Trial Outcomes & Findings for Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome (NCT NCT00527878)

Last Updated: 2013-02-04

Results Overview

Patients received one year of treatment medication and one year of placebo. New infections (bacterial, fungal, viral or parasitic) were defined as those requiring an addition or change of an antimicrobial (including topical, oral or intravenous therapies) or those requiring a medical procedure (i.e., incision and drainage of a skin abscess, warm soaks to aid abscess drainage or sinus drainage).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

1 year on intervention

Results posted on

2013-02-04

Participant Flow

16 participants were screened but only 14 participants were randomized. Two individuals decided not to participate.

Participant milestones

Participant milestones
Measure	Placebo/Ranitidine Ranitidine will be dosed orally at 150 mg twice daily for adults, and at 2-4 mg/kg/dose twice daily for children with a maximum dose of 150 mg twice daily. Liquid formulations will be provided for individuals unable to swallow pills. Subjects will be randomized to receive placebo for 12 months followed by 12 months of the ranitidine.	Ranitidine/Placebo Ranitidine will be dosed orally at 150 mg twice daily for adults, and at 2-4 mg/kg/dose twice daily for children with a maximum dose of 150 mg twice daily. Liquid formulations will be provided for individuals unable to swallow pills. Subjects will be randomized to receive ranitidine for 12 months followed by 12 months of the ranitidine.
Intervention 1 STARTED	7	7
Intervention 1 COMPLETED	5	4
Intervention 1 NOT COMPLETED	2	3
Intervention 2 STARTED	5	4
Intervention 2 COMPLETED	5	4
Intervention 2 NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients n=16 Participants
Age, Categorical <=18 years	6 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age Continuous	25.2 years STANDARD_DEVIATION 13.6 • n=99 Participants
Sex: Female, Male Female	8 Participants n=99 Participants
Sex: Female, Male Male	8 Participants n=99 Participants
Region of Enrollment United States	16 participants n=99 Participants

PRIMARY outcome

Timeframe: 1 year on intervention

Population: Patients that completed both arms of the study (24 months)

Outcome measures

Outcome measures
Measure	Placebo n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the placebo for both arms.	Ranitidine n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the treatment for both arms.
Number of Infections in Subjects With HIES.	4 infections Interval 2.0 to 25.0	4 infections Interval 0.0 to 15.0

SECONDARY outcome

Timeframe: 12 months placebo/12 months ranitidine

Population: Patients that completed the study

Patients reported the number of new skin infections

Outcome measures

Outcome measures
Measure	Placebo n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the placebo for both arms.	Ranitidine n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the treatment for both arms.
New Skin Infections	1 skin infections Interval 0.0 to 14.0	0 skin infections Interval 0.0 to 10.0

SECONDARY outcome

Timeframe: 12 months placebo and 12 months ranitidine

Population: Patients who completed the study

Number of new infection while on placebo or study drug

Outcome measures

Outcome measures
Measure	Placebo n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the placebo for both arms.	Ranitidine n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the treatment for both arms.
New Lung Infections	1 new lung infections Interval 0.0 to 4.0	0 new lung infections Interval 0.0 to 4.0

SECONDARY outcome

Timeframe: one year on ranitidine and one year on placebo

Population: Patients who completed the study

Scoring that was completed every 3 months. Clinical severity scored had outcomes that could range from 0 to 121 with 0 being the least severe and 121 being the most severe.

Outcome measures

Outcome measures
Measure	Placebo n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the placebo for both arms.	Ranitidine n=9 Participants Crossover study in which patients received one year of placebo and one year of ranitidine. This analysis will include the treatment for both arms.
Clinical Severity Score	9.8 score on a scale Interval 3.2 to 27.5	8.5 score on a scale Interval 3.5 to 30.3

Adverse Events

Placebo

Serious events: 4 serious events

Other events: 10 other events

Deaths: 0 deaths

Ranitidine

Serious events: 4 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=11 participants at risk this was a crossover study and patients received both placebo and study drug (ranitidine), both of which for 12 months, unless they terminated the study.	Ranitidine n=12 participants at risk This was a crossover study and patients received 12 months of ranitidine and 12 months of placebo, unless they terminated the study.
Respiratory, thoracic and mediastinal disorders asthma	0.00% 0/11 • Number of events 1 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Cardiac disorders hypotension	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations pneumonia	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations skin abscess	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations groin abscess	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations central line infection	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Respiratory, thoracic and mediastinal disorders death from respiratory failure	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.

Other adverse events

Additional Information

Alexandra Freeman MD, Staff Clinician, Lead Associate Investigator for Protocol

NIAID, NIH

Phone: 301-594-9045

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Placebo n=11 participants at risk this was a crossover study and patients received both placebo and study drug (ranitidine), both of which for 12 months, unless they terminated the study.	Ranitidine n=12 participants at risk This was a crossover study and patients received 12 months of ranitidine and 12 months of placebo, unless they terminated the study.
Infections and infestations sinusitis	36.4% 4/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	50.0% 6/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations otitis	36.4% 4/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	33.3% 4/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations upper respiratory tract infection	36.4% 4/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	41.7% 5/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Gastrointestinal disorders gastric ulcer	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Blood and lymphatic system disorders anemia	27.3% 3/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Blood and lymphatic system disorders neutropenia	27.3% 3/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	33.3% 4/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Respiratory, thoracic and mediastinal disorders cough	27.3% 3/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	16.7% 2/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations abscess	36.4% 4/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	41.7% 5/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Respiratory, thoracic and mediastinal disorders chest pain	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Musculoskeletal and connective tissue disorders fracture	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Immune system disorders pruritus	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	16.7% 2/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Gastrointestinal disorders vomiting	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	16.7% 2/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations Streptococcal sore throat	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Skin and subcutaneous tissue disorders rash	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	25.0% 3/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Musculoskeletal and connective tissue disorders arthralgias	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Hepatobiliary disorders elevated alkaline phosphatase	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations candidiasis	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	16.7% 2/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations fever	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Skin and subcutaneous tissue disorders folliculitis	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Gastrointestinal disorders nausea	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
General disorders Dental problems	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Gastrointestinal disorders abdominal pain	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
General disorders pain	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
General disorders tongue ulcer	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations pneumonia	18.2% 2/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Respiratory, thoracic and mediastinal disorders dyspnea	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Hepatobiliary disorders elevated Liver function tests	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Gastrointestinal disorders occult blood in stool	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Eye disorders eyelid stye	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations infection	9.1% 1/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	0.00% 0/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.
Infections and infestations urinary tract infection	0.00% 0/11 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.	8.3% 1/12 Adverse events were not analyzed for each participant as 7 participants did not complete the study and so we were not able to evaluate for adverse events for both placebo and study medication.