Trial Outcomes & Findings for ACTIMMUNE in Intermediate Osteopetrosis (NCT NCT02666768)

NCT ID: NCT02666768

Last Updated: 2024-10-22

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 5 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2024-10-22

Participant Flow

Participant milestones

Measure	Gamma Interferon-1b Gamma interferon-1b 100 micrograms (mcg) subcutaneously (SC) 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Overall Study STARTED	5
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	Gamma Interferon-1b Gamma interferon-1b 100 micrograms (mcg) subcutaneously (SC) 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Overall Study Withdrawal by Subject	4

Baseline Characteristics

ACTIMMUNE in Intermediate Osteopetrosis

Baseline characteristics by cohort

Measure	Gamma Interferon-1b n=5 Participants Gamma interferon-1b 100 micrograms (mcg) subcutaneously (SC) 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Age, Continuous	36.9 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants
Genotype Chloride Voltage-Gated Channel 7 (CLCN7) mutation	3 Participants n=5 Participants
Genotype T cell immune regulator 1 (TCIRG) mutation	1 Participants n=5 Participants
Genotype Unknown	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

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.

Outcome measures

Measure	Gamma Interferon-1b n=5 Participants Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher	0 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 1 participants withdrew due to flu-like side effects prior to 2nd pQCT measurement. 1 participant has no data for this measurement due to size of pQCT gantry.

BMD measured by peripheral quantitative computed tomography (pQCT) in bone area w/ BMD<169mg/m3

Outcome measures

Measure	Gamma Interferon-1b n=3 Participants Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Percent Change From Baseline in Bone Mineral Density (BMD)	0 percent change Interval -15.0 to 2.0

SECONDARY outcome

Timeframe: 6 months

Population: Only 1 participant completed study to 12 months, so 6 month data are reported here.

Outcome measures

Measure	Gamma Interferon-1b n=4 Participants Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Change From Baseline in White Blood Cell Count (WBC)	-0.5 x1000 cells/uL Interval -1.2 to 1.0

SECONDARY outcome

Timeframe: 6 months

Population: One participant was \<18 years old so did not complete the RAND 36-Item Health Survey

Survey name: RAND 36-Item Health Survey (Version 1.0) Scale name: Pain Scale range: 0-100; Higher score means less pain

Outcome measures

Measure	Gamma Interferon-1b n=4 Participants Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Change From Baseline in Pain	-0.8 change in units on the pain scale Interval -2.0 to 0.3

Adverse Events

Gamma Interferon-1b

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Gamma Interferon-1b n=5 participants at risk Gamma interferon-1b 100 mcg SC 3 times weekly for 12 months Interferon gamma-1b: gamma interferon-1b dose escalation over first 4 weeks of study to 100 mcg SC 3 times weekly
Blood and lymphatic system disorders Neutropenia	40.0% 2/5 • Number of events 3 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Blood and lymphatic system disorders Anemia	40.0% 2/5 • Number of events 2 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Endocrine disorders Hyperglycemia	40.0% 2/5 • Number of events 2 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Gastrointestinal disorders Abdominal pain	40.0% 2/5 • Number of events 3 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Gastrointestinal disorders Nausea	60.0% 3/5 • Number of events 3 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
General disorders Fatigue	80.0% 4/5 • Number of events 5 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
General disorders Flu-like reaction	100.0% 5/5 • Number of events 13 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
General disorders Injection site reactions	60.0% 3/5 • Number of events 11 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Infections and infestations Sinusitis/sinus pain/sinus congestion	40.0% 2/5 • Number of events 4 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Musculoskeletal and connective tissue disorders Bone pain - generalized	40.0% 2/5 • Number of events 2 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Musculoskeletal and connective tissue disorders Fracture	40.0% 2/5 • Number of events 3 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Musculoskeletal and connective tissue disorders Joint pain	40.0% 2/5 • Number of events 4 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Musculoskeletal and connective tissue disorders Myalgia	60.0% 3/5 • Number of events 5 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Nervous system disorders Headache	80.0% 4/5 • Number of events 8 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Renal and urinary disorders Proteinuria	40.0% 2/5 • Number of events 2 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.
Skin and subcutaneous tissue disorders Rash	40.0% 2/5 • Number of events 2 • Adverse event data were collected from day 1 to end of study or participant withdrawal, up to year 1.

Additional Information

Dr. Lynda Polgreen

The Lundquist Institute at Harbor-UCLA Medical Center

Phone: 310-222-1961

Email: lpolgreen@lundquist.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place