Primary objective: Safety.Secondary objective: Efficacy.
ID
Source
Brief title
Condition
- Hypothalamus and pituitary gland disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Adverse events.
Secondary outcome
• To document the efficacy of pasireotide s.c. in normalizing UFC at Week 12
and 24,
separately,
• To document the efficacy of pasireotide s.c. in achieving at least 50%
reduction of UFC from
baseline at Week 12 and 24, separately,
• To document the changes in clinical signs and symptoms,
• To document the changes in patient-reported outcome questionnaires
(CushingQoL and
WPAI-GH),
• To document the effects of pasireotide s.c. on the GH/IGF-I axis.
Background summary
The trial is planned as an Expanded Access study to provide access and to
further document
the safety and the efficacy of pasireotide in patients affected by Cushing*s
disease. While
regulatory approval is sought, there are no means available for patients with
Cushing*s disease
to receive pasireotide outside of a clinical trial. Patients with post-surgery
active disease or
patients who recur after surgery and de novo patients that are not candidates
to surgery do not
have an access path to this new agent. Implementation of an Expanded Access
Program will
allow access to pasireotide for patients with Cushing*s disease.
Pasireotide is an injectable somatostatin analogue.
The rationale for this study is to give patients with Cushing*s disease access
to pasireotide s.c.
as no medical treatment for Cushing*s disease is approved. Thus, a single arm,
open label
design is justified in this context.
Study objective
Primary objective: Safety.
Secondary objective: Efficacy.
Study design
Open-label non-comparative phase III 6900 µg bid.
Treatment up to 31-12-2013 or until product is commercially available (whatever
comes first). In case product is not yet commencially available in NL on
31-12-2013, the sponsor will arrange continuation of treatment
300 patient (approx. 4 in NL).
Intervention
Treatment with pasireotide s.c.
Study burden and risks
Risks: Adverse effects of study medication. If applicable: (minor)
risks/inconveniences of CRH stimulation test.
Burden:
Based on 1 year treatment:
15 visitsin approx. 1 year.
Females are not permitted to removed body hair in the 3 weeks prior to a visit.
14x physical examination.
13x blood tests (approx. 20 ml/visit, fasting). Screening for hepatitis B-C.
10x 24 h urine collection.
3x pregnancy test.
14x ECG.
5x echo gall bladder.
7x completion of 2 questionnaires (qulaity of life and work productivity).
If not performed yet: CRH stimulation test.
Optional: 2x cortisol levels in saliva, assessment of tumor sample removed
during prior surgery.
Raapopseweg 1
Arnhem 6824 DP
NL
Raapopseweg 1
Arnhem 6824 DP
NL
Listed location countries
Age
Inclusion criteria
1. male or female patients aged 18 years or above
2. patients with confirmed diagnosis of Cushing's disease as evidenced by:
* Mean urninary free cortisol of three 24-hour urine samples collected during the 3-week screening period above the upper limit of the laboratory non range
* morning plasma ACTH within the normal or above normal range
* either MRI confirmation of pituitary adenoma (greater than or equal to 0.6 cm), or
inferior petrosal sinus gradient >3 after CRH stimulation (if IPSS had previously
been performed without CRH, a central to peripheral prestimulation
gradient > 2 is required. If IPSS had not previously been performed, IPSS
with CRH stimulation is required).
3. Patients with de novo Cushing*s disease must not be considered as candidates for pituitary
surgery
4. Karnofsky performance status >60
5. For patients on previous medical treatment for Cushing*s disease the following washout
periods must be completed before screening assessments are performed (see protocol section 5.2, item 6 for details).
Exclusion criteria
1. Radiotherapy of the pituitary <4 weeks before screening or patient who has not recovered
from side effects
2. Patients with compression of the optic chiasm causing acute clinically significant visual
field defect
3. Patients with Cushing*s syndrome due to ectopic ACTH secretion
4. Patients with hypercortisolism secondary to adrenal tumors or nodular (primary) bilateral
adrenal hyperplasia
5. Patients who have a known inherited syndrome as the cause for hormone over secretion
6. Patients with a diagnosis of glucocorticoid-remedial aldosteronism (GRA)
7. Patients who have undergone major surgery within 1 month prior to screening
8. Patients with known gallbladder or bile duct disease, acute or chronic pancreatitis (see protocol page 26 for exceptions)
9. Diabetic patients whose blood glucose is poorly controlled as evidenced by HbA1C >8%
10. Patients who have clinically significant impairment in cardiovascular function or are at
risk thereof (see protocol for details)
11. Female patients who are pregnant or lactating, or are of childbearing potential and not
practicing a medically acceptable method of birth control.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | clinicaltrials.gov; NCT01374906 |
| EudraCT | EUCTR2010-024165-44-NL |
| CCMO | NL38992.044.11 |