Acromegaly
Conditions
Keywords
Acromegaly, IONIS-GHR-LRx
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.
Interventions
DRUGIONIS GHR-LRx
Participants will receive IONIS GHR-LRx by subcutaneous injection.
DRUGSomatostatin Receptor Ligand (SRL)
Participants will receive Somatostatin Receptor Ligand (lanreotide, octreotide) once monthly.
Sponsors
Ionis Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 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 |
| 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 |
| Percentage of Participants who Begin Other Acromegaly Medication | Up to approximately 16 months |
| 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 |
Countries
Hungary, Lithuania, Poland, Russia, Serbia, United States
Outcome results
None listed