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Healthy VolunteersPhase 1NCT05330286

Study to Compare the Pharmacokinetics, Safety and Tolerability of the Pediatric and Adult Branaplam Formulation in Healthy Adults and the Effect of Food on the Latter.

A Phase 1 study testing LMI070 in people with Healthy Volunteers, run by Novartis Pharmaceuticals at 1 site in United Kingdom.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a Phase 1 study designed to assess the relative bioavailability (BA), safety and tolerability and PK of the pediatric and adult formulations of branaplam. Read more about this study

Who is it for?
Ages 18 to 60 yearsHealthy volunteers can take part
Conditions the study is for
Healthy Volunteers
What is being tested?
LMI070
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
1 siteUnited Kingdom
Study dates
13 Apr 2022to 19 Aug 2022
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 60 years
  • All sexes
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • Healthy male and non-childbearing potential female participants, 18 to 60 years of age inclusive, and in good health as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests at screening and baseline 1 (laboratory parameters are listed in Table 8-4.
  • Participants must weigh at least 50 kg at screening to participate in the study, and must have a body mass index within the range of 18.0 to 30.0 kg/m2 as measured at screening. Body mass index = Body weight (kg) / [Height (m)]2.
  • At screening and baseline vital signs (systolic blood pressure, diastolic blood pressure and pulse rate) will be assessed in the supine position and again in the standing position (after at least 3 minutes in each position). Oral body temperature will also be taken with the other supine vital sign assessments. Supine vital signs must be within the following ranges at screening and baseline 1:
  • oral body temperature 35.0-37.5 °C (inclusive)
  • systolic blood pressure, 90-139 mmHg (inclusive)
  • diastolic blood pressure, 50-89 mmHg (inclusive)
  • pulse rate, 40-90 bpm (inclusive) Participants should be excluded if their standing vital signs (relative to supine) show findings which, in the opinion of the Investigator, are associated with clinical manifestation of postural hypotension (i.e. absence of any other cause). An Investigator should carefully consider enrolling participants with either a > 20 mmHg decrease in systolic blood pressure or a > 10 mmHg decrease in diastolic blood pressure accompanied by a > 20 bpm increase in pulse rate.

Who cannot join

  • Participants who have received any investigational medicinal product in a clinical research study within the 90 days or 5 half-lives, whichever is longer, prior to Period 1 Day 1.
  • Participants who have previously been administered investigational medicinal product in this study. Participants who have taken part in Part 1 are not permitted to take part in Part 2.
  • Significant illness, which has not resolved within two (2) weeks prior to initial dosing.
  • Men planning to father children in the near future (next 6 months).
  • Male participant who reports to have a pregnant or nursing (lactating) partner.
  • Sexually active males unwilling to adhere to the contraception requirements of the study as detailed below:
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  • A condom is required for all sexually active male participants to prevent them from fathering a child AND to prevent delivery of the investigational drug via seminal fluid to their partner.
  • Males with partners of childbearing potential must use a condom during intercourse while taking investigational drug and for 118 days after stopping investigational drug (duration to cover one spermatogenesis cycle plus 5 half-lives).
  • Additionally, male participants with female partners of childbearing potential should also use another highly effective method of contraception. Highly effective contraception methods include:
  • Partner's bilateral tubal occlusion Male participant sterilization (vasectomy; at least 6 months prior to screening).
  • Partner's use of oral (estrogen and progesterone; or progesterone only that inhibits ovulation), injected, or implanted hormonal methods of contraception or placement of an intrauterine device or intrauterine system, or other forms of hormonal contraception that have comparable efficacy (failure rate < 1%), for example hormone vaginal ring or transdermal hormone contraception. In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months.
  • Total abstinence (when this is in line with the preferred and usual lifestyle of the participant). Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
  • Males with partners of non-childbearing potential must use a condom during intercourse while taking investigational drug and for 28 days after stopping investigational drug (duration to cover 5 half lives).
  • In addition, male participants should not donate sperm for 118 days after stopping investigational drug.
  • Women of childbearing potential who report to be pregnant or nursing (lactating). Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant. Women are considered not of child-bearing potential if they are post-menopausal or have had surgical bilateral salpingectomy or bilateral oophorectomy (with or without hysterectomy) or total hysterectomy at least six weeks before screening. Women are considered post menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) and serum follicle stimulating hormone concentration of ≥40 IU/L. Follicle stimulating hormone and luteinizing hormone testing is required of any female participant, regardless of reported reproductive/menopausal status at screening. Refer to Section 8.4.3 Pregnancy and Assessments of Fertility.

