Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03375047
• Date of registration: 07/09/2017
• Prospective Registration: Yes
• Primary sponsor: Translate Bio, Inc.
• Public title: Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis RESTORE-CF
• Scientific title: A Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic Fibrosis
• Date of first enrolment: May 10, 2018
• Target sample size: 40
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03375047
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Elizabeth Brown

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF as defined by both of the following:

- Two CF disease-causing CFTR mutations in Class I or II (genotype confirmed at the
screening visit).

- Chronic sinopulmonary disease and/or gastrointestinal/nutritional abnormalities
consistent with CF disease.

- Clinically stable CF disease, as judged by the investigator.

- FEV1 =50% and =90% of the predicted normal for age, gender, and height at screening.

- Resting oxygen saturation =92% on room air (pulse oximetry).

Exclusion Criteria:

- An acute upper or lower respiratory infection, pulmonary exacerbation, clinically
significant episode of hemoptysis or change in chronic respiratory medications
(including antibiotics) for CF lung disease within 28 days prior to dosing with
investigational product on Day 1.

- Receiving treatment with ivacaftor monotherapy (KALYDECO)

- For all groups except Daily dosing: Receiving treatment with triple combination
therapy (TRIKAFTA).

- Subjects with a Class III, IV, or V CFTR gene mutation in at least 1 allele.

- Infection with highly virulent bacteria associated with accelerated decline in
pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia,
Burkholderia dolosa, Mycobacterium abscessus).

Treatment with ORKAMBI or SYMDEKO is not an exclusion for this study.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: MRT5005

Drug: Normal saline

Primary Outcome(s)

Types, frequency and severity of treatment-emergent Adverse Events [Time Frame: 12 months after last dose]

Secondary Outcome(s)

Biological activity of nebulized MRT5005 [Time Frame: 4 weeks after last dose]

Secondary ID(s)

MRT5005-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors