Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Patients With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
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ClinicalTrials.gov Identifier: NCT03548415 |
Recruitment Status :
Completed
First Posted : June 7, 2018
Last Update Posted : March 29, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Acromegaly | Drug: IONIS-GHR-LRx Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 43 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Double-Blind, Placebo-Controlled, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of ISIS 766720 (IONIS-GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor) Administered Once Every 28 Days for 16 Weeks in Patients With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands (SRL) |
Actual Study Start Date : | September 13, 2018 |
Actual Primary Completion Date : | April 2, 2021 |
Actual Study Completion Date : | April 2, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: IONIS-GHR-LRx
Single Dose of IONIS GHR-LRx administered subcutaneously once every 28 days for 16 weeks
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Drug: IONIS-GHR-LRx
Single Dose of IONIS GHR-LRx administered subcutaneously once every 28 days for 16 weeks |
Placebo Comparator: Placebo
Placebo (sterile saline 0.9%) Calculated volume to match active comparator administered subcutaneously every 28 days for 16 weeks
|
Drug: Placebo
Placebo (sterile saline 0.9%) Calculated volume to match active comparator administered subcutaneously every 28 days for 16 weeks |
- Efficacy of IONIS GHR-LRx as percent change in serum IGF-1 from Baseline to 28 days after last dose [ Time Frame: Baseline and 28 days after the last dose ]
- Incidence and severity of adverse events that are related to treatment with IONIS GHR-LRx. [ Time Frame: Up to 211 days ]
- Proportion of patients achieving normalized IGF-1 levels to within 1.2 times of gender and age limits at 28 days after last dose [ Time Frame: 28 days after the last dose ]
- Proportion of patients achieving normalized IGF-1 levels to within 1.0 times of gender and age limits at 28 days after last dose [ Time Frame: 28 days after the last dose ]
- Change from Baseline in serum IGF-1 over time [ Time Frame: Up to 31 weeks ]
- Percent change from Baseline in Serum IGF-1 over time [ Time Frame: Up to 31 weeks ]

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males or females with documented diagnosis of Acromegaly, aged 18-75 years old (inclusive) at the time of informed consent
- Patients must be on stable maximum or maximally tolerated dose of SRL (Lanreotide Autogel or Octreotide LAR, per treating physician judgment) every 28 days for a minimum of 3 months prior to screening and will be required to continue their stable dose of SRL throughout the study. Prior use of other medications for treating acromegaly is allowed but not within 6 weeks of screening.
- At Screening, serum IGF-1 (performed at central lab) between 1.3 to 5 x ULN, inclusive, adjusted for age and sex
- Females must be non-pregnant and non-lactating, and either surgically sterile, post-menopausal, abstinent, or using 1 highly effective method of birth control
Exclusion Criteria:
- Patients who received surgery for pituitary adenoma within the last 6 months before the trial, or planning to receive surgery during the trial
- Patients who received radiotherapy for pituitary adenoma within the last 3 years before the trial, and/or planning to receive radiotherapy during the trial
- Patients with pituitary tumor that, per Investigator judgement, is worsening as assessed by pituitary/sellar MRI protocol at Screen or within 6 months of screening
- Evidence of decompensated cardiac function per medical judgement and/or NYHA class 3 or 4
- Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment
- Patients may not have chronic systemic use of glucocorticoids, weight loss medications or participate in weight loss programs within 2 months before randomization and during study participation.
- Patients on anti-diabetes medication or estrogen containing medications must be on a stable dose and regimen for >= 3 months prior to screening and throughout the trial
- Patients taking GLP-1 agonists or insulin can be allowed with prior consultation with the Sponsor Medical Monitor

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): NCT03548415

Responsible Party: | Ionis Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT03548415 |
Other Study ID Numbers: |
ISIS 766720-CS2 2017-004259-22 ( EudraCT Number ) |
First Posted: | June 7, 2018 Key Record Dates |
Last Update Posted: | March 29, 2022 |
Last Verified: | March 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Acromegaly, IONIS-GHR-LRx |
Acromegaly Bone Diseases, Endocrine Bone Diseases Musculoskeletal Diseases Hyperpituitarism Pituitary Diseases |
Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Endocrine System Diseases |