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

Sponsor

Ionis Pharmaceuticals, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT03967249

Collaborator

(none)

Enrollment

Locations

Arm

35.4

Actual Duration (Months)

1.8

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Acromegaly	Drug: IONIS GHR-LRx Drug: SRL	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

39 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-Label Extension Trial of IONIS GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor Administered Monthly Subcutaneously to Patients With Acromegaly Being Treated With Long-Acting Somatostatin Receptor Ligands (SRL)

Actual Study Start Date :

Jul 25, 2019

Actual Primary Completion Date :

Mar 31, 2022

Actual Study Completion Date :

Jul 7, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: IONIS GHR-LRx + SRL IONIS GHR-LRx (as per dose in previous study) will be administered subcutaneously once every 28 days for 53 weeks.	Drug: IONIS GHR-LRx IONIS GHR-LRx subcutaneous injection Drug: SRL Participants will receive SRL once monthly

Arm

Intervention/Treatment

Experimental: IONIS GHR-LRx + SRL

IONIS GHR-LRx (as per dose in previous study) will be administered subcutaneously once every 28 days for 53 weeks.

Drug: IONIS GHR-LRx

IONIS GHR-LRx subcutaneous injection

Drug: SRL

Participants will receive SRL once monthly

Outcome Measures

Primary Outcome Measures

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

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alabama at Birmingham	Birmingham	Alabama	United States	35294
2	Northwestern University	Evanston	Illinois	United States	60208
3	Palm Research Center, Inc.	Las Vegas	Nevada	United States	89149
4	Memorial Sloan Kettering Cancer Center	New York	New York	United States	10065
5	Cleveland Clinic	Cleveland	Ohio	United States	44195
6	Oregon Health & Science University	Portland	Oregon	United States	97239
7	Magyar Honvedseg Allami Egeszsegugyi Kozpont, II. sz Belgyogyaszat Osztaly	Budapest		Hungary	1062
8	Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos - Endocrinology clinic	Kaunas		Lithuania	LT-50161
9	Vaidoto Urbanaviciaus Individuali imone - Endokrinologijos klinika	Vilnius		Lithuania	9112
10	Centrum Nowoczesnych Terapii "Dobry Lekarz" Sp. z o. o.	Kraków		Poland	31-011
11	Twoja Przychodnia - Centrum Medyczne Nowa Sol	Nowa Sól		Poland	67-100
12	Mazowiecki Szpital Brodnowski	Warsaw		Poland	03-242
13	Multi-field Medical Clinic Anturium LLC	Barnaul		Russian Federation	656043
14	Interregional Clinical Diagnostic Center	Kazan		Russian Federation	420101
15	Kemerovo Regional Clinical Hospital n.a. S.V. Belyaev	Kemerovo		Russian Federation	650066
16	Federal State Budget Institution "National Medical Research Center of Endocrinology" of the Ministry of Healthcare of the Russian Federation	Moscow		Russian Federation	117036
17	I.M. Sechenov Moscow First State Medical University	Moscow		Russian Federation	119991
18	Novosibirsk State Regional Clinical Hospital	Novosibirsk		Russian Federation	630087
19	Orenburg Regional Clinical Hospital	Orenburg		Russian Federation	460018
20	Almazov National Medical Research Centre	Saint Petersburg		Russian Federation	194156
21	State Budget Healthcare Institution of the Tver Region (Regional Clinical Hospital)	Tver		Russian Federation	170036
22	Clinical Center of Serbia	Belgrade		Serbia	11000

Sponsors and Collaborators

Ionis Pharmaceuticals, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Ionis Pharmaceuticals, Inc.

ClinicalTrials.gov Identifier:

NCT03967249

Other Study ID Numbers:

ISIS 766720-CS3
2019-000591-42

First Posted:

May 30, 2019

Last Update Posted:

Aug 8, 2022

Last Verified:

Aug 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Ionis Pharmaceuticals, Inc.

Acromegaly, IONIS-GHR-LRx

Additional relevant MeSH terms:

Acromegaly

Study Results

No Results Posted as of Aug 8, 2022