A Phase 1/2 interventional study of Efavaleukin Alfa in Chronic Graft Versus Host Disease cGVHD, sponsored by Amgen. Terminated at 14 sites in 4 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2023-11-22.
Sponsored by Amgen · Phase 1/2, Interventional, and Treatment
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Among 300 studies in Bronchiolitis Obliterans Syndrome with a site in United States — 48 open to participants now, 71 under way, 7 of them phase 3. Bronchiolitis Obliterans Syndrome · United States.
Among the 300 studies in Bronchiolitis Obliterans Syndrome with a site in United States, this one is the 148th largest by enrollment of the 260 interventional ones that give a count, is one of 13 that started in 2018, is one of 35 in phase 1/2, and was the 183rd registered.
714 studies on the registry are indexed under Bronchiolitis Obliterans Syndrome; 128 are open to participants now.
Browse Bronchiolitis Obliterans Syndrome studies →48 of the 300 studies in Bronchiolitis Obliterans Syndrome with a site in United States are open now. Recruiting first, the newest first:
City of Hope Medical Center · Sites in United States
Ages 18+
Sarah Nikiforow, MD · Sites in United States
Ages 2+
Incyte Corporation · Sites in United States, Belgium, Germany and 3 more countries
Ages 2–17
Fred Hutchinson Cancer Center · Sites in United States
Ages 18+
Novartis Pharmaceuticals · Sites in United States, Australia, Brazil and 14 more countries
Ages 1–18
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 100 Bronchiolitis Obliterans Syndrome studies recruiting now, 45 (45%) set no upper age limit and 45 (45%) stop at 75 or younger; 25 take children under 18; 12 take healthy volunteers.
This study: 18 Years to 100 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
300 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 65 · Phase 2 117 · Phase 3 33 · Phase 4 18
First in 2001; this study’s year is marked. 13 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 32 is below the median of 50 across 565 interventional studies indexed under Bronchiolitis Obliterans Syndrome.
Median enrollment 45 across the 260 interventional studies, 187 across the 39 observational. Of the 209 that completed or were terminated, 110 (53%) have results posted.
This tab as a report to print or save as PDF → With an account.
112 of 300 have an industry lead sponsor. The 10 most frequent:
Amgen is the lead sponsor of 1,047 studies on the registry; 51 are open to participants now.
Of its 246 completed or terminated interventional studies of FDA-regulated products, 166 (67%) have results posted.
Amgen has 4 studies filed under Bronchiolitis Obliterans Syndrome — fewer than ten, so there is no comparison to draw.
20 of the studies in Bronchiolitis Obliterans Syndrome with a site in United States that are under way reach primary completion within a year, 4 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01804686Phase 3Janssen Research & Development, LLCA Long-term Extension Study of PCI-32765 (Ibrutinib) | 2026-12-31 |
| NCT07395232Phase 3Dompé Farmaceutici S.p.AAn Efficacy and Safety Study of DFL24498 in the Treatment of AKC | 2027-03 |
| NCT02181257Phase 3Washington University School of MedicineExtracorporeal Photopheresis for Medicare Recipients of Lung Allografts | 2027-04 |
| NCT06585774Phase 3Incyte CorporationA Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease | 2027-09-28 |
| NCT06018766Steven Hays, MDLAM-001 in Lung Transplant Recipients With Bronchiolitis Obliterans Syndrome. | 2026-12 |
Add all 20 to your calendar (.ics) → With an account.
Among the 300 studies in Bronchiolitis Obliterans Syndrome with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
Phase 1b: To evaluate the safety and tolerability of multiple ascending doses of efavaleukin alfa in subjects with steroid refractory chronic graft versus host disease (cGVHD).
Phase 2: To evaluate the efficacy of efavaleukin alfa in subjects with steroid refractory cGVHD as measured by overall response rate (ORR) at 16 weeks according to the 2014 cGVHD NIH Consensus Criteria.
Due to early termination, the Phase 2 portion of this study was not conducted.
