A Phase 2 interventional study of GHR-LRX in Acromegaly, sponsored by Ionis Pharmaceuticals, Inc.. Completed at 22 sites in 10 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2024-10-03.
Sponsored by Ionis Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
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.
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.
202 studies on the registry are indexed under Acromegaly; 19 are open to participants now.
This study's enrollment of 34 is below the median of 43 across 115 interventional studies indexed under Acromegaly.
Browse Acromegaly studies →Ionis Pharmaceuticals, Inc. is the lead sponsor of 118 studies on the registry; 10 are open to participants now.
Of its 49 completed or terminated interventional studies of FDA-regulated products, 22 (45%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Have had pituitary surgery (e.g. transsphenoidal) unless there was a 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
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Drug: GHR-LRX
Participants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
Drug: GHR-LRX
GHR-LRX was administered by SC injection.
Also known as: ISIS 766720
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.
Time frame: Baseline to Week 27
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).
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)
Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's ULN.
Time frame: 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.
Time frame: 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.
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
Participants were enrolled at 22 investigational sites in United States of America, Estonia, Hungary, Italy, Latvia, Lithuania, Poland, Romania, Russia, and Serbia from 4 January 2021 to 18Jan2022.
| Milestone | GHR-LRX 120 mg | GHR-LRX 160 mg |
|---|---|---|
| Started | 18 | 16 |
| Per protocol set (initial dose) | 18 | 14 |
| Increased dose from initial 120mg to maximum dose 160mg (week 17 to week 45) | 15 | 0 |
| Completed | 5 | 3 |
| Not completed | 13 | 13 |
| Withdrew: Investigator judgement | 3 | 4 |
| Withdrew: Voluntary withdrawal | 1 | 0 |
| Withdrew: Pregnancy | 0 | 1 |
| Withdrew: Adverse event or serious adverse event | 1 | 3 |
| Withdrew: Reason not specified | 8 | 5 |
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.
| Percent change | GHR-LRX 120 mg (Per Protocol Set) | GHR-LRX 160 mg (Per Protocol Set) | GHR-LRX Total (Per Protocol Set) |
|---|---|---|---|
| Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27 | -6.20 ± 10.803 | -7.16 ± 20.100 | -7.09 ± 19.402 |
Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's upper limit of normal (ULN).
| percentage of participants | GHR-LRX 120 mg | GHR-LRX 160 mg | GHR-LRX Total |
|---|---|---|---|
| Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27) | 0 (0.0 to 84.2) | 0 (0.0 to 14.2) | 0 (0.0 to 13.2) |
Normalized IGF-1 level is defined as the ratio of the serum IGF-1 level and the participant's ULN.
| percentage of participants | GHR-LRX 120 mg | GHR-LRX 160 mg | GHR-LRX Total |
|---|---|---|---|
| Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27) | 0 (0.0 to 84.2) | 0 (0.0 to 14.2) | 0 (0.0 to 13.2) |
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative change from baseline indicated improvement.
