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Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03708900
Recruitment Status : Recruiting
First Posted : October 17, 2018
Last Update Posted : May 6, 2022
Sponsor:
Information provided by (Responsible Party):
RECORDATI GROUP

Study Description
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Brief Summary:
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease.

Condition or disease Intervention/treatment Phase
Cushing's Disease Drug: LCI699 Phase 2

Detailed Description:
The period 1 study duration will be 12 weeks. The study will include a screening period of up to 4 weeks prior to Day 0 (baseline) (to allow for an adequate washout period from any medications that may modify cortisol levels). All subjects being treated with osilodrostat at 12 weeks and obtaining benefit from therapy, per investigator judgment, will be offered participation in an optional 9-month extension period, during which assessment of the PD activity and safety/tolerability of osilodrostat will be done. Patients who do not enter the optional extension period will have a safety follow up visit 4 weeks later.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Multicenter, Open-label, Non-comparative Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Tolerability of Osilodrostat in Children and Adolescent Patients With Cushing's Disease
Actual Study Start Date : April 28, 2021
Estimated Primary Completion Date : February 21, 2023
Estimated Study Completion Date : November 29, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: LCI699 (osilodrostat)
Subjects with cushing's disease taking LCI699 (osilodrostat)
 Drug: LCI699
osilodrostat (LCI699) is in the form of tablets for oral administration and comes in the following tablet strengths: 1 milligram (mg), 5 mg, and 10mg.
Other Name: osilodrostat


Outcome Measures
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Primary Outcome Measures :
  1. Core Study: Evaluate the pharmacokinetics (PK) of osilodrostat using Pharmacokinetic parameter - Cmax - of osilodrostat up to Week 12 in children and adolescents 6 to less than 18 years of age with Cusihing's Disease [ Time Frame: up to Week 12 ]
    evaluate the pharmacokinetics (PK) by Cmax of osilodrostat in children and adolescents 6 to less than 18 years of age with Cushing's Disease

  2. Core Study: Evaluate the pharmacokinetics of osilodrostat using Pharmacokinetic parameter - Ctrough - of osilodrostat up to Week 12 in children and adolescents 6 to less than 18 years of age with Cushing's Disease [ Time Frame: up to Week 12 ]
    evaluate the pharmacokinetics (PK) by Ctrough of osilodrostat in children and adolescents 6 to less than 18 years of age with Cushing's Disease


Secondary Outcome Measures :
  1. Core Study: Percentage of patients with normal mean urinary free cortisol (mUFC) at week 6 and week 12 (or end of treatment) [ Time Frame: week 6, week 12 (or end of treatment) ]
    The assessment in the core period will be done by taking the percentage of patients with normal mUFC at week 6 and week 12 (or end of treatment).

  2. Core Study: Change from baseline in mean urinary free cortisol (mUFC) during the core study period [ Time Frame: Baseline, 12 weeks ]
    The assessment will be done by comparison of change from the baseline in mUFC during core study period on patients

  3. Extension: Efficacy of osilodrostat as measured by mUFC levels up to Month 12 [ Time Frame: up to month 12 ]
    The assessment of efficacy of osilodrostat to be measured by mUFC levels up to 12 months on patients


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Cushing's disease of endogenous origin: Who have failed surgery (or) who are awaiting surgery (or) for whom surgery is not an immediate option.

  • The diagnosis of Cushing's disease must be confirmed by each of the following:

    • The clinical criterion of decreasing growth percentiles with increasing weight (as evidenced by the presence of a contrast in height and BMI standard deviation (SD) scores, defined as height standard deviation score (SDS) < 0 and BMI SDS > 0, and a strong clinical suspicion of Cushing's disease, such as photographic evidence of a change in facial appearance);
    • Abnormal low-dose (0.5 mg Q6h x 48 hours) dexamethasone suppression test, defined as plasma cortisol levels > 1.8 mcg/dl, at time point 48 hours after the first dose of dexamethasone;
    • Measurable morning ACTH levels, assessed before 10 am;
    • Two 24-hour urinary free cortisol values > 1.3 x ULN
    • If the dexamethasone suppression test does not meet the above mentioned criteria, the diagnosis of Cushing's disease may be confirmed by the following: Midnight serum cortisol levels > upper limit of normal (ULN), assessed while the patient is sleeping and after pre-cannulation (OR) two samples of late night salivary cortisol greater than ULN for the assay
  • Able to swallow study drug tablets (not crushed or split)
  • Parents or legal guardians able to provide consent/assent

Exclusion Criteria:

  • Patients with macroadenoma complicated by compressive symptoms (requiring urgent surgical intervention) or at high risk for compressive symptoms due to mass effect of tumor (concern of corticotroph tumor progression)
  • Hypercortisolism not due to Cushing's disease
  • Insufficient washout period from any other medication used to lower cortisol levels (5 half-lives of any drug)
  • Use of other investigational drugs at the time of enrollment, or within 30 days, or prior to completion of a wash-out duration that is at least 5 half- lives of the drug, at the time of enrollment, whichever is longer. Local regulations may require a longer wash-out period or specify other limitations for participation in an investigational trial, in which case they will be applicable as well.
  • Body weight <30kg

Other protocol-defined inclusion/exclusion may apply.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03708900


Contacts
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Contact: Recordati +390248787456 casi.m@recordati.it
Contact: Recordati +4161 205 61 00

Locations
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Belgium
Recordati Investigative Site Recruiting
Jette, Brussel, Belgium, 1090
Bulgaria
Recordati Investigative Site Recruiting
Varna, Bulgaria, 9010
Italy
Recordati Investigative Site Recruiting
Pisa, PI, Italy, 56124
Recordati Investigative Site Recruiting
Roma, Italy, 00165
Slovenia
Recordati Investigative Site Recruiting
Ljubljana, Slovenia, 1525
United Kingdom
Recordati Investigative Site Not yet recruiting
Liverpool, United Kingdom, L12 2AP
Recordati Investigative Site Recruiting
London, United Kingdom, E11BB
Sponsors and Collaborators
RECORDATI GROUP
Investigators
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Study Director: Recordati AG Recordati AG
More Information
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Responsible Party: RECORDATI GROUP
ClinicalTrials.gov Identifier: NCT03708900    
Other Study ID Numbers: CLCI699C2203
2018-001522-25 ( EudraCT Number )
First Posted: October 17, 2018    Key Record Dates
Last Update Posted: May 6, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by RECORDATI GROUP:
Cushing's disease (CD)
LCI699
osilodrostat
Pituitary Gland
 Adrenocorticotropic Hormone (ACTH)
Urinary Free Cortisol (UFC)
mean Urinary Free Cortisol (mUFC)
Additional relevant MeSH terms:
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ACTH-Secreting Pituitary Adenoma
Pituitary ACTH Hypersecretion
Hyperpituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Endocrine System Diseases
Adenoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Pituitary Neoplasms
Endocrine Gland Neoplasms
Neoplasms by Site