A Phase 2 interventional study of ACTIMMUNE in Autosomal Dominant Osteopetrosis Type 2, sponsored by Indiana University. Completed at 1 site in United States. Open to participants aged 3 Years to 65 Years. Per ClinicalTrials.gov, last updated 2021-01-08.
Sponsored by Indiana University · Phase 2, Interventional, and Treatment
This study is an open label use of ACTIMMUNE for patients with Autosomal Dominant Osteopetrosis Type 2(ADO2). Effects of treatment will be evaluated after 14 weeks on ACTIMMUNE by bone resorption markers. This study will treat 12 patients with ADO2 recruited from Indiana University and Riley Hospital for Children at Indiana University Health.
This is a single center, open-label, dose-escalation study evaluating the efficacy, as defined by biochemical endpoints, and safety profiles of ACTIMMUNE in ADO2 subject.
The investigators will treat 12 ADO2 subjects (children or adults age 3-65) with Actimmune® via a dose escalation protocol to a dose of 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks. If serum CTX does not increase by more than 25% by week 8, the dose will be increased to 100 µg/m2 subcutaneously TIW.
Individual subjects in whom ACTIMMUNE administration increases bone resorption markers during the 14 weeks of this trial will be eligible for a 1 year extension trial.
22 studies on the registry are indexed under Osteopetrosis; 4 are open to participants now.
This study's enrollment of 12 is below the median of 15 across 17 interventional studies indexed under Osteopetrosis.
Browse Osteopetrosis studies →Indiana University is the lead sponsor of 986 studies on the registry; 198 are open to participants now.
Of its 148 completed or terminated interventional studies of FDA-regulated products, 118 (80%) 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.
Subject is diagnosed with ADO2 as determined by the investigator.
Individuals will be screened who have either been diagnosed with osteopetrosis and have a clinical phenotype and/or family history that is consistent with ADO2, have been told that they have an abnormally high bone density (>3SD above mean for age and sex), or a clinical presentation consistent with ADO2. Initial contact will be with members of ADO2 kindreds who have known disease.
Exclusion Criteria:
0 = Normal
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.
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.
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.
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.
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 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.
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.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
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.
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.
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.
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.
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.
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.
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.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
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.
ACTIMMUNE 50 µg/m2 subcutaneously three times per week (TIW) for 8 weeks
Drug: ACTIMMUNE
Changes in Bone Resorption Markers From Baseline to 14 Weeks.
Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks
Time frame: baseline, 14 weeks
Change in Bone Turnover Markers Between After Completion of 6-12 Weeks of Treatment
Evaluate for changes in bone turnover markers including TRAP5b, NTX, CTX/TRAP5b ratio after 6-12 weeks of treatment.
Time frame: 6-12 weeks
| Milestone | Treatment |
|---|---|
| Started | 12 |
| Completed | 12 |
| Not completed | 0 |
Evaluate for changes in bone resorption markers including CTX, NTX/creatinine ratio between baseline and 14 weeks
| percentage of change | Treatment |
|---|---|
| Percent change of CTX | 2.2 (-35.7 to 90.5) |
| Percent change of NTX/creatinine ratio | -2.1 (-50.5 to 55.5) |
Evaluate for changes in bone turnover markers including TRAP5b, NTX, CTX/TRAP5b ratio after 6-12 weeks of treatment.
| percentage of change | Treatment |
|---|---|
| TRAP5B | -15.34 (-57.07 to 13.1) |
| NTX | -2.09 (-50.51 to 55.54) |
| CTX/TRAP5b ratio | 26.31 (-35.91 to 144.57) |
Collected over From signing consent till final followup at 18 weeks (4 weeks after stopping study drug). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment | 0/12 (0%) | 0/12 (0%) | 10/12 (83.3%) |
| Event | Treatment |
|---|---|
| HeadacheNervous system disorders | 10/12 |
| FatigueNervous system disorders | 9/12 |
| nauseaGastrointestinal disorders | 9/12 |
| flu like symptomsProduct Issues | 8/12 |
| myalgiaMusculoskeletal and connective tissue disorders | 7/12 |
| white blood cell decreasedBlood and lymphatic system disorders | 6/12 |
| arthralgiaMusculoskeletal and connective tissue disorders | 5/12 |
| diarrheaGastrointestinal disorders | 5/12 |
| neutrophil count decreasedBlood and lymphatic system disorders | 5/12 |
| vomitingGastrointestinal disorders | 5/12 |
| Age, Categorical(Participants) | Treatment |
|---|---|
| <=18 years | 3 |
| Between 18 and 65 years | 9 |
| >=65 years | 0 |
| Age, Continuous(years) | Treatment |
|---|---|
| Total | 30.48 (8.1 to 50.8) |
| Sex: Female, Male(Participants) | Treatment |
|---|---|
| Female | 6 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | Treatment |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 12 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Treatment |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 11 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Treatment |
|---|---|
| United States | 12 |
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Studies of the drug tested here: Interferon gamma-1b