AcromegalyPhase 2NCT04522180
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
A Phase 2 study testing GHR-LRX in people with Acromegaly, run by Ionis Pharmaceuticals, Inc. at 22 sites in 10 countries.
Trial summary
The purpose of this study was to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acromegaly
- What is being tested?
- GHR-LRX
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 22 sitesin 10 countries
- Study dates
- 4 Jan 2021to 4 May 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females with a documented diagnosis of Acromegaly* who are 18 to 75 years old (inclusive) at the time of informed consent.
- Have had pituitary surgery (e.g. transsphenoidal) unless there was a contraindication to surgery and are either acromegaly medical treatment naïve, or who had not taken any other acromegaly medications prior to the screening visit as outlined below
- bromocriptine: 2 weeks
- cabergoline: 4 weeks
- quinagolide: 4 weeks
- octreotide daily injection (SC) or oral formulation: 4 weeks
Show all 12Show fewer
- pegvisomant: 4 weeks
- octreotide LAR: 3 months
- pasireotide LAR: 4 months
- lanreotide (all formulations): 3 months
- At Screening, serum IGF-1 (performed at the central lab) between 1.3 to 5 × 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
Who cannot join
- Participants who received surgery for pituitary adenoma within the last 3 months before the trial, and/or planning to receive surgery during the trial
- Participants who received radiotherapy for pituitary adenoma within the last 2 years before the trial, and/or planning to receive radiotherapy during the trial
- Participants with a pituitary tumor that, per Investigator judgment, is worsening (e.g., either growing or at risk of compressing or abutting the optic chiasm or other vital structures) as assessed by pituitary/sellar MRI protocol at Screening or within 3 months of Screening. CT scan is allowed if MRI is contraindicated
- Evidence of decompensated cardiac function per medical judgement and/or New York Heart Association (NYHA) Class 3 or 4
- Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment
- Symptomatic cholelithiasis, and/or choledocholithiasis
Trial locations and contacts
22 sites in 10 countries.
22 sites in 10 countries
United States7 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Cleveland Clinic
Cleveland, Ohio, 44195
Endocrinology Associates, Inc
Columbus, Ohio, 43201
Palm Research Center Inc.
Las Vegas, Nevada, 89148
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Oregon Health & Science University (OHSU)
Portland, Oregon, 97239
Poland3 sites
Zespol Oddzialow Chorob Wewnetrznych, Endokrynologii i Diabetologii
Warsaw, 03-242
Uniwersytecki Szpital Kliniczny, im. Jana Mikulicza-Radeckiego we Wroclawiu,
Wroclaw, 50-556
Centrum Badan Klinicznych Piotr Napora Lekarze Sp. p.
Wroclaw, 51-162
Estonia2 sites
East-Tallinn Central Hospital
Tallinn, 10138
Tartu University Hospital
Tartu, 50406
Italy2 sites
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, 00168
Lithuania2 sites
Vaidoto Urbanaviciaus Individuali imone
Alytus, 63246
Hospital of Lithuanian University of Health Sciences (LSMU) Kauno klinikos
Kaunas, 50161
Serbia2 sites
Clinical Center of Serbia
Belgrade, 11000
Clinical Center of Vojvodina
Novi Sad, 21000
Romania1 site
Centrul Medical Unirea Bucuresti, Endocrinologie
Bucharest, 060044
Russia1 site
I.M. Sechenov Moscow First State Medical University
Moscow, 119146
About this study
Official title: An Open Label, Randomized, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor, Administered Monthly as Monotherapy in Patients With Acromegaly
The purpose of this study was to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.
Read the detailed description
This was a multi-center, open-label, randomized, Phase 2 study of IONIS-GHR-LRx in up to 40 participants with acromegaly. Participants were randomized to 1 of 2 treatment groups to receive IONIS GHR-LRx monthly for 73 weeks. At the end of 73 weeks, participants entered a 14-week post-treatment (PT) evaluation period.
- Conditions
- Acromegaly
- Being tested
- GHR-LRX
- Keywords
- acromegaly, IONIS-GHR-LRx
- Medical subject headings
- Acromegaly
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GHR-LRXDrug
Also called ISIS 766720
GHR-LRX was administered by SC injection.
| Study group | Role | What they receive |
|---|---|---|
| GHR-LRX 120 mg | Experimental | Participants received GHR-LRX 120 mg subcutaneous (SC) injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3). Receives: Drug: GHR-LRX |
| GHR-LRX 160 mg | Experimental | Participants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3). Receives: Drug: GHR-LRX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. Baseline of IGF-1 is defined as the average value of Screening and Day 1. A negative percent change from Baseline indicated improvement.