Inclusion Criteria Subjects are eligible to be included in the study only if all of the following criteria apply:
Exclusion Criteria Subjects are excluded from the study if any of the following criteria apply.
positive Quantiferon or T-SPOT test o a positive PPD and a history of Bacillus Calmette-Guérin vaccination are allowed with a negative Quantiferon or T-SPOT test and negative chest x-ray
Quantiferon or T-SPOT test are allowed if they have ALL of the following at screening:
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
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.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
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.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
Efavaleukin will be administered as multiple ascending doses (MAD) across cohorts 1-5. Each dosing cohort will consist of between 3 and 6 subjects who will receive efavaleukin alfa subcutaneously (SC) either every week or every 2 weeks plus protocol permitted background therapy for 52 weeks. At the discretion of the Sponsor, following discussion and agreement between the principal investigator and medical monitor, subjects responding to efavaleukin alfa (as assessed by the end of week 50), who wish to continue treatment, may continue to receive efavaleukin alfa treatment at their current dosing regimen.
Drug: Efavaleukin Alfa
The phase 2 portion of this study will be conducted as a single arm, multi-center, open label trial in subjects with steroid refractory chronic graft versus Host Disease (cGVHD). All subjects will receive the recommended phase 2 dose (RP2D) of efavaleukin alfa for up to 52 weeks plus protocol permitted background therapy for cGVHD. Due to early study termination the Phase 2 portion of the study was never opened.
Drug: Efavaleukin Alfa
Phase 1: This study will be conducted as a multiple ascending dose (MAD) study. Each dosing cohort will consist of between 3 and 6 subjects who will receive efavaleukin alfa subcutaneously (SC) either every week or every 2 weeks plus protocol permitted background therapy for 52 weeks. At the discretion of the Sponsor, following discussion and agreement between the principal investigator and medical monitor, subjects responding to efavaleukin alfa (as assessed by the end of week 50), who wish to continue treatment, may continue to receive efavaleukin alfa treatment at their current dosing regimen for up to an additional 208 weeks through an extended dosing period. All subjects who continue to receive efavaleukin alfa during the extended dosing period will be reevaluated every 6 months for their response to treatment. Phase 2: All subjects will receive the recommended phase 2 dose (RP2D) of efavaleukin alfa for 52 weeks.
Also known as: AMG 592
Phase 1b: Number of Participants Who Experienced a Dose-limiting Toxicity (DLT)
A DLT was defined as a: * Non-hematological toxicity ≥grade-4(per common terminology criteria for adverse events \[CTCAE\] v4.03) related to efavaleukin alfa. Non-hematological lab abnormalities without clinical significance weren't considered DLTs. * Hematological toxicity ≥grade 4 related to efavaleukin alfa defined as decreases in peripheral counts (absolute neutrophil count or platelets) persisting longer than 72 hrs, as measured by 2 separate results, that were not related to malignant disease relapse, infection, or other etiologies. * Constitutional events (ie, fever, fatigue) ≥grade 3 that were classified as serious adverse events by the investigator and related to efavaleukin alfa. * Infection is considered an expected complication of chronic graft versus host disease (cGVHD) and its treatment. Only grade 4 or 5 infections considered by the investigator to be related to efavaleukin alfa were reviewed by the dose level review meeting to determine whether it was considered a DLT.
Time frame: Up to 4 weeks after first dose of study drug administration
Phase 1b: Number of Participants Who Experienced a Treatment-related Adverse Event (AE)
A treatment-related AE was any untoward medical occurrence in a clinical study participant deemed to have a possibly causal relationship to the study treatment as determined by the investigator.
Time frame: Day 1 until the end of study; median (min, max) duration was 38.01 (3.27, 139.81) weeks
Phase 1b: Number of Participants Who Experienced a Treatment-emergent AE
A treatment-emergent AE was any untoward medical occurrence in a clinical study participant that occurred after first dose.
