Theophylline Effects in the Fontan Circulation

NCT05717049 | Completed Phase 2

• 1 other identifier: EU CT 2022-500301-41-00
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

A single group treatment phase 2 single-arm no-masking study to assess safety and efficacy of a short-term oral treatment with theophylline (ATC-no. R03D A04) in terms of improvements in cardiorespiratory fitness, health-related quality of life, cardiac performance and respiratory function in male and female adolescents aged 16 to 25 years with a Fontan-type surgical palliation of univentricular congenital heart disease.

Conditions

Congenital Heart Disease

Keywords

Fontan circulation; Cardiorespiratory fitness; Pulmonary function; Cardiac function; Quality of life

Outcome Measures

Primary Outcomes (5)

• Feasibility and safety - adverse events

  Frequency of treatment emerged AE/SAE

  12 weeks

• Feasibility and safety - tolerability

  Freedom from participant dropout based on tolerability of the study intervention

  12 weeks

• Feasibility and safety - drug dosing

  Number of participants requiring dose reduction after first serum concentration

  12 weeks

• Feasibility and safety - arrhythmia

  Freedom of arrhythmogenic side effects of the study treatment leading to patient dropout

  12 weeks

• Cardiorespiratory fitness

  Difference in oxygen uptake at anaerobic/ventilatory threshold pre/post treatment

  12 weeks

Secondary Outcomes (5)

• Health-related quality-of-life

  12 weeks

• Health-related quality-of-life (SF 36)

  12 weeks

• Cardiac performance

  12 weeks

• Respiratory function

  12 weeks

• Sleep disordered breathing

  12 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

12 week oral treatment (theophylline)

Drug: Theophylline Tablets

Interventions

Theophylline Tablets DRUG

12 week oral treatment

Also known as: ATC R03D A04

Treatment

Eligibility Criteria

• Age: 16 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participants with univentricular congenital heart disease with a Fontan-type palliation
• Who are able to perform all diagnostic and monitoring procedures necessary during trial period, in particular being able to perform a symptom-limited cardiopulmonary exercise test on an upright ergometer bicycle.

You may not qualify if:

• Considered and assessed eligible for administration of Theo-Dur® (theophylline) as specified in the SmPC.
• Body mass index (BMI) within the range 18.5 - 34.9 kg/m2 (inclusive).
• Contraceptive use by women is not under any national / local regulations in Norway.
• Male participants: no restrictions.
• Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
• For participants \< 18 years, all (both) parents or caregivers with parental responsibilities have to sign the consent form in addition to the participant.
• Current or previous (last 12 months) tachyarrhythmia which has been cause of medical investigation or hospitalization.
• sinus rhythm or regular supraventricular rhythm (visible P-waves) regardless P-wave angle
• nodal rhythm
• isolated extra beats (supraventricular or ventricular) of a frequency considered clinically non-significant
• Systemic hypertension (systolic or diastolic blood pressure above 95 percentile)
• Biochemical signs of more than mild liver disease or liver failure indicated by one of the following:
• INR \> 1.5 in the absence of warfarin treatment,
• ALAT more than twice the upper normal limit
• Bilirubin more than twice the upper normal limit

Sponsors & Collaborators

• Oslo University Hospital: lead
• South-Eastern Norway Regional Health Authority: collaborator
• Norwegian Health Association: collaborator
• Norwegian Association for Children with Congenital Heart Disease: collaborator

Study Sites (1)

Oslo University Hospital, Rikshospitalet, Department of Paediatric Cardiology

Oslo, 0424, Norway

Location

Related Links

• EU Clinical Trials database, use search term "theophylline"

MeSH Terms

Conditions

Heart Defects, Congenital

Interventions

Theophylline

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Xanthines; Alkaloids; Heterocyclic Compounds; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Thomas Möller, MD PhD

  Oslo University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator, MD PhD, senior consultant / senior researcher

Study Record Dates

• First Submitted: January 16, 2023
• First Posted: February 8, 2023
• Study Start: February 9, 2023
• Primary Completion: September 15, 2023
• Study Completion: September 15, 2023
• Last Updated: September 21, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

NO (1)