Cystic FibrosisPhase 2NCT04302662

Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs

A Phase 2 study testing MS1819-SD in people with Cystic Fibrosis and Cystic Fibrosis Gastrointestinal Disease, run by AzurRx SAS at 10 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 12 years and older
Conditions the study is for: Cystic Fibrosis, Cystic Fibrosis Gastrointestinal Disease, Cystic Fibrosis of Pancreas
What is being tested?: MS1819-SD
How many people: 20 participants
Where: 10 sitesin 2 countries
Study dates: 27 Jun 2019to 3 Jun 2021
Phase: Phase 2
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 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Signed and dated informed consent form.
Age > 12 years at the time of screening
Male or female.
Under stable dose of PPE ≥ 1 month. Stable dose is defined as dose of medication not changed during this time period and the medication must be commercially available and be administered in the recommended dose range.
A nutritional status as defined by:
BMI ≤ 22.0 kg/m2 for female patients

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BMI ≤ 23.0 kg/m2 for male patients
BMI ≤ 50th percentile for patients 12 to < 18 years of age.
Cystic fibrosis, based on 2 clinical features consistent with CF in the opinion of the investigator AND sweat chloride concentration > 60 mmol/L by pilocarpine iontophoresis.
Faecal pancreatic elastase-1 < 100 µg/g of stools at screening.
Baseline CFA < 80% with a maximum daily dose of 10,000 lipase units/kg/day.
Clinically stable with no documented evidence of significant respiratory symptoms that would require administration of intravenous antibiotics, oxygen supplementation, or hospitalization within the 30 days of screening.
Male and female patients, if of childbearing potential, must use a reliable method of contraception during the study. A reliable method of birth control is defined as one of the following: oral or injectable contraceptives, intrauterine device, contraceptive implants, tubal ligation, hysterectomy, or a double-barrier method (diaphragm with spermicidal foam or jelly, or a condom), abstinence or vasectomy. Periodic abstinence (calendar, symptothermal, or post-ovulation methods) is not an acceptable method of contraception. The preferred and usual lifestyle of the patient must also be evaluated in determining if sexual abstinence is a reliable method of birth control.
Be considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator.

Who cannot join

Established or suspected fibrosing colonopathy.
Total or partial gastrectomy.
A history of solid organ transplant or significant surgical resection of the bowel; significant resection of the bowel is defined as any resection of the terminal ileum or ileocecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (eg, increased of new need for pancreatic enzyme supplementation compared with preoperative status to maintain the same nutritional status) should also be excluded.
Any chronic diarrheal illness unrelated to pancreatic insufficiency (eg, infectious gastroenteritis, sprue, inflammatory bowel disease)
Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product (IMP).
Bilirubin > 1.5 times upper limit normal (ULN).

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Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5 times ULN.
Alkaline phosphatase (ALP) > 5 times ULN.
Gamma glutamyltransferase (GGT) > 5 times ULN.
Signs and/or symtoms of liver cirrhosis or portal hypertension (eg, splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF
Patients with a known allergy to the stool marker.
Feeding via an enteral tube during 6 months before screening
Routine use of anti-diarrheals, anti-spasmodics, or cathartic laxatives, or a change in chronic osmotic laxatives (eg, polyethylene glycol) regimen in the previous laxative therapy within the last 12 months before screening
History of severe constipation with < 1 evacuation/week under appropriate laxative therapy within the last 12 months before screening.
Documentation of distal intestinal pseudo-obstruction syndrome within the last 12 months before screening.
Forced Expiratory Volume ≤ 30% at the screening visit.
Lactation or known pregnancy or positive pregnancy test at both screening and baseline for women of childbearing potential.
Participation in another clinical study involving an IMP within 30 days before inclusion or concomitantly with this study.
Poorly controlled diabetes according the investigator's judgement.

Trial locations and contacts

10 sites in 2 countries.

Country

City, postcode or hospital

10 sites in 2 countries

Turkey (Türkiye)6 sites

Çukurova University School of Medicine
Adana
Hacettepe University School of Medicine
Ankara
Akdeniz University School of Medicine
Antalya
Cerrahpasa University School of Medicine
Istanbul
Mamara University School of Medicine
Istanbul
Necmettin Erbakan University,Meram School of Medicine
Konya

Other trials in Turkey (Türkiye)

Hungary4 sites

Országos Korányi TBC és Pulmonológiai Intézet Cisztás Fibrózis Részleg
Budapest, 1122
Borsod-Abaúj-Zemplén Megyei Központi Kórház és Egyetemi Oktatókórház, Gyermekegészségügyi Központ
Miskolc, 3526
Somogy Megyei Kaposi Mór Oktató Kórház, Mosdósi telephelye Mosdósi Gyermekrehabilitációs és Gyermekpulmonológiai Egység
Mosdós, 7257
Tüdőgyógyintézet Törökbálint Gyermekpulmonológiai Osztály és Szakrendelés
Törökbálint, 2045

Other trials in Hungary

About this study

Official title: A Multicenter, Open-label Phase 2 Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs, to Investigate the Efficacy and Safety of This Combination for the Compensation of Severe Exocrine Pancreatic Insufficiency in CF Patients Not Fully Compensated With Only PPEs

This is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). The new medication is called MS1819 spray dried (MS1819-SD) which is a lipase produced by the Lip2 gene of Yarrowia lipolytica using recombinant DNA technology.

The primary purpose of this study is to investigate the efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only.

This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients.

The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods.

The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.