Time frame: Day 1 until the end of study; median (min, max) duration was 38.01 (3.27, 139.81) weeks
Phase 1b: Number of Participants Who Experienced a Treatment-emergent Serious AE
A treatment-emergent serious AE was any untoward medical occurrence in a clinical study participant that occurred after first dose that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or another medically important serious event.
Time frame: Day 1 until the end of study; median (min, max) duration was 38.01 (3.27, 139.81) weeks
Participants were enrolled into this study at sites in Belgium, France, Japan, and the United States.
| Milestone | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Started | 7 | 7 | 7 | 6 | 5 |
| Completed up to week 52 and opted for extended dosing | 2 | 1 | 2 | 3 | 0 |
| Completed up to week 52 and did not opt for extended dosing | 0 | 1 | 1 | 0 | 1 |
| Completed | 2 | 2 | 3 | 3 | 1 |
| Not completed | 5 | 5 | 4 | 3 | 4 |
| Withdrew: Protocol-specified criteria | 1 | 1 | 0 | 0 | 2 |
| Withdrew: Requirement for alternative therapy | 1 | 1 | 2 | 0 | 0 |
| Withdrew: Disease progression | 1 | 0 | 1 | 2 | 0 |
| Withdrew: Adverse event | 2 | 1 | 0 | 0 | 2 |
| Withdrew: Ineligibility determined | 0 | 1 | 0 | 0 | 0 |
| Withdrew: Death | 0 | 1 | 1 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 1 | 0 |
A DLT was defined as a: * Non-hematological toxicity ≥grade-4(per common terminology criteria for adverse events \[CTCAE\] v4.03) related to efavaleukin alfa. Non-hematological lab abnormalities without clinical significance weren't considered DLTs. * Hematological toxicity ≥grade 4 related to efavaleukin alfa defined as decreases in peripheral counts (absolute neutrophil count or platelets) persisting longer than 72 hrs, as measured by 2 separate results, that were not related to malignant disease relapse, infection, or other etiologies. * Constitutional events (ie, fever, fatigue) ≥grade 3 that were classified as serious adverse events by the investigator and related to efavaleukin alfa. * Infection is considered an expected complication of chronic graft versus host disease (cGVHD) and its treatment. Only grade 4 or 5 infections considered by the investigator to be related to efavaleukin alfa were reviewed by the dose level review meeting to determine whether it was considered a DLT.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Phase 1b: Number of Participants Who Experienced a Dose-limiting Toxicity (DLT) | 0 | 0 | 0 | 0 | 0 |
A treatment-related AE was any untoward medical occurrence in a clinical study participant deemed to have a possibly causal relationship to the study treatment as determined by the investigator.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Phase 1b: Number of Participants Who Experienced a Treatment-related Adverse Event (AE) | 5 | 5 | 6 | 6 | 4 |
A treatment-emergent AE was any untoward medical occurrence in a clinical study participant that occurred after first dose.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Phase 1b: Number of Participants Who Experienced a Treatment-emergent AE | 6 | 6 | 7 | 6 | 4 |
A treatment-emergent serious AE was any untoward medical occurrence in a clinical study participant that occurred after first dose that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or another medically important serious event.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Phase 1b: Number of Participants Who Experienced a Treatment-emergent Serious AE | 5 | 3 | 3 | 4 | 3 |