Trial locations and contacts

1 site in 1 country.

United Kingdom1 site

  • Novartis Investigative Site

    Mere Way, Nottingham, NG11 6JS

Other trials in United Kingdom

About this study

Official title: Randomized, Single-dose, Open-label, Two-part, Two-period, Cross-over Study to Compare the Pharmacokinetics, Safety and Tolerability of the Pediatric With an Adult Formulation of Branaplam and to Investigate the Adult Formulation in Fed and Fasted State in Healthy Participants

This is a Phase 1 study designed to assess the relative bioavailability (BA), safety and tolerability and PK of the pediatric and adult formulations of branaplam.

Read the detailed description

This study is to compare the pharmacokinetics, safety and tolerability of the pediatric and adult branaplam formulation in healthy adults. The study will also clarify if dosing with food can affect the PK of the adult formulation in order to guide recommendations on dosing relative to meals in subsequent studies. It is two-part, two-period, cross-over study in healthy participants which means that participants will receive both doses and take the treatment with and without food. The total study duration for each participant is expected to be up to approximately 88 days, including the Screening period and safety FU call.

Participants will be required to be stay at the site overnight for 6 days during each period to receive dose and have multiple blood draws.

Being tested
LMI070
Keywords
healthy, healthy participants, adult, Relative bioavailability study, branaplam, LMI070

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LMI070Drug

    oral solution

  • LMI070Drug

    Oral solution

  • LMI070Drug

    Oral solution given in fasted state

  • LMI070Drug

    Oral solution given in fed state

Study groups in NCT05330286
Study groupRoleWhat they receive
Pediatric FormulationExperimentalPart 1 - healthy participants receiving pediatric formulation
Receives: Drug: LMI070
Adult FormulationExperimentalPart 1 - healthy participants receiving adult formulation
Receives: Drug: LMI070
Adult formulation fasted stateExperimentalPart 2- healthy participants receiving adult formulation in fasted conditions
Receives: Drug: LMI070
Adult formulation Fed stateExperimentalPart 2- Part 2- healthy participants receiving adult formulation in fed conditions
Receives: Drug: LMI070

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part 1: Plasma Cmax

    Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15

  2. Part 1: Plasma AUClast

    Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  3. Part 1: Plasma AUCinf

    Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  4. Part 2:Plasma Cmax

    Investigate food effect on the pharmacokinetics of the adult formulation of branaplam

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  5. Part 2: Plasma AUClast

    Investigate food effect on the pharmacokinetics of the adult formulation of branaplam

    Measured: Day1, 2, 3, 4, 5, 8, 11 and 15 in each period

  6. Part 2: Plasma AUCinf

    Investigate food effect on the pharmacokinetics of the adult formulation of branaplam

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

Secondary outcomes (8)

  1. Number of Adverse events and Serious adverse events

    Any clinically significant events from other safety assessments will be recorded as adverse events as evaluated by investigator.

    Measured: Day 1 up to 30 days after last drug administration

  2. Parts 1 & 2: Plasma Cmax

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  3. Parts 1 & 2: Plasma AUClast

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  4. Parts 1 &2: Plasma AUCinf

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  5. Parts 1 & 2: Plasma Tmax

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  6. Parts 1 & 2: T1/2

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  7. Parts 1 & 2: Plasma Vz/F

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

  8. Parts 1 & 2: Plasma CL/F

    Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112

    Measured: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Apr 2022
  2. Study start13 Apr 2022
  3. Primary completion19 Aug 2022
  4. Study completion19 Aug 2022

Why it stopped: The compound is no longer suitable for further clinical development due to a safety finding in a later phase program.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05330286
Sponsor's protocol ID
CLMI070A02104
Status last verified
Jul 2023
First submitted
8 Apr 2022
Last updated
14 Jul 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing LMI070 in people with Healthy Volunteers, run by Novartis Pharmaceuticals at 1 site in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: The compound is no longer suitable for further clinical development due to a safety finding in a later phase program.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Plasma Cmax (time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05330286.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2023.

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