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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04243837




Registration number
NCT04243837
Ethics application status
Date submitted
21/01/2020
Date registered
28/01/2020
Date last updated
30/11/2020

Titles & IDs
Public title
LYT-100 in Healthy Volunteers and BCRL
Scientific title
A Phase 1 Multiple Ascending Dose and Food Effect Study in Healthy Volunteers to Determine the Pharmacokinetics and Maximally Tolerated Dose of Deupirfenidone (LYT-100) Followed by a Randomized Double-Blind Placebo-Controlled Phase 2a in Patients With Breast Cancer-Related Upper Limb Secondary Lymphoedema
Secondary ID [1] 0 0
LYT-100-2020-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Breast Cancer Related Lymphoedema 0 0
Lymphoedema 0 0
Condition category
Condition code
Cancer 0 0 0 0
Breast
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LYT-100
Other interventions - Matching Placebo
Treatment: Drugs - LYT-100 Food Effect
Treatment: Drugs - LYT-100 BCRL
Treatment: Drugs - Placebo BCRL

Experimental: LYT-100 in healthy volunteers with Food - LYT-100, multiple ascending

Placebo Comparator: Placebo in healthy volunteers with Food - Placebo, multiple administrations

Experimental: LYT-100 in healthy volunteers, Fasted - LYT-100, Dose below MTD for 1 dose

Placebo Comparator: Placebo in healthy volunteers, Fasted - Placebo, for 1 administration

Experimental: LYT-100 in healthy volunteers, Fed - LYT-100, Dose below MTD for 1 dose

Placebo Comparator: Placebo in healthy volunteers, Fed - Placebo, for 1 administration

Experimental: LYT-100 in patients with BCRL - LYT-100 BID for 6 months

Placebo Comparator: Placebo in patients with BCRL - Placebo BID for 6 months


Treatment: Drugs: LYT-100
LYT-100 is a deuterated form of pirfenidone, an orally active small molecule drug.
HV subjects will receive multiple ascending doses, starting at 100 mg BID up to 1000 mg, with food for 5-days.

Other interventions: Matching Placebo
Inactive capsule(s)

Treatment: Drugs: LYT-100 Food Effect
LYT-100 is a deuterated form of pirfenidone, an orally active small molecule drug.
HV subjects will receive one dose of LYT-100 or Placebo

Treatment: Drugs: LYT-100 BCRL
BCRL patients will receive LYT-100 BID for 6 months

Treatment: Drugs: Placebo BCRL
BCRL patients will receive Placebo BID for 6 months

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability: treatment-emergent adverse events (TEAEs) - Evaluate the safety and tolerability of LYT-100 as measured by TEAEs
Timepoint [1] 0 0
7 days (main time frame)
Primary outcome [2] 0 0
Use pharmacokinetics to characterize the plasma concentration of LYT-100 - Measure observed concentration of LYT-100 in blood plasma
Timepoint [2] 0 0
7 days (main time frame)
Secondary outcome [1] 0 0
Efficacy signals of LYT-100 - Explore efficacy signals of LYT-100 in breast cancer patients with lymphedema
Timepoint [1] 0 0
6 months
Secondary outcome [2] 0 0
Effect of food on the pharmacokinetic profile of LYT-100 based on plasma concentration of LYT-100 - Measure concentration of LYT-100 in blood plasma in fed or fasted subjects
Timepoint [2] 0 0
2 days (main time frame)

Eligibility
Key inclusion criteria
Main

Part 1 and 2: Healthy Volunteers

1. Male or female between 18 and 75 years old (inclusive) at the time of screening.

2. In good general health at screening, free from clinically significant unstable
medical, surgical or psychiatric illness, at the discretion of the Investigator.

Part 3: Patients with BRCL

1. Female or male between 18 and 80 years old (inclusive) at the time of informed
consent.

2. At least 6 months since any type of breast cancer surgery (excluding fine needle
aspiration biopsy [FNA]), at the time of study screening. No intention to have breast
reconstructive surgery, nipple reconstruction and/or tattooing during the course of
the study.

3. At least 3 months since completion of all types of treatment for breast cancer,
including but not limited to neoadjuvant, radiotherapy, chemotherapy and
immunotherapy, at the time of study screening.

4. At least 3 months treatment of stable adjuvant treatment with hormonal or anti-HER2
therapy at the time of screening, with no planned changes to this therapy throughout
the duration of the study.

5. Diagnosis of primary breast cancer, and without evidence of recurrence of breast
cancer and/or metastasis for at least 6 months since breast cancer surgery, as
determined at screening and baseline.

6. Documented evidence of Stage 1 or 2 lymphedema.

7. Receiving standard of care compression or agreeable to using care compression, or no
compression at all = 4 weeks prior to screening and throughout the study.

Main
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Part 1 and 2: Healthy Volunteers

1. History or presence of malignancy at screening or baseline, with the exception of
adequately treated localised skin cancer (basal cell or squamous cell carcinoma) or
carcinoma in-situ of the cervix.

2. Clinically significant infection within 28 days of the start of dosing, or infections
requiring parenteral antibiotics within the 6 months prior to screening.

3. Chronic administration (defined as more than 14 consecutive days) of
immunosuppressants or other immune-modifying drugs within 3 months prior to study drug
administration; corticosteroids are permitted at the discretion of the Investigator).

4. History or presence at screening or baseline of a condition associated with
significant immunosuppression

Part 3: Patients with BRCL

1. Bilateral lymphoedema or history of bilateral axillary lymph node removal (i.e.,
sentinel lymph node or axillary lymph node dissection), or primary lymphoedema or
lymphatic or vascular malformation, determined at screening.

2. Chronic administration (defined as more than 14 consecutive days) of
immunosuppressants or other immune-modifying drugs within 3 months prior to study drug
administration; corticosteroids are permitted at the discretion of the PI.

3. Recent history (in the 8 weeks prior to screening) of cellulitis, lymphangitis,
dermatitis, necrotizing fasciitis, or current open wounds or sores in the affected
extremity.

4. Stage III lymphoedema, or history of clinically diagnosed secondary lymphoedema
greater than 2 years, determined at screening.

5. Initiated use of compression or manual lymphatic drainage or other lymphoedema
therapies at the start of the study within 4 weeks of the screening visit. Rescreening
is allowed following a course of stable compression regimen of > 4 weeks.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1/Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
University of the Sunshine Coast - Sippy Downs
Recruitment hospital [2] 0 0
Nucleus Network VIC - Melbourne
Recruitment hospital [3] 0 0
Flinders University - Adelaide
Recruitment hospital [4] 0 0
Macquarie University Health Sciences Centre - Sydney
Recruitment postcode(s) [1] 0 0
- Sippy Downs
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
- Adelaide
Recruitment postcode(s) [4] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
PureTech
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Novotech (Australia) Pty Limited
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Part 1 is a multiple ascending dose trial in healthy volunteers (HV) of LYT-100 to determine
safety, tolerability, and pharmacokinetic (PK) profile under fed conditions.

Part 2 is a single dose safety, tolerability, and PK trial in HV of LYT-100 under fed and
fasted conditions.

Part 3 is a trial of up to 6-months duration for safety, tolerability, clinical efficacy
assessments, PK and biomarkers analysis in breast carcinoma patients with secondary
lymphoedema
Trial website
https://clinicaltrials.gov/show/NCT04243837
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Christopher Korth
Address 0 0
Country 0 0
Phone 0 0
+1 617-226-4813
Fax 0 0
Email 0 0
ckorth@puretechhealth.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04243837