| nanograms per milliliter (ng/mL) | GHR-LRX 120 mg | GHR-LRX 160 mg | GHR-LRX Total |
|---|---|---|---|
| At Week 3 | -30.00 ± 44.908 | 5.03 ± 49.932 | 1.74 ± 49.896 |
| At Week 5 | -50.67 ± 101.186 | -45.53 ± 87.817 | -46.01 ± 87.341 |
| Week 7 | -84.33 ± 133.829 | -39.46 ± 80.258 | -43.66 ± 84.558 |
| Week 9 | -109.00 ± 111.959 | -25.28 ± 93.924 | -33.13 ± 96.909 |
| Week 11 | -157.67 ± 144.923 | -14.28 ± 64.463 | -27.73 ± 83.134 |
| Week 13 | -89.67 ± 130.989 | -38.01 ± 68.885 | -42.85 ± 75.013 |
| Week 15 | -112.00 ± 112.618 | -28.97 ± 79.942 | -37.28 ± 85.124 |
| Week 17 | -89.33 ± 136.433 | 136.433 ± 88.212 | -41.82 ± 92.034 |
| Week 21 | -123.25 ± 136.118 | -51.91 ± 92.746 | -57.39 ± 95.030 |
| Week 25 | -99.75 ± 126.926 | -36.99 ± 78.236 | -41.82 ± 81.034 |
| Week 27 | -58.75 ± 87.328 | -49.45 ± 112.579 | -50.16 ± 109.415 |
| Week 29 | -90.25 ± 126.219 | -40.25 ± 80.174 | -44.25 ± 82.145 |
| Week 33 | -75.25 ± 110.662 | -53.12 ± 72.494 | -54.89 ± 73.247 |
| Week 37 | -98.75 ± 179.252 | -32.55 ± 107.499 | -37.85 ± 110.760 |
| Week 41 | -93.25 ± 146.018 | -43.12 ± 97.911 | -47.13 ± 99.341 |
| Week 45 | -86.75 ± 175.009 | -52.80 ± 110.595 | -55.75 ± 112.282 |
| Week 49 | -81.75 ± 109.955 | -64.59 ± 105.493 | -66.23 ± 103.184 |
| Week 53 | -90.25 ± 158.745 | -68.71 ± 81.822 | -70.86 ± 85.793 |
| Week 57 | -73.75 ± 91.570 | -59.43 ± 90.080 | -60.93 ± 87.744 |
| Week 61 | -125.75 ± 199.051 | -83.08 ± 112.428 | -88.10 ± 117.206 |
| Week 65 | 74.00 ± NA | -103.30 ± 102.760 | -88.52 ± 110.540 |
| Week 69 | -22.00 ± NA | -85.59 ± 53.018 | -78.53 ± 53.934 |
| Week 73 | -1.00 ± NA | -91.18 ± 79.166 | -79.91 ± 79.928 |
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. A negative percent change from Baseline indicated improvement.
| percent change | GHR-LRX 120 mg | GHR-LRX 160 mg | GHR-LRX Total |
|---|---|---|---|
| At Week 3 | -3.73 ± 5.072 | 2.64 ± 11.936 | 2.04 ± 11.571 |
| At Week 5 | -5.67 ± 12.296 | -8.28 ± 18.158 | -8.04 ± 17.554 |
| At Week 7 | -9.57 ± 16.297 | -7.16 ± 16.637 | -7.39 ± 16.360 |
| At Week 9 | -17.43 ± 11.285 | -2.95 ± 16.995 | -4.31 ± 16.955 |
| At Week 11 | -26.56 ± 15.966 | -1.15 ± 16.235 | -3.54 ± 17.638 |
| At Week 13 | -10.32 ± 17.934 | -6.35 ± 16.837 | -6.72 ± 16.679 |
| At Week 15 | -17.81 ± 13.377 | -5.40 ± 21.168 | -6.64 ± 20.698 |
| At Week 17 | -9.36 ± 20.529 | -4.59 ± 20.631 | -5.04 ± 20.338 |
| At Week 21 | -19.07 ± 8.695 | -9.98 ± 22.994 | -10.68 ± 22.261 |
| At Week 25 | -13.28 ± 12.010 | -6.58 ± 16.164 | -7.10 ± 15.794 |
| At Week 27 | -6.20 ± 10.803 | -7.16 ± 20.100 | -7.09 ± 19.402 |
| At Week 29 | -10.55 ± 14.242 | -7.92 ± 18.173 | -8.13 ± 17.656 |
| At Week 33 | -8.10 ± 13.484 | -10.10 ± 16.351 | -9.94 ± 15.905 |
| At Week 37 | -6.25 ± 27.791 | -4.29 ± 25.161 | -4.45 ± 24.755 |
| At Week 41 | -8.89 ± 19.345 | -9.01 ± 23.250 | -9.00 ± 22.608 |
| At Week 45 | -3.24 ± 29.617 | -8.26 ± 23.791 | -7.82 ± 23.590 |
