Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

Phase 2

Completed

• Conditions: Acromegaly
• Interventions: Drug: IONIS GHR-LRx; Drug: Somatostatin Receptor Ligand (SRL)

• Registration Number: NCT03967249
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

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.

Study Timeline

• Study First Submitted: 2019-05-23
• Study First Submitted QC: 2019-05-28
• Study First Posted: 2019-05-30
• Study Start: 2019-07-25
• Primary Completion: 2022-03-31
• Study Completion: 2022-07-07
• Disposition First Submitted: 2022-09-14
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-17
• Disposition First Posted: 2023-03-22
• Last Update Posted: 2023-03-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
IONIS GHR-LRx + Somatostatin Receptor Ligand (SRL)	IONIS GHR-LRx	IONIS GHR-LRx (as per dose in previous study) will be administered subcutaneously once every 28 days for 53 weeks.
IONIS GHR-LRx + Somatostatin Receptor Ligand (SRL)	Somatostatin Receptor Ligand (SRL)	IONIS GHR-LRx (as per dose in previous study) will be administered subcutaneously once every 28 days for 53 weeks.

Primary Outcome Measures

Name	Time	Method
The Incidence of Adverse Events	Up to approximately 16 months	Incidence of adverse events (AEs), serious AEs, treatment related AEs, AEs leading to withdrawal

Secondary Outcome Measures

Name	Time	Method
Percent Change from Baselines in Insulin-like Growth Factor I (IGF-1) Levels	Baseline and at Week 26 and Week 53
Percentage of Participants who Begin Other Acromegaly Medication	Up to approximately 16 months
Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.0 Times of Gender and Age-Adjusted Upper Limits	Week 26 and at 28 days after the Week 53 dose
Time From First Dose of IONIS GHR-LRx in this open label extension (CS3) to Date of Initiation of Other Acromegaly Medications	Up to approximately 16 months
Percentage of Participants who Achieve Normalized IGF-1 Levels to Within 1.2 Times of Gender and Age-Adjusted Upper Limits	Week 26 and at 28 days after the Week 53 dose

Trial Locations

Locations (22)

Mazowiecki Szpital Brodnowski; 🇵🇱 Warsaw, Poland

Interregional Clinical Diagnostic Center; 🇷🇺 Kazan, Russian Federation

Federal State Budget Institution "National Medical Research Center of Endocrinology" of the Ministry of Healthcare of the Russian Federation; 🇷🇺 Moscow, Russian Federation

Clinical Center of Serbia; 🇷🇸 Belgrade, Serbia

Memorial Sloan Kettering Cancer Center; 🇺🇸 New York, New York, United States

Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos - Endocrinology clinic; 🇱🇹 Kaunas, Lithuania

Vaidoto Urbanaviciaus Individuali imone - Endokrinologijos klinika; 🇱🇹 Vilnius, Lithuania

Magyar Honvedseg Allami Egeszsegugyi Kozpont, II. sz Belgyogyaszat Osztaly; 🇭🇺 Budapest, Hungary

Centrum Nowoczesnych Terapii "Dobry Lekarz" Sp. z o. o.; 🇵🇱 Kraków, Poland

Twoja Przychodnia - Centrum Medyczne Nowa Sol; 🇵🇱 Nowa Sól, Poland

Multi-field Medical Clinic Anturium LLC; 🇷🇺 Barnaul, Russian Federation

I.M. Sechenov Moscow First State Medical University; 🇷🇺 Moscow, Russian Federation

Novosibirsk State Regional Clinical Hospital; 🇷🇺 Novosibirsk, Russian Federation

Kemerovo Regional Clinical Hospital n.a. S.V. Belyaev; 🇷🇺 Kemerovo, Russian Federation

Orenburg Regional Clinical Hospital; 🇷🇺 Orenburg, Russian Federation

State Budget Healthcare Institution of the Tver Region (Regional Clinical Hospital); 🇷🇺 Tver, Russian Federation

Almazov National Medical Research Centre; 🇷🇺 Saint Petersburg, Russian Federation

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Northwestern University; 🇺🇸 Evanston, Illinois, United States

Palm Research Center, Inc.; 🇺🇸 Las Vegas, Nevada, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States