Testing granulocyte-colony stimulating factor (GCSF) in patients with Friedreich Ataxia

The pharmacological effects of granulocyte-colony stimulating factor (GCSF) on frataxin expression in patients with Friedreich Ataxia - GCSF for Friedreich Ataxia

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-003084-34-GB

Enrollment

Registered

2017-11-28

Start date

2018-01-22

Completion date

Unknown

Last updated

2020-02-01

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

Conditions

Friedreich Ataxia MedDRA version: 20.0 Level: LLT Classification code 10003592 Term: Ataxia cerebellar System Organ Class: 100000004852 MedDRA version: 20.0 Level: PT Classification code 10017374 Term: Friedreich's ataxia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Granocyte Product Name: Granocyte Pharmaceutical Form: Injection INN or Proposed INN: rHuG-CSF Other descriptive name: Lenog

Eligibility

Inclusion criteria

Inclusion criteria: Genetic diagnosis of FRDA Age of over 18 and under 60 Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 7 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: Pregnancy, breastfeeding or lactation Significant abnormalities on baseline bloods (full blood count, renal and liver function) Previous diagnosis of haematological disorder (including malignancy) Previous history of splenomegaly Previous history of autoimmune disease Previous history of pulmonary infiltrates, pulmonary fibrosis or haemoptysis

Design outcomes

Primary

Measure	Time frame
Main Objective: Does administration of GCSF lead to improvements in blood markers of Friedreich Ataxia? We will study a small number of patients with the condition and will administer GCSF (at identical doses to those given to ‘healthy’ people prior to bone marrow donation) for a short period of time. We will define whether administration of the drug leads to changes in blood markers which would indicate a positive response to the drug. The study will also allow us to decide what blood markers we can monitor in the subsequent trial. This has not been studied before and is a vital step in the development of a stem cell research trial. Once information has been obtained from this study, a larger trial of GCSF in FRDA can be developed. ;Secondary Objective: Is administration of a single course of GCSF to people with FRDA safe?;Primary end point(s): Frataxin gene and protein expression in peripheral blood cells after GCSF administration in patients with FRDA ;Timepoint(s) of evaluation of this end point: Assessed in blood samples taken on day 5, 6, 8, 10, 14 and 19 (from the start of the drug dosing, so blood sampling will occur initially on the last day of dosing)	—

Measure

Time frame

Main Objective: Does administration of GCSF lead to improvements in blood markers of Friedreich Ataxia? We will study a small number of patients with the condition and will administer GCSF (at identical doses to those given to ‘healthy’ people prior to bone marrow donation) for a short period of time. We will define whether administration of the drug leads to changes in blood markers which would indicate a positive response to the drug. The study will also allow us to decide what blood markers we can monitor in the subsequent trial. This has not been studied before and is a vital step in the development of a stem cell research trial. Once information has been obtained from this study, a larger trial of GCSF in FRDA can be developed. ;Secondary Objective: Is administration of a single course of GCSF to people with FRDA safe?;Primary end point(s): Frataxin gene and protein expression in peripheral blood cells after GCSF administration in patients with FRDA ;Timepoint(s) of evaluation of this end point: Assessed in blood samples taken on day 5, 6, 8, 10, 14 and 19 (from the start of the drug dosing, so blood sampling will occur initially on the last day of dosing)

—

Secondary

Measure	Time frame
Secondary end point(s): Safety of administration of GCSF in patients with FRDA;Timepoint(s) of evaluation of this end point: Baseline observations (pulse, blood pressure and temperature) and routine laboratory blood work-up (full blood count, liver and renal function) will be taken 2 weeks after drug administration has finished (day 19).	—

Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 14, 2026