Trial Outcomes & Findings for Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1 (NCT NCT00490100)
NCT ID: NCT00490100
Last Updated: 2015-07-03
Results Overview
Rates of adverse events related to study drug
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
6 participants
Primary outcome timeframe
1 year
Results posted on
2015-07-03
Participant Flow
Participant milestones
| Measure |
Treatment
Participants are young males with X-linked severe combined immunodeficiency complicated by growth failure. The participants will receive Insulin-like Growth Facor (Increlex) twice a day for up to 2 years.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
Baseline characteristics by cohort
| Measure |
Treatment
n=6 Participants
|
|---|---|
|
Age, Categorical
<=18 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearRates of adverse events related to study drug
Outcome measures
| Measure |
Treatment
n=3 Participants
XSCID patients with growth failre treated with Increlex, recombinant human IGF-1.
Increlex
|
|---|---|
|
Safety of Study Drug
|
0 participants
|
PRIMARY outcome
Timeframe: During intervention, up to 2 yearsPopulation: Only participants who completed study
Growth rate on intervention is compared with growth rate before intervention for each participant.
Outcome measures
| Measure |
Treatment
n=3 Participants
XSCID patients with growth failre treated with Increlex, recombinant human IGF-1.
Increlex
|
|---|---|
|
Change in Growth Rate on Study Drug
|
5.2 cm/year
Interval 1.2 to 8.6
|
Adverse Events
Treatment
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment
n=6 participants at risk
|
|---|---|
|
Gastrointestinal disorders
Diarrhea (clostridium diff)
|
16.7%
1/6
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
16.7%
1/6
|
|
Infections and infestations
Lung infection
|
33.3%
2/6
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
1/6
|
Additional Information
De Ravin, SukSee
National Institute of Allergy and Infectious Diseases
Phone: +1 301 496 6772
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place