Conditions: Cystic Fibrosis, Cystic Fibrosis Gastrointestinal Disease, Cystic Fibrosis of Pancreas
Being tested: MS1819-SD
Keywords: Exocrine Pancreatic Insufficiency
Medical subject headings: Cystic Fibrosis, Exocrine Pancreatic Insufficiency

What participants receive

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

MS1819-SDDrug
Patients will receive increasing doses from the lowest to a maximum dose of MS1819-SD on top of a stable dose of PPEs. The total treatment phase will range from 39 to 51 days.

What is being measured

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

Coefficient of fat absorption
determination of fat absorption based on fat intake and fat excretion over 3 days on high fat meal
Measured: 15 days
Adverse Events
AE, SAE, SUSAR, immunoallergic reactions
Measured: 81 days

Secondary outcomes (6)

Weight of stools
evaluation of changes in weight of stools from baseline (PPEs only) to each treatment period
Measured: 15 days
number of daily evacuations
evaluation of changes in daily evacuations from baseline (PPEs only) to each treatment period
Measured: 15 days
Steatorrhea,
evaluation of changes in steatorrhea from baseline (PPEs only) to each treatment period
Measured: 15 days
Creatorrhea
evaluation of changes in creatorrhea from baseline (PPEs only) to each treatment period
Measured: 15 days
Body weight
evaluation of changes in body weight from baseline (PPEs) to each treatment period
Measured: 15 days
Consistency of stools
evaluation of consistency of stools from baseline (PPEs) to each treatment period
Measured: 15 days

Timeline

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

27 Jun 20193 Jun 2021

First posted10 Mar 2020
Study start27 Jun 2019
Primary completion3 Jun 2021
Study completion3 Jun 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: Sequential assignment: eligible patients will be receiving increased doses from lower, middle to upper range of MS1819-SD on top of a stable dose of PPEs
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04302662
Sponsor's protocol ID: MS1819/18/02
Status last verified: Aug 2021
First submitted: 4 Mar 2020
Last updated: 19 Aug 2021
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Natural History of Bronchiectasis
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Phase 2 Study to Assess the Safety and Efficacy of ANG003
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Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Open-label Phase 2 Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs, to Investigate the Efficacy and Safety of This Combination for the Compensation of Severe Exocrine Pancreatic Insufficiency in CF Patients Not Fully Compensated With Only PPEs
ClinicalTrials.gov ID: NCT04302662
Acronym / sponsor ID: MS1819/18/02
Conditions: Cystic Fibrosis, Cystic Fibrosis Gastrointestinal Disease, Cystic Fibrosis of Pancreas
Keywords: Exocrine Pancreatic Insufficiency
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: AzurRx SASIndustry (pharma, biotech, devices)
Study start: 27 Jun 2019 (actual)
Primary completion: 3 Jun 2021 (actual)
Study completion: 3 Jun 2021 (actual)
First posted: 10 Mar 2020 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Model description: Sequential assignment: eligible patients will be receiving increased doses from lower, middle to upper range of MS1819-SD on top of a stable dose of PPEs

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (14 listed)

Signed and dated informed consent form.
Age > 12 years at the time of screening
Male or female.
Under stable dose of PPE ≥ 1 month. Stable dose is defined as dose of medication not changed during this time period and the medication must be commercially available and be administered in the recommended dose range.
A nutritional status as defined by:

Key exclusion criteria (19 listed)

Established or suspected fibrosing colonopathy.
Total or partial gastrectomy.
A history of solid organ transplant or significant surgical resection of the bowel; significant resection of the bowel is defined as any resection of the terminal ileum or ileocecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (eg, increased of new need for pancreatic enzyme supplementation compared with preoperative status to maintain the same nutritional status) should also be excluded.
Any chronic diarrheal illness unrelated to pancreatic insufficiency (eg, infectious gastroenteritis, sprue, inflammatory bowel disease)
Known hypersensitivity or other severe reaction to any ingredient of the investigational medicinal product (IMP).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
MS1819-SDPatients will receive increasing doses from the lowest to a maximum dose of MS1819-SD on top of a stable dose of PPEs. The total treatment phase will range from 39 to 51 days.	Drug	–

Primary outcome

Coefficient of fat absorption
Time frame: 15 days
determination of fat absorption based on fat intake and fat excretion over 3 days on high fat meal
Adverse Events
Time frame: 81 days
AE, SAE, SUSAR, immunoallergic reactions

6 secondary outcomes

Weight of stools
Time frame: 15 days
number of daily evacuations
Time frame: 15 days
Steatorrhea,
Time frame: 15 days
Creatorrhea
Time frame: 15 days
Body weight
Time frame: 15 days
Consistency of stools
Time frame: 15 days

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2021.
Individual participant dataSharing planned: Undecided

Related trials for Cystic Fibrosis

RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 2
Phase 2 Study to Assess the Safety and Efficacy of ANG003
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Recruiting
Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
Cystic Fibrosis (CF), Bronchiectasis, Non-Cystic Fibrosis
Not yet recruitingObservational study
Vascular Health and Function in Cystic Fibrosis
Cystic Fibrosis, Endothelial Dysfunction
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

10 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing MS1819-SD in people with Cystic Fibrosis and Cystic Fibrosis Gastrointestinal Disease, run by AzurRx SAS at 10 sites in 2 countries.

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 14 inclusion and 19 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: finished, as of the sponsor's last update on 19 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Turkey (Türkiye), Hungary. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Coefficient of fat absorption (time frame: 15 days). The study lists 2 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 NCT04302662.

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 19 Aug 2021.