| At Week 49 | -10.12 ± 11.603 | -12.83 ± 22.932 | -12.57 ± 21.924 |
| At Week 53 | -6.37 ± 23.892 | -14.81 ± 19.811 | -13.97 ± 19.697 |
| At Week 57 | -10.11 ± 8.205 | -10.50 ± 19.795 | -10.46 ± 18.763 |
| At Week 61 | -11.72 ± 26.723 | -18.94 ± 25.675 | -18.09 ± 25.044 |
| At Week 65 | 35.41 ± NA | -21.36 ± 25.392 | -16.63 ± 29.235 |
| At Week 69 | -10.53 ± NA | -23.32 ± 14.391 | -21.89 ± 14.120 |
| At Week 73 | -0.48 ± NA | -22.62 ± 20.556 | -19.85 ± 20.578 |
Collected over From Day 1 up to the end of study follow-up (up to week 121).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GHR-LRX 120 mg (Safety Set) | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| GHR-LRX 160 mg (Safety Set) | 0/31 (0%) | 2/31 (6.5%) | 26/31 (83.9%) |
| GHR-LRX Total (Safety Set) | 0/34 (0%) | 3/34 (8.8%) | 29/34 (85.3%) |
| Event | GHR-LRX 120 mg (Safety Set) | GHR-LRX 160 mg (Safety Set) | GHR-LRX Total (Safety Set) |
|---|---|---|---|
| COVID-19 pneumoniaInfections and infestations | 1/3 | 0/31 | 1/34 |
| Atrioventricular block second degreeCardiac disorders | 0/3 | 1/31 | 1/34 |
| Cholecystitis chronicHepatobiliary disorders | 0/3 | 1/31 | 1/34 |
| Event | GHR-LRX 120 mg (Safety Set) | GHR-LRX 160 mg (Safety Set) | GHR-LRX Total (Safety Set) |
|---|---|---|---|
| Renal cystRenal and urinary disorders | 2/3 | 0/31 | 2/34 |
| HeadacheNervous system disorders | 0/3 | 11/31 | 11/34 |
| COVID-19Infections and infestations | 1/3 | 6/31 | 7/34 |
| Respiratory tract infection viralInfections and infestations | 1/3 | 3/31 | 4/34 |
| Pyelonephritis chronicInfections and infestations | 1/3 | 0/31 | 1/34 |
| TremorNervous system disorders | 1/3 | 0/31 | 1/34 |
| NauseaGastrointestinal disorders | 1/3 | 4/31 | 5/34 |
| FlatulenceGastrointestinal disorders | 1/3 | 1/31 | 2/34 |
| HaemorrhoidsGastrointestinal disorders | 1/3 | 1/31 | 2/34 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/3 | 3/31 | 4/34 |
Safety analysis set included all participants who were randomized and received at least 1 dose of GHR-LRX
| Age, Continuous(Years) | GHR-LRX 120 mg | GHR-LRX 160 mg | Total |
|---|---|---|---|
| Mean | 63.0 ± 3.46 | 51.9 ± 11.76 | 52.9 ± 11.68 |
| Sex: Female, Male(Participants) | GHR-LRX 120 mg | GHR-LRX 160 mg | Total |
|---|---|---|---|
| Female | 1 | 16 | 17 |
| Male | 2 | 15 | 17 |
| Ethnicity (NIH/OMB)(Participants) | GHR-LRX 120 mg | GHR-LRX 160 mg | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 3 | 31 | 34 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | GHR-LRX 120 mg | GHR-LRX 160 mg | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 3 | 30 | 33 |
| More than one race | 0 | 1 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jan 2021 to primary completion in Jul 2022, 18 months.
Results were first posted in Oct 2024, 2 years 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.