalities (including QT/QTc interval > 450 ms) at any time prior to dosing at Visit 2
- Treatment with any drugs that might prolong QT/QTc
Inclusion criteria dor Adult GHD Subjects:
- Confirmed GH deficiency with a low IGF-1
- 3 months of stable treatment for those requiring hormone replacement therapy for hormones deficiencies other than GHD
- subjects with hypogonadism must be treated with sex steroid therapy, excluding women over 50 yr of age
- women on estrogen therapy, for whatever reason, must be on stable treatment for ar least 3 months prior to study
Exclusion criteria for Adult GHD Subjects:
- Untreated hypothyroidism
- Known hypersensitivity to any excipient in study medication
- Inability or unwillingness to comply with study procedures
- Intracranial lesions stable for less than 12 months
- GH therapy within one month of study entry
- Clinically significant cardiovascular, or cerebrovascular disease
- Current active malignancy other than non-melanoma skin cancer
- Renal or hepatic dysfunction (> 3 x ULN LFEs - ASAT; ALAT; GGT or creatinine > 2x ULN)
- Pregnancy or lactation
- Active Cushing's disease
- Clinically relevant ECG abnormalities (including QT/QTc interval > 450 ms) at any time prior to dosing at Visit 2
- Treatment with any drugs that might prolong QT/QTc
Contacts and Locations| United States, Arizona | |
| Celerion | |
| Tempe, Arizona, United States, 85283 | |
| United States, California | |
| Cedars-Sinai Medical Center | |
| Los Angeles, California, United States, 90048 | |
| Harbor-UCLA Medical Center | |
| Torrance, California, United States, 90502 | |
| United States, Illinois | |
| Northwestern University | |
| Chicago, Illinois, United States, 60611-3008 | |
| Radiant Research Inc. | |
| Chicago, Illinois, United States, 60654 | |
| United States, Maryland | |
| John Hopkins University | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Nebraska | |
| Celerion | |
| Lincoln, Nebraska, United States, 68502 | |
| United States, New Jersey | |
| Celerion | |
| Neptune, New Jersey, United States, 07753 | |
| United States, Oregon | |
| Oregon Health & Science University | |
| Portland, Oregon, United States, 97239 | |
| United States, Texas | |
| Baylor College of Medicine | |
| Houston, Texas, United States, 77030 | |
| Cetero Research | |
| San Antonio, Texas, United States, 78229-4801 | |
| United States, Washington | |
| VA Puget Sound HCS University of Washington | |
| Tacoma, Washington, United States, 98493 | |
| Principal Investigator: | Beverly MK Biller, MD | Massachusetts General Hospital, Boston |
More Information
No publications provided
| Responsible Party: | Ines Altemose, Director of Clinical Operations, AEterna Zentaris Inc. |
| ClinicalTrials.gov Identifier: | NCT00448747 History of Changes |
| Other Study ID Numbers: | AEZS 130 047 |
| Study First Received: | March 16, 2007 |
| Last Updated: | July 12, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by AEterna Zentaris:
|
Ghrelin mimetic, growth hormone secretagogue |
Additional relevant MeSH terms:
|
Dwarfism, Pituitary Endocrine System Diseases Dwarfism Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Bone Diseases, Endocrine Hypopituitarism Pituitary Diseases |
Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 22, 2013