Collected over Mortality: from enrollment until the end of study; median (min, max) duration was 38.44 (3.56, 140.09) weeks. Serious and non-serious adverse events: from Day 1 until the end of study; median (min, max) duration was 38.01 (3.27, 139.81) weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 | 0/7 (0%) | 5/7 (71.4%) | 6/7 (85.7%) |
| Cohort 2 | 1/7 (14.3%) | 3/7 (42.9%) | 6/7 (85.7%) |
| Cohort 3 | 1/7 (14.3%) | 3/7 (42.9%) | 7/7 (100%) |
| Cohort 4 | 0/6 (0%) | 4/6 (66.7%) | 6/6 (100%) |
| Cohort 5 | 0/5 (0%) | 3/5 (60%) | 3/5 (60%) |
| Event | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Drug hypersensitivityImmune system disorders | 0/7 | 0/7 | 0/7 | 0/6 | 2/5 |
| PneumoniaInfections and infestations | 1/7 | 0/7 | 2/7 | 0/6 | 0/5 |
| Pseudomonal sepsisInfections and infestations | 0/7 | 0/7 | 0/7 | 0/6 | 1/5 |
| Capillary leak syndromeVascular disorders | 0/7 | 0/7 | 0/7 | 0/6 | 1/5 |
| CellulitisInfections and infestations | 0/7 | 0/7 | 0/7 | 1/6 | 0/5 |
| Parainfluenzae virus infectionInfections and infestations | 0/7 | 0/7 | 0/7 | 1/6 | 0/5 |
| Liver function test increasedInvestigations | 0/7 | 0/7 | 0/7 | 1/6 | 0/5 |
| NephrolithiasisRenal and urinary disorders | 0/7 | 0/7 | 0/7 | 1/6 | 0/5 |
| Cardiac arrestCardiac disorders | 0/7 | 1/7 | 0/7 | 0/6 | 0/5 |
| Pericardial effusionCardiac disorders | 0/7 | 1/7 | 0/7 | 0/6 | 0/5 |
| Event | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 |
|---|---|---|---|---|---|
| Injection site reactionGeneral disorders | 0/7 | 2/7 | 4/7 | 2/6 | 2/5 |
| Abdominal painGastrointestinal disorders | 3/7 | 1/7 | 0/7 | 1/6 | 0/5 |
| FatigueGeneral disorders | 1/7 | 3/7 | 0/7 | 2/6 | 0/5 |
| Injection site erythemaGeneral disorders | 3/7 | 1/7 | 0/7 | 2/6 | 0/5 |
| Decreased appetiteMetabolism and nutrition disorders | 3/7 | 2/7 | 0/7 | 0/6 | 0/5 |
| RashSkin and subcutaneous tissue disorders | 2/7 | 0/7 | 3/7 | 0/6 | 0/5 |
| Oedema peripheralGeneral disorders | 2/7 | 1/7 | 0/7 | 1/6 | 2/5 |
| HypokalaemiaMetabolism and nutrition disorders | 0/7 | 0/7 | 1/7 | 0/6 | 2/5 |
| Steroid diabetesMetabolism and nutrition disorders | 0/7 | 0/7 | 0/7 | 0/6 | 2/5 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 0/7 | 2/7 | 2/7 | 1/6 | 2/5 |
Measured in the safety analysis set, which included all participants who received at least 1 dose of efavaleukin alfa.
| Age, Continuous(years) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Total |
|---|---|---|---|---|---|---|
| Mean | 48.9 ± 12.5 | 52.9 ± 17.4 | 49.4 ± 12.2 | 55.3 ± 7.6 | 64.6 ± 5.9 | 53.5 ± 12.7 |
| Sex: Female, Male(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Total |
|---|---|---|---|---|---|---|
| Female | 3 | 3 | 2 | 3 | 1 | 12 |
| Male | 4 | 4 | 5 | 3 | 4 | 20 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Total |
|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 0 | 0 | 0 | 1 |
| Not Hispanic or Latino | 1 | 5 | 6 | 6 | 5 | 23 |
| Unknown or Not Reported | 5 | 2 | 1 | 0 | 0 | 8 |
| Race (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 2 | 2 | 2 | 7 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 | 0 | 0 |
| White | 2 | 4 | 4 | 4 | 3 | 17 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 5 | 2 | 1 | 0 | 0 | 8 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Time frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Access criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
Data sharing details ↗ · www.amgen.com
Ran from Apr 2018 to primary completion in Oct 2022, 53 months.
Results were first posted in Nov 2023, 1 year 1 month after primary completion.
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Studies of the drug tested here: Efavaleukin alfa
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