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

NCT ID: NCT03548415

Last Updated: 2022-11-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-09-13
• Study Completion Date: 2021-04-02

Brief Summary

The purpose of this study was to assess the safety, tolerability, and efficacy of IONIS-GHR-LRx in up to 60 participants with acromegaly.

Detailed Description

This short-term study assessed changes in serum insulin-like growth factor 1 (IGF-1) over a 16-week treatment period in a participant population diagnosed with acromegaly being treated with long-acting somatostatin receptor ligands (SRL).

Conditions

Acromegaly

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo

Participants received placebo by subcutaneous injection (SC) once every 4 weeks for 16 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo administered subcutaneously.

Cohort A: IONIS GHR-LRx, 60 mg

Participants received IONIS GHR-LRx, 60 milligrams (mg), SC, once every 4 weeks for 16 weeks.

Group Type: EXPERIMENTAL

IONIS-GHR-LRx

Intervention Type: DRUG

IONIS GHR-LRx administered subcutaneously.

Cohort B: IONIS GHR-LRx, 80 mg

Participants received IONIS GHR-LRx, 80 mg, SC, once every 4 weeks for 16 weeks.

Group Type: EXPERIMENTAL

IONIS-GHR-LRx

Intervention Type: DRUG

IONIS GHR-LRx administered subcutaneously.

Cohort C: IONIS GHR-LRx, 120 mg

Participants received IONIS GHR-LRx, 120 mg, SC, once every 4 weeks for 16 weeks.

Group Type: EXPERIMENTAL

IONIS-GHR-LRx

Intervention Type: DRUG

IONIS GHR-LRx administered subcutaneously.

Cohort D: IONIS GHR-LRx, 160 mg

Participants received IONIS GHR-LRx, 160 mg, SC, once every 4 weeks for 16 weeks.

Group Type: EXPERIMENTAL

IONIS-GHR-LRx

Intervention Type: DRUG

IONIS GHR-LRx administered subcutaneously.

Interventions

IONIS-GHR-LRx

IONIS GHR-LRx administered subcutaneously.

Intervention Type: DRUG

Placebo

Placebo administered subcutaneously.

Intervention Type: DRUG

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. Participants 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 insulin-like growth factor 1 (IGF-1) (performed at central lab) between 1.3 to 5 x upper limit of normal (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. Participants who received surgery for pituitary adenoma within the last 6 months before the trial, or planning to receive surgery during the trial

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

3. Participants with pituitary tumor that, per Investigator judgement, is worsening as assessed by pituitary/sellar magnetic resonance imaging (MRI) protocol at Screen or within 6 months of screening

4. Evidence of decompensated cardiac function per medical judgement and/or New York Heart Association (NYHA) class 3 or 4

5. Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment

6. Participants 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. Participants 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. Participants taking glucagon-like peptide 1 (GLP-1) agonists or insulin can be allowed with prior consultation with the Sponsor Medical Monitor

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Alabama at Birmingham (UAB)

Birmingham, Alabama, United States

St. Joseph's Hospital and Medical Center

Phoenix, Arizona, United States

Northwestern University

Chicago, Illinois, United States

Palm Research Center, Inc.

Las Vegas, Nevada, United States

Palm Research Center, Inc.

Las Vegas, Nevada, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Oregon Health & Science University (OHSU)

Portland, Oregon, United States

Magyar Honvedseg Allami Egeszsegugyi Kozpont, II. sz Belgyogyaszat Osztaly

Budapest, , Hungary

Debreceni Egyetem Klinikai Kozpont

Debrecen, , Hungary

Szeged University - Szent-Gyorgyi Albert Clinical Center - I. Belgyógyászati Klinika (Internal Medicine)

Szeged, , Hungary

Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos - Hospital of Oncology

Kaunas, , Lithuania

Vaidoto Urbanaviciaus Individuali imone - Endokrinologijos klinika

Vilnius, , Lithuania

B_Serwis Popenda Sp. J. Specjalistyczna Przychodnia Lekarsk

Chorzów, , Poland

Uniwersyteckie Centrum Kliniczne im. Prof. K. Gibinskiego Slaskiego Uniwersytetu Medycznego w Katowicach

Katowice, , Poland

Centrum Nowoczesnych Terapii Dobry Lekarz Sp. z o. o.

Krakow, , Poland

Twoja Przychodnia - Centrum Medyczne Nowa Sol

Nowa Sól, , Poland

Mazowiecki Szpital Brodnowski - Zespol Oddzialow Chorob Wewnetrznych, Endokrynologii i Diabetologii

Warsaw, , Poland

Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.

Wroclaw, , Poland

Centrul Medical Unirea - Bucuresti, Endocrinologie

Bucharest, , Romania

Spitalul Clinic Judetean de Urgenta Cluj - Napoca

Cluj-Napoca, , Romania

Spitalul Clinic Judetean Mures

Târgu Mureş, , Romania

Spitalul Clinic Judetean de Urgenta Timisoara

Timișoara, , Romania

Multi-field Medical Clinic Anturium LLC

Barnaul, , Russia

Interregional Clinical Diagnostic Center

Kazan', , Russia

Kuzbass Clinical Hospital n.a. S.V. Belyaev

Kemerovo, , Russia

Federal State Budget Institution "National Medical Research Center of Endocrinology" of the Ministry of Healthcare of the Russian Federation

Moscow, , Russia

I.M. Sechenov Moscow First State Medical University

Moscow, , Russia

Novosibirsk State Regional Clinical Hospital

Novosibirsk, , Russia

Orenburg Regional Clinical Hospital, Endocrinology Department

Orenburg, , Russia

Rostov State Medical University

Rostov-on-Don, , Russia

Almazov National Medical Research Centre

Saint Petersburg, , Russia

State Budget Healthcare Institution of the Tver Region

Tver', , Russia

Clinical Center of Serbia

Belgrade, , Serbia

Countries

United States; Hungary; Lithuania; Poland; Romania; Russia; Serbia

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2017-004259-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ISIS 766720-CS2

Identifier Type: -

Identifier Source: org_study_id