A Phase 2 interventional study of Interferon gamma-1b in Osteopetrosis, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. Completed at 2 sites in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2024-10-22.
Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Phase 2, Interventional, and Treatment
This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12 months. The investigators hypothesize that ACTIMMUNE will be tolerated by participants for the full 12 months and result in decreased disease severity.
Osteopetrosis is a rare inherited metabolic bone disease characterized by impaired osteoclast function resulting in defective bone resorption and generalized high bone mass and mineral density (BMD). In patients with severe disease, this high bone mass compromises bone marrow space leading to marrow failure and frequent infections, along with hepatosplenomegaly from extramedullary hematopoiesis. Currently, the only treatment for individuals with severe forms of osteopetrosis is hematopoietic cell transplantation (HCT), however survival in patients with osteopetrosis treated with HCT is only around 55%. Therefore, this treatment is only indicated in select individuals with life-threatening complications of their disease. Thus additional treatments for osteopetrosis are needed both for individuals who are not candidates for HCT and to prolong the time until HCT is needed.
Interferon gamma (IFN-γ) is a naturally occurring cytokine that has been shown to have anti-microbial and anti-viral immunomodulatory effects, and is a potent stimulator of superoxide anion production which in turn promotes the formation and activation of osteoclasts. Two previous studies of IFN-γ1b in a small group of individuals with osteopetrosis found a decrease in trabecular bone area, an increase in marrow space, a decrease in the number of severe infections requiring antibiotic therapy, and an increase in superoxide generation by granulocyte-macrophage colonies.
Therefore, the investigators will conduct an early phase 2, multi-center, open-label, 12-month clinical trial of ACTIMMUNE (IFN-γ1b) treatment of patients with intermediate osteopetrosis to determine the following:
22 studies on the registry are indexed under Osteopetrosis; 4 are open to participants now.
This study's enrollment of 5 is below the median of 15 across 17 interventional studies indexed under Osteopetrosis.
Browse Osteopetrosis studies →Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center is the lead sponsor of 67 studies on the registry; 1 is open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 5 (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.
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.
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).
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.
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 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.
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.
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.
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.
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.
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.
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.
Gamma interferon-1b 100 mcg subcutaneous (SC) 3 times weekly for 12 months
Drug: Interferon gamma-1b
gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Also known as: ACTIMMUNE
Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher
Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 defines a Grade 4 event as having life-threatening consequences, and/or urgent intervention indicated.
Time frame: 12 months
Percent Change From Baseline in Bone Mineral Density (BMD)
BMD measured by peripheral quantitative computed tomography (pQCT) in bone area w/ BMD\<169mg/m3
Time frame: 6 months
Change From Baseline in White Blood Cell Count (WBC)
Time frame: 6 months
Change From Baseline in Pain
Survey name: RAND 36-Item Health Survey (Version 1.0) Scale name: Pain Scale range: 0-100; Higher score means less pain
Time frame: 6 months
| Milestone | Gamma Interferon-1b |
|---|---|
| Started | 5 |
| Completed | 1 |
| Not completed | 4 |
| Withdrew: Withdrawal by subject | 4 |
Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 defines a Grade 4 event as having life-threatening consequences, and/or urgent intervention indicated.
| Participants | Gamma Interferon-1b |
|---|---|
| Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher | 0 |
BMD measured by peripheral quantitative computed tomography (pQCT) in bone area w/ BMD\<169mg/m3
| percent change | Gamma Interferon-1b |
|---|---|
| Percent Change From Baseline in Bone Mineral Density (BMD) | 0 (-15 to 2) |
| x1000 cells/uL | Gamma Interferon-1b |
|---|---|
| Change From Baseline in White Blood Cell Count (WBC) | -0.5 (-1.2 to 1) |
Survey name: RAND 36-Item Health Survey (Version 1.0) Scale name: Pain Scale range: 0-100; Higher score means less pain
| change in units on the pain scale | Gamma Interferon-1b |
|---|---|
| Change From Baseline in Pain | -0.8 (-2.0 to 0.3) |
Collected over Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Gamma Interferon-1b | 0/5 (0%) | 0/5 (0%) | 5/5 (100%) |
| Event | Gamma Interferon-1b |
|---|---|
| Flu-like reactionGeneral disorders | 5/5 |
| FatigueGeneral disorders | 4/5 |
| HeadacheNervous system disorders | 4/5 |
| NauseaGastrointestinal disorders | 3/5 |
| Injection site reactionsGeneral disorders | 3/5 |
| MyalgiaMusculoskeletal and connective tissue disorders | 3/5 |
| NeutropeniaBlood and lymphatic system disorders | 2/5 |
| AnemiaBlood and lymphatic system disorders | 2/5 |
| HyperglycemiaEndocrine disorders | 2/5 |
| Abdominal painGastrointestinal disorders | 2/5 |
| Age, Continuous(years) | Gamma Interferon-1b |
|---|---|
| Median | 36.9 (15.1 to 59.9) |
| Sex: Female, Male(Participants) | Gamma Interferon-1b |
|---|---|
| Female | 4 |
| Male | 1 |
| Ethnicity (NIH/OMB)(Participants) | Gamma Interferon-1b |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 4 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Gamma Interferon-1b |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 3 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Gamma Interferon-1b |
|---|---|
| United States | 5 |
| Genotype(Participants) | Gamma Interferon-1b |
|---|---|
| Chloride Voltage-Gated Channel 7 (CLCN7) mutation | 3 |
| T cell immune regulator 1 (TCIRG) mutation | 1 |
| Unknown | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Interferon gamma-1b
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center