Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Patients With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

Brief Title

Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Patients With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

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)

Brief Summary

      The purpose is to assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in up to 60
      Patients with Acromegaly
    

Detailed Description

      This short-term study will assess changes in serum insulin-like growth factor 1 (IGF-1) over
      a 16-week treatment period in a patient population diagnosed with Acromegaly being treated
      with Long-acting Somatostatin Receptor Ligands (SRL)
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Efficacy of IONIS GHR-LRx as percent change in serum IGF-1 from Baseline to 28 days after last dose

Secondary Outcome

 Proportion of patients achieving normalized IGF-1 levels to within 1.2 times of gender and age limits at 28 days after last dose

Condition

Acromegaly

Intervention

IONIS-GHR-LRx

Study Arms / Comparison Groups

 IONIS-GHR-LRx

Description:  Single Dose of IONIS GHR-LRx administered subcutaneously once every 28 days for 16 weeks

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

43

Start Date

September 13, 2018

Completion Date

April 2021

Primary Completion Date

February 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Males or females with documented diagnosis of Acromegaly, aged 18-75 years old
             (inclusive) at the time of informed consent

          2. 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.

          3. At Screening, serum IGF-1 (performed at central lab) between 1.3 to 5 x ULN,
             inclusive, adjusted for age and sex

          4. 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:

          1. Patients who received surgery for pituitary adenoma within the last 6 months before
             the trial, or planning to receive surgery during the trial

          2. Patients who received radiotherapy for pituitary adenoma within the last 3 years
             before the trial, and/or planning to receive radiotherapy during the trial

          3. 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

          4. Evidence of decompensated cardiac function per medical judgement and/or NYHA class 3
             or 4

          5. Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment

          6. 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.

          7. 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

          8. Patients taking GLP-1 agonists or insulin can be allowed with prior consultation with
             the Sponsor Medical Monitor
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Austria

Location Countries

Austria

NCT ID

NCT03548415

Organization ID

ISIS 766720-CS2

Secondary IDs

2017-004259-22

Responsible Party

Sponsor

Study Sponsor

Ionis Pharmaceuticals, Inc.

Study Sponsor

, , 

Verification Date

January 2021