Improvement of physical abilities in patients with cystinosis

Prospective study to improve cardiorespiratory performance and muscle strength in patients with cystinosis using the Galileo vibration plate

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN15626850

Enrollment

Registered

2023-09-21

Start date

2020-07-01

Completion date

Unknown

Last updated

2023-10-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Improvement of cardiorespiratory performance and muscle strength in patients with cystinosis Genetic Diseases

Interventions

The patients will be divided into two age groups depending on their age at the time of randomization. Due to the very rare underlying disease, a matched-pair design was chosen as the study design. Pat

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Confirmed diagnosis of infantile nephropathic cystinosis 2. Prepubertal patients from 6 to 13 years of age or from the age of 18 3. Consent of the patient (18 years and older) or legal representative (for minors)

Exclusion criteria

Exclusion criteria: 1. Patients who are likely to undergo a serious intervention (e.g. kidney transplant, orthopedic surgery) during the duration of the study or for whom there are contraindications for training on Galileo vibration platforms according to the official recommendation 2. Adolescents who have started puberty (boys: testicular volume 3 ml, girls: Tanner stage from B2). Since puberty in these patients starts late, the prepubertal age group was extended to the age of 13 years. The pubertal age group was excluded due to the hormonal changes during this period.

Design outcomes

Primary

Measure	Time frame
At baseline, 3, 6, and 12 months: 1. Muscle function tests (power & strength) using Leonardo Mechanograph (Novotec) (Measurement of ground reaction forces) with s2LJ (single two-legged jumps) 2. HRT (Heel-Rise test) 3. m1LH (multiple one-legged hopping on the forefoot with the knee outstretched) 4. Grip strength 5. 6-minute walk test were performed	—

Secondary

Measure	Time frame
Clinical parameters at baseline, 3, 6, and 12 months: 1. Blood pressure (sphygmomanometer) 2. Pulse oximetry 3. Quality of life and wellbeing (children and adolescents: KINDL-R questionnaire, adults SF-36 questionnaire) 4. Clinical (orthopedic surgery, fractures) and laboratory assessment (kidney function, end stage renal disease, dialysis, transplantation, cystine score) physiotherapeutic and orthopedic examination at baseline and 12 months only	—

Measure

Time frame

Clinical parameters at baseline, 3, 6, and 12 months: 1. Blood pressure (sphygmomanometer) 2. Pulse oximetry 3. Quality of life and wellbeing (children and adolescents: KINDL-R questionnaire, adults SF-36 questionnaire) 4. Clinical (orthopedic surgery, fractures) and laboratory assessment (kidney function, end stage renal disease, dialysis, transplantation, cystine score) physiotherapeutic and orthopedic examination at baseline and 12 months only

—

Countries

Germany, Italy

Contacts

Public ContactSonja Froschauer

sonja.froschauer@cystinose-stiftung.de+49 1771760755

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026