Clinical trial NCT03967249: Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
Official title: An Open-Label Extension Trial of IONIS GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor Administered Monthly Subcutaneously to Patients With Acromegaly Being Treated With Long-Acting Somatostatin Receptor Ligands (SRL)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acromegaly
Keywords: Acromegaly, IONIS-GHR-LRx
Lead sponsor: Ionis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-05-30
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and efficacy of extended dosing with IONIS GHR-LRx in participants with acromegaly as add-on to somatostatin receptor ligands (SRL) therapy.

Detailed description: This extension study will include participants up to 60 participants with acromegaly from Study NCT03548415. All participants will remain on the same dose from the previous study and receive IONIS GHR-LRx (SC) injection once every 28 days as add-on to SRL therapy for 53 weeks. At the end of 53 weeks, participants will enter a 14-week post-treatment (PT) evaluation period.

Eligibility criteria: Inclusion Criteria:

* Randomized in index trial (CS2) and completed the entire study, or completion of the treatment period for CS2 and completed or plan to complete PTWK5 visit with an acceptable safety profile, per investigator judgment
* Participants with confirmed stable monthly regimen of SRL for 3 months prior to screening
* Able and willing to participate in a 53-week treatment and 14-week post-treatment study

Exclusion Criteria:

* Treatment with any other acromegaly medications taken prior to Day 1 within the time period: bromocriptine: 2 weeks, carbergoline: 4 weeks, quinagolide: 4 weeks, pegvisomant: 4 weeks and pasireotide: 4 months
* Participant who received surgery for pituitary adenoma in the last 3 months prior to screening and participants needing and/or planning to receive surgery for the pituitary adenoma during the trial
* Unwilling to comply with required study procedures during the treatment and post-treatment periods

Primary outcomes:
measure: The Incidence of Adverse Events
timeFrame: Up to approximately 16 months
Secondary outcomes:
measure: Percent Change from Baselines in Insulin-like Growth Factor I (IGF-1) Levels
timeFrame: Baseline and at Week 26 and Week 53
measure: Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.2 Times of Gender and Age-Adjusted Upper Limits
timeFrame: Week 26 and at 28 days after the Week 53 dose
measure: Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.0 Times of Gender and Age-Adjusted Upper Limits
timeFrame: Week 26 and at 28 days after the Week 53 dose
measure: Percentage of Participants who Begin Other Acromegaly Medication
timeFrame: Up to approximately 16 months
measure: Time From First Dose of IONIS GHR-LRx in this open label extension (CS3) to Date of Initiation of Other Acromegaly Medications
timeFrame: Up to approximately 16 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Evanston
country: United States
facility: Northwestern University
state: Illinois
city: Las Vegas
country: United States
facility: Palm Research Center, Inc.
state: Nevada
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Budapest
country: Hungary
facility: Magyar Honvedseg Allami Egeszsegugyi Kozpont, II. sz Belgyogyaszat Osztaly
city: Kaunas
country: Lithuania
facility: Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos - Endocrinology clinic
city: Vilnius
country: Lithuania
facility: Vaidoto Urbanaviciaus Individuali imone - Endokrinologijos klinika
city: Kraków
country: Poland
facility: Centrum Nowoczesnych Terapii "Dobry Lekarz" Sp. z o. o.
city: Nowa Sól
country: Poland
facility: Twoja Przychodnia - Centrum Medyczne Nowa Sol
city: Warsaw
country: Poland
facility: Mazowiecki Szpital Brodnowski
city: Barnaul
country: Russian Federation
facility: Multi-field Medical Clinic Anturium LLC
city: Kazan
country: Russian Federation
facility: Interregional Clinical Diagnostic Center
city: Kemerovo
country: Russian Federation
facility: Kemerovo Regional Clinical Hospital n.a. S.V. Belyaev
city: Moscow
country: Russian Federation
facility: Federal State Budget Institution "National Medical Research Center of Endocrinology" of the Ministry of Healthcare of the Russian Federation
city: Moscow
country: Russian Federation
facility: I.M. Sechenov Moscow First State Medical University
city: Novosibirsk
country: Russian Federation
facility: Novosibirsk State Regional Clinical Hospital
city: Orenburg
country: Russian Federation
facility: Orenburg Regional Clinical Hospital
city: Saint Petersburg
country: Russian Federation
facility: Almazov National Medical Research Centre
city: Tver
country: Russian Federation
facility: State Budget Healthcare Institution of the Tver Region (Regional Clinical Hospital)
city: Belgrade
country: Serbia
facility: Clinical Center of Serbia
Study officials:
References: