Trial Outcomes & Findings for Extension Study (Extended Access) of Cyclosporine Inhalation Solution (CIS) in Lung Transplant and Hematopoietic Stem Cell Transplant Recipients for the Treatment of Bronchiolitis Obliterans (NCT NCT01273207)

NCT ID: NCT01273207

Last Updated: 2020-09-18

Results Overview

Continued improvement in lung function as defined by 10% or more increase in FEV1

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: 6 Months
• Results posted on: 2020-09-18

Participant Flow

The lung transplant arm was closed during the study due to lack of subject enrollment

Participant milestones

Measure	Inhaled Cyclosporine in HSCT Participants Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Overall Study STARTED	7
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Extension Study (Extended Access) of Cyclosporine Inhalation Solution (CIS) in Lung Transplant and Hematopoietic Stem Cell Transplant Recipients for the Treatment of Bronchiolitis Obliterans

Baseline characteristics by cohort

Measure	Inhaled Cyclosporine in HSCT Participants n=7 Participants Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 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	1 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
Status of Pulmonary Disease at Initial Enrollment Stable Pulmonary Disease (Stable FEV1)	4 Participants n=5 Participants
Status of Pulmonary Disease at Initial Enrollment Progressive Pulmonary Disease (Declining FEV1)	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: Inhaled Cyclosporine in hematopoietic stem cell transplant (HSCT) participants

Continued improvement in lung function as defined by 10% or more increase in FEV1

Outcome measures

Measure	Inhaled Cyclosporine in HSCT Participants n=7 Participants Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Improvement in Lung Function - Increase in FEV1	0 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: Inhaled Cyclosporine in hematopoietic stem cell transplant (HSCT) participants

Stabilization in Pulmonary Function Test (PFT) as defined by less than 10% increase in FEV1 or less than 10% decline in FEV1

Outcome measures

Measure	Inhaled Cyclosporine in HSCT Participants n=7 Participants Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Disease Stablization - Decrease in FEV1 or Less Than 10% Increase in FEV1	7 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: Inhaled Cyclosporine in hematopoietic stem cell transplant (HSCT) participants

Disease progression as defined by a 20% or more decline in FEV1, or those who require an increase in immunosuppressive therapies by at least 25% or the addition of new immunosuppressive therapies.

Outcome measures

Measure	Inhaled Cyclosporine in HSCT Participants n=7 Participants Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Disease Progression - Decrease in FEV1 or Additional/Increase in Immunosuppressive Therapies	0 Participants

Adverse Events

Inhaled Cyclosporine in HSCT Participants

Serious events: 1 serious events

Other events: 7 other events

Deaths: 1 deaths

Serious adverse events

Measure	Inhaled Cyclosporine in HSCT Participants n=7 participants at risk Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Blood and lymphatic system disorders Post transplant lymphoproliferative disorder	14.3% 1/7 • 6 months
Gastrointestinal disorders Diarrhoea	14.3% 1/7 • 6 months
Gastrointestinal disorders Nausea	14.3% 1/7 • 6 months
Gastrointestinal disorders Vomiting	14.3% 1/7 • 6 months
General disorders Malaise	14.3% 1/7 • 6 months
Infections and infestations Influenza	14.3% 1/7 • 6 months
Infections and infestations Pneumonia	14.3% 1/7 • 6 months
Infections and infestations Septic shock	14.3% 1/7 • 6 months
Investigations Body temperature increased	14.3% 1/7 • 6 months
Investigations Oxygen saturation decreased	14.3% 1/7 • 6 months
Investigations Sputum culture positive	14.3% 1/7 • 6 months

Other adverse events

Measure	Inhaled Cyclosporine in HSCT Participants n=7 participants at risk Hemopoietic Stem Cell transplant (HSCT) subjects with Bronchiolitis Obliterans Syndrome (BOS) received cyclosporine inhalation solution (CIS) at maximum tolerated dose not exceeding 300 mg administered three times per week
Blood and lymphatic system disorders Haemoglobinaemia	28.6% 2/7 • 6 months
Cardiac disorders Oedema peripheral	28.6% 2/7 • 6 months
Eye disorders Cataract	14.3% 1/7 • 6 months
Eye disorders Dry eye	14.3% 1/7 • 6 months
Gastrointestinal disorders Nausea	14.3% 1/7 • 6 months
General disorders Fatigue	42.9% 3/7 • 6 months
General disorders Oedema	14.3% 1/7 • 6 months
Infections and infestations Influenza	14.3% 1/7 • 6 months
Infections and infestations Meningitis	14.3% 1/7 • 6 months
Infections and infestations Upper respiratory tract infection	42.9% 3/7 • 6 months
Musculoskeletal and connective tissue disorders Muscle spasms	14.3% 1/7 • 6 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Ovarian cyst	14.3% 1/7 • 6 months
Nervous system disorders Headache	28.6% 2/7 • 6 months
Reproductive system and breast disorders Breast mass	14.3% 1/7 • 6 months
Respiratory, thoracic and mediastinal disorders Cough	28.6% 2/7 • 6 months
Respiratory, thoracic and mediastinal disorders Dyspnoea	57.1% 4/7 • 6 months
Respiratory, thoracic and mediastinal disorders Wheezing	14.3% 1/7 • 6 months
Vascular disorders Dizziness	14.3% 1/7 • 6 months

Additional Information

Dr Richard Childs

National Heart Lung and Blood Institute

Phone: +301-451-7128

Email: childsr@nhlbi.nih.gov

Results disclosure agreements

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