A study to establish the tolerabilty and adverse effects in people with a severe form of Cystic Fibrosi (delta F 508 homozygous) when using a combination of two products - cyteamine (a licensed medicine being used in a new disease) and EGCG (a food supplement).

Phase 1

• Conditions: DeltaF508 Homozygous Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2017-001259-29-GB
• Lead Sponsor: niversity of LIverpool

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-12-21
• Last Update Posted: 25 May 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 44

Inclusion Criteria

Adult Inclusion Criteria

1.Patient managed by respiratory team at Leeds General Infirmary
2.Patient has homozygous ?F508 Cystic Fibrosis
3.18-30 years old at screening visit
4.Written informed consent obtained from patient. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.
5.Patient must have a good understanding of the English language, in which the consent forms are available, and understand the requirements for reporting of any AE to the Investigator.
6.If female, be willing to have a pregnancy test and use one or more of the following methods of highly effective contraception:
Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
Oral
Intravaginal
Transdermal
Progestogen-only hormonal contraception associated with inhibition of ovulation
Oral
Injectable
Implantable
Intrauterine device (IUD)
Intrauterine hormone-releasing system ( IUS)
Bilateral tubal occlusion
Vasectomised partner
Sexual abstinence
7.Patient willing and able to comply with protocol including swallowing capsules and venepuncture/cannulation weekly for 6 weeks

Paediatric Inclusion Criteria

8.Patient managed by respiratory team at Alder Hey Children’s NHS Foundation Trust
9.Patient has homozygous ?F508 Cystic Fibrosis
10.5-17 years at screening visit
11.Written informed consent from parent/legal guardian if patient <16 years or from patient if =16 years. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.
12.Verbal or written age appropriate assent from the patient.
13.Patient and parent/legal guardian must have a good understanding of the English language, in which the consent/assent forms are available, and understand the requirements for reporting of any AE to the Investigator.
14.If female, and peri/post pubertal, must be willing to have a pregnancy test and use one or more of the following methods of highly effective contraception:
Sexual abstinence
Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
Oral
Intravaginal
Transdermal
Progestogen-only hormonal contraception associated with inhibition of ovulation
Oral
Injectable
Implantable
Intrauterine device (IUD)
Intrauterine hormone-releasing system ( IUS)
Bilateral tubal occlusion
Vasectomised partner
15.Patient and parent/legal guardian willing and able to comply with protocol including swallowing capsules and venepuncture/cannulation weekly for 6 weeks

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Adult Exclusion Criteria

1.Use of EGCG, or drinking green tea within 2 weeks of the first dose of study and during study period
2.Use of Ivacaftor, Lumacaftor or other mutations specific therapy
3.Drinking black tea within 2 weeks of the first dose of study drugs, and during the study period
4.Known hypersensitivity to Cysteamine, EGCG, or penicillamine
5.Known hypersensitivity to the excipients of the study drugs (i.e. microcrystalline cellulose, pregelatinized starch, magnesium stearate/sodium lauryl sulfate, colloidal silicon dioxide, croscarmellose sodium gelatin, titanium dioxide, black ink on hard capsules containing E172).
6.Recent infective exacerbation of CF. Patient must not have had oral antibiotics within 4 weeks of screening or IV antibiotics within 6 weeks of screening
7.Any clinically significant acute illness within 4 weeks of the start of dose administration.
8.Any clinically significant medical condition, other than CF that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., severe reflux, psychiatric condition or behavioural disorder).
9.Patient on regular itraconazole
10.Liver function tests deemed too abnormal by a clinician experienced in the management of cystic fibrosis.
11.Renal function abnormal for age.
12.Significantly low red cell folate level
13.Pregnant or breastfeeding
14.Written consent not obtained.
15.The patient is deemed unsuitable at the discretion of the investigator.
16.Patients unable to swallow tablets/capsule by mouth

Paediatric Exclusion Criteria

1.Use of EGCG, or drinking green tea within 2 weeks of the first dose of study and during study period
2.Use of Ivacaftor, Lumacaftor or other mutations specific therapy
3.Drinking black tea within 2 weeks of the first dose of study drugs, and during the study period
4.Known hypersensitivity to cysteamine or EGCG
5.Known hypersensitivity to the excipients of the study drugs (i.e. microcrystalline cellulose, pregelatinized starch, magnesium stearate/sodium lauryl sulfate, colloidal silicon dioxide, croscarmellose sodium gelatin, titanium dioxide, black ink on hard capsules containing E172).
6.Recent infective exacerbation of CF. Patient must not have had oral antibiotics within 4 weeks of screening or IV antibiotics within 6 weeks of screening
7.Any clinically significant acute illness within 4 weeks of the start of dose administration.
8.Any clinically significant medical condition, other than CF that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., severe reflux, psychiatric condition or behavioural disorder).
9.Patient on regular itraconazole
10.Liver function tests deemed too abnormal by a clinician experienced in the management of cystic fibrosis.
11.Renal function abnormal for age.
12.Significantly low red cell folate level
13.Pregnant or breastfeeding
14.Written consent not obtained.
15.Participant who understands the study (judged on case by case basis, from approximately age 7) declines assent.
16.The patient is deemed unsuitable at the discretion of the investigator.
17.Patients unable to swallow tablets/capsule by mouth

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified