Trial Outcomes & Findings for Efficacy and Safety of a 0.1% Tacrolimus Nasal Ointment as a Treatment for Epistaxis in Hemorrhagic Hereditary Telangiectasia (HHT) (NCT NCT03152019)

Results Overview

Efficacy of tacrolimus nasal ointment on nosebleeds when administered for 6 weeks

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

up to 12 weeks

Results posted on

2020-07-01

Participant Flow

Participant milestones

Participant milestones
Measure	Protopic® 0.1% (Tacrolimus) Ointment Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.
Overall Study STARTED	25	25
Overall Study COMPLETED	25	24
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Protopic® 0.1% (Tacrolimus) Ointment Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.
Overall Study Adverse Event	0	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Protopic® 0.1% (Tacrolimus) Ointment n=26 Participants Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment n=24 Participants Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Total n=50 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=26 Participants	0 Participants n=24 Participants	0 Participants n=50 Participants
Age, Categorical Between 18 and 65 years	26 Participants n=26 Participants	23 Participants n=24 Participants	49 Participants n=50 Participants
Age, Categorical >=65 years	0 Participants n=26 Participants	1 Participants n=24 Participants	1 Participants n=50 Participants
Sex: Female, Male Female	9 Participants n=26 Participants	14 Participants n=24 Participants	23 Participants n=50 Participants
Sex: Female, Male Male	17 Participants n=26 Participants	10 Participants n=24 Participants	27 Participants n=50 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment France	26 participants n=26 Participants	24 participants n=24 Participants	50 participants n=50 Participants

PRIMARY outcome

Timeframe: up to 12 weeks

Population: n=26 included in per protocol analysis because of a wrong allocation: 1 patient in placebo group who received tacrolimus

Efficacy of tacrolimus nasal ointment on nosebleeds when administered for 6 weeks

Outcome measures

Outcome measures
Measure	Protopic® 0.1% (Tacrolimus) Ointment n=26 Participants Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment n=24 Participants Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.
Percentage of Patients Experiencing an Improvement in Their Nosebleeds	10 Participants	9 Participants

SECONDARY outcome

Timeframe: up to 12 weeks

Tolerance will be evaluated by recording adverse reactions and adverse events during the treatment period and the follow up period and by clinical examinations during the follow-up period.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

Evaluate efficacy on clinical criteria : epistaxis frequency before and after treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

To evaluate efficacy of tacrolimus nasal ointment on duration of nosebleeds before and after treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

before and after treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

before and after treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

To evaluate efficacy on quality of life with SF36 before and after treatment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 12 weeks

To evaluate efficacy on severity epistaxis score with ESS before and after treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 6 weeks

To evaluate systemic absorption after tacrolimus nasal administrations.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 6 weeks

To evaluate systemic absorption after tacrolimus nasal administrations.

Outcome measures

Outcome data not reported

Adverse Events

Protopic® 0.1% (Tacrolimus) Ointment

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo Ointment

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Protopic® 0.1% (Tacrolimus) Ointment n=26 participants at risk Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment n=24 participants at risk Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.
Infections and infestations Endocarditis	0.00% 0/26 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	4.2% 1/24 • Number of events 1 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus
Infections and infestations Septic shock	0.00% 0/26 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	4.2% 1/24 • Number of events 1 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus
Respiratory, thoracic and mediastinal disorders Epistaxis	3.8% 1/26 • Number of events 1 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	4.2% 1/24 • Number of events 1 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus

Other adverse events

Other adverse events
Measure	Protopic® 0.1% (Tacrolimus) Ointment n=26 participants at risk Protopic® 0.1% ointment, packed in blinded tube of 30g. Protopic® (Tacrolimus) 0.1% ointment: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.	Placebo Ointment n=24 participants at risk Same formulation as the Protopic® 0.1% ointment but without tacrolimus, packed in blinded tube of 30g. Placebo: About 0,1g of ointment is administered by the patient on nasal mucosa of each nostril twice a day for 6 weeks.
Injury, poisoning and procedural complications Thermal burn	34.6% 9/26 • Number of events 10 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	12.5% 3/24 • Number of events 3 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus
Nervous system disorders parosmia	7.7% 2/26 • Number of events 2 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	0.00% 0/24 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus
General disorders malaise	0.00% 0/26 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	12.5% 3/24 • Number of events 3 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus
Musculoskeletal and connective tissue disorders back pain	0.00% 0/26 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus	8.3% 2/24 • Number of events 2 • Day 0, day 15, day 31, day 43, day 85 n=26 in PP because of a wrong allocation, 1 patient in placebo group who received tacrolimus

Additional Information

Dr Dupuis Girod

Hospices Civils de Lyon

Phone: 427856522

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place