Measured: Baseline to Week 27
Secondary outcomes (4)
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27)
Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's upper limit of normal (ULN).
Measured: At Week 27
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27)
Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's ULN.
Measured: At Week 27
Change From Baseline in Serum IGF-1 Over Time
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative change from baseline indicated improvement.
Measured: Up to approximately 80 weeks
Percent Change From Baseline in Serum IGF-1 Over Time
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative percent change from Baseline indicated improvement.
Measured: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 21, 25, 27, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2020
- Study start4 Jan 2021
- Primary completion15 Jul 2022
- Study completion4 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AcromegalyStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04522180
- Sponsor's protocol ID
- ISIS 766720-CS5
- Other IDs
- 2020-000675-20, NCT04522180
- Status last verified
- Sep 2024
- First submitted
- 18 Aug 2020
- Last updated
- 3 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Randomized, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx, an Antisense Inhibitor of the Growth Hormone Receptor, Administered Monthly as Monotherapy in Patients With Acromegaly
- ClinicalTrials.gov ID
- NCT04522180
- Acronym / sponsor ID
- ISIS 766720-CS5
- Conditions
- Acromegaly
- Keywords
- acromegaly, IONIS-GHR-LRx
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Jan 2021 (actual)
- Primary completion
- 15 Jul 2022 (actual)
- Study completion
- 4 May 2023 (actual)
- First posted
- 21 Aug 2020 (actual)
- Results
- Posted 3 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.
Detailed description
This was a multi-center, open-label, randomized, Phase 2 study of IONIS-GHR-LRx in up to 40 participants with acromegaly. Participants were randomized to 1 of 2 treatment groups to receive IONIS GHR-LRx monthly for 73 weeks. At the end of 73 weeks, participants entered a 14-week post-treatment (PT) evaluation period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GHR-LRX 120 mg | Experimental | Drug: GHR-LRXParticipants received GHR-LRX 120 mg subcutaneous (SC) injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3). |
| GHR-LRX 160 mg | Experimental | Drug: GHR-LRXParticipants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Males or females with a documented diagnosis of Acromegaly* who are 18 to 75 years old (inclusive) at the time of informed consent.
- Have had pituitary surgery (e.g. transsphenoidal) unless there was a contraindication to surgery and are either acromegaly medical treatment naïve, or who had not taken any other acromegaly medications prior to the screening visit as outlined below
- At Screening, serum IGF-1 (performed at the central lab) between 1.3 to 5 × 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
Key exclusion criteria (6 listed)
- Participants who received surgery for pituitary adenoma within the last 3 months before the trial, and/or planning to receive surgery during the trial
- Participants who received radiotherapy for pituitary adenoma within the last 2 years before the trial, and/or planning to receive radiotherapy during the trial
- Participants with a pituitary tumor that, per Investigator judgment, is worsening (e.g., either growing or at risk of compressing or abutting the optic chiasm or other vital structures) as assessed by pituitary/sellar MRI protocol at Screening or within 3 months of Screening. CT scan is allowed if MRI is contraindicated
- Evidence of decompensated cardiac function per medical judgement and/or New York Heart Association (NYHA) Class 3 or 4
- Clinical evidence of symptomatic hyperprolactinemia that would necessitate treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GHR-LRXAlso: ISIS 766720GHR-LRX was administered by SC injection. | Drug | GHR-LRX 120 mg, GHR-LRX 160 mg |
Primary outcome
Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27
Time frame: Baseline to Week 27
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. Baseline of IGF-1 is defined as the average value of Screening and Day 1. A negative percent change from Baseline indicated improvement.
4 secondary outcomes
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27)
Time frame: At Week 27
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27)
Time frame: At Week 27
Change From Baseline in Serum IGF-1 Over Time
Time frame: Up to approximately 80 weeks
Percent Change From Baseline in Serum IGF-1 Over Time
Time frame: Baseline, Week 3, 5, 7, 9, 11, 13, 15, 17, 21, 25, 27, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
22 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GHR-LRX in people with Acromegaly, run by Ionis Pharmaceuticals, Inc. at 22 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Poland, Estonia, Italy, Lithuania and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27 (time frame: Baseline to Week 27). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2024.