Trial Outcomes & Findings for Safety, Tolerability, Pharmacokinetics and Efficacy of SPR720 for the Treatment of Patients With Mycobacterium Avium Complex (MAC) Pulmonary Disease (NCT NCT04553406)

Results Overview

SPR719 is the active moiety of the prodrug SPR720. Blood samples were planned to be taken at a subset of study sites in order to conduct intensive pharmacokinetic (PK) evaluation.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

Day 1 and Day 28 pre-dose and 1, 2, 4, 8, 12, and 24 hours post-dose

Results posted on

2022-02-28

Participant Flow

A total of 90 participants were planned to be enrolled across 4 treatment groups. As a result of early discontinuation of the study, only 2 treatment groups were initiated, with 1 participant enrolled in each group.

Participant milestones

Participant milestones
Measure	SPR720 500 mg SPR720 500 mg administered orally once daily for 28 days.	Placebo Placebo administered orally once daily for 28 days.
Overall Study STARTED	1	1
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	SPR720 500 mg SPR720 500 mg administered orally once daily for 28 days.	Placebo Placebo administered orally once daily for 28 days.
Overall Study Study Discontinuation	0	1

Baseline Characteristics

Safety, Tolerability, Pharmacokinetics and Efficacy of SPR720 for the Treatment of Patients With Mycobacterium Avium Complex (MAC) Pulmonary Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.	Total n=2 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized White	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 and Day 28 pre-dose and 1, 2, 4, 8, 12, and 24 hours post-dose

Population: No participants were enrolled at study sites that were conducting intensive PK evaluations.

SPR719 is the active moiety of the prodrug SPR720. Blood samples were planned to be taken at a subset of study sites in order to conduct intensive pharmacokinetic (PK) evaluation.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 1 and Day 28 pre-dose and 1, 2, 4, 8, 12, and 24 hours post-dose

Population: No participants were enrolled at study sites that were conducting intensive PK evaluations.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 1 and Day 28 pre-dose and 1, 2, 4, 8, 12, and 24 hours post-dose

Population: No participants were enrolled at study sites that were conducting intensive PK evaluations.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 1 and Day 28 pre-dose and 1, 2, 4, 8, 12, and 24 hours post-dose

Population: No participants were enrolled at study sites that were conducting intensive PK evaluations.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From first dose of study drug (Day 1) up to 28 days after last dose (56 days)

Population: All treated participants

An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational/experimental) product, whether related to this product or not. This includes any newly occurring event or previous condition that has increased in severity or frequency since starting active or randomized treatment. The Investigator assessed the intensity for each AE reported during the study using the latest version of the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.0, as Mild, Moderate, Severe, Life-threatening, or Death.

Outcome measures

Outcome measures
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) All TEAEs	1 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Mild TEAEs	1 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Moderate TEAEs	0 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Severe TEAEs	0 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) TEAEs leading to discontinuation of study drug	0 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) TEAEs leading to discontinuation from study	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Days 1, 7, 14, 21, 28, and 56

Population: All treated participants

Full physical examination were conducted on Day 1 and 28 days after last dose (Day 56) and included, at a minimum, assessment of the following systems: skin, head, ears, eyes, nose and throat, respiratory system, cardiovascular system, gastrointestinal system, neurological condition, blood and lymphatic systems, and the musculoskeletal system. Symptom-directed physical examinations were conducted at study visits on Days 7, 14, 21, and 28.

Outcome measures

Outcome measures
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.
Number of Participants With Clinically Meaningful Change in Physical Examination Findings	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Day 1 to Day 56

Population: All treated participants

Outcome measures

Outcome measures
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.
Number of Participants Who Received Any Concomitant Medication During the Study	1 Participants	1 Participants

SECONDARY outcome

Timeframe: Days 1, 7, 14, 21, 28, and 56

Population: Individual laboratory test results are not reported in order to protect patient confidentiality.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Days 1, 7, 14, 21, 28, and 56

Population: All treated participants

Clinical laboratory tests included serum chemistry, hematology, coagulation tests, and urinalysis. The investigator determined whether any changes in laboratory values were clinically significant based on the condition of the participant and the extent and duration of the deviation from the reference range.

Outcome measures

Outcome measures
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.
Number of Participants With Clinically Significant Out-of-normal Range Laboratory Tests Clinical chemistry	0 Participants	0 Participants
Number of Participants With Clinically Significant Out-of-normal Range Laboratory Tests Hematology	0 Participants	0 Participants
Number of Participants With Clinically Significant Out-of-normal Range Laboratory Tests Urinalysis	0 Participants	0 Participants
Number of Participants With Clinically Significant Out-of-normal Range Laboratory Tests Coagulation tests	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Days 1, 7, 14, 21, 28, and 56

Population: No participants had out-of-range laboratory test results hence shift tables could not be created.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Days 1, 7, 14, 21, 28, and 56

Population: Individual vital sign measurement results are not reported in order to protect patient confidentiality.

Vital signs measurements included systolic and diastolic blood pressure, pulse, temperature, and respiratory rate.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Days 1, 14, 28, and 56

Population: All treated participants

Standard 12-lead electrocardiogram (ECG) assessments included heart rate, cardiac rhythm, PR interval, RR interval, QRS interval, QT interval and QTC interval. Clinical significance was determined by the investigator.

Outcome measures

Outcome measures
Measure	SPR720 500 mg n=1 Participants SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 Participants Placebo administered orally once daily for 28 days.
Number of Participants With Clinically Significant Abnormal Electrocardiogram Findings	0 Participants	0 Participants

Adverse Events

SPR720 500 mg

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	SPR720 500 mg n=1 participants at risk SPR720 500 mg administered orally once daily for 28 days.	Placebo n=1 participants at risk Placebo administered orally once daily for 28 days.
Gastrointestinal disorders Diarrhea	100.0% 1/1 • From first dose of study up to 28 days after last dose (56 days).	0.00% 0/1 • From first dose of study up to 28 days after last dose (56 days).
Gastrointestinal disorders Vomiting	100.0% 1/1 • From first dose of study up to 28 days after last dose (56 days).	0.00% 0/1 • From first dose of study up to 28 days after last dose (56 days).
Gastrointestinal disorders Nausea	100.0% 1/1 • From first dose of study up to 28 days after last dose (56 days).	100.0% 1/1 • From first dose of study up to 28 days after last dose (56 days).

Additional Information

Dr. Jon Bruss

SperoTherapeutics

Phone: (+1) 269-352-3766

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The PI may not publish the results prior to the first multi-site publication. If there is no multi-site publication within 18 months after Trial completion the PI may publish results from the trial, subject to the following. PI will submit proposed Publication to Sponsor 60 days prior to submission of the Publication. PI will, at Sponsor's request, delay such Publication for up to 90 days for Sponsor to obtain appropriate intellectual property protection.
Publication restrictions are in place

Restriction type: OTHER