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Completed

NCT Number: NCT01255358

Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).

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Key information

Conditions

Nervous System Disorder Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Syndrome Nervous System Diseases

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Spedali Civili, Brescia, Italy

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About this study

This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • neurological signs of AT
  • patients in autonomous gait or helped by a support
  • proven molecular diagnosis of AT
  • Males and females aged > 3 years
  • Body weight >15 kg
  • Plasma levels of Lymphocytes CD4+/mm3 > 500 (for patients aged 3-6 years) or > 200 (older than 6 years)
  • written IC to participate.

Exclusion criteria

  • Current or previous neoplastic disease
  • History of severe impairment of the immunological system
  • Chronic conditions representing a contraindication to the use of steroid drugs
  • Non compliance with the study request
  • Any previous steroid assumption within 30 days before starting ERY-DEX
  • Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
  • Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods

Treatment and study plan

Dexamethasone

Drug

Dexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).

The treatment has to be repeated at intervals of 30 days (±10 days)

Other names: Dexamethasone sodium phosphate, Dex 21P

Primary outcomes

  1. Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population

    Time frame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)

    ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.

  2. Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population

    Time frame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)

    ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.

Secondary outcomes

  1. Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population

    Time frame: At visit 7 (final visit, day 180)

    An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:

    • very much improved (1),
    • slightly improved (2),
    • no change (3),
    • slightly worsened (4),
    • very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.

  2. Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population

    Time frame: At visit 7 (final visit, day 180)

    An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:

    • very much improved (1),
    • slightly improved (2),
    • no change (3),
    • slightly worsened (4),
    • very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.

  3. Mean Change From V4 to V7 in Ocular Motility - ITT Population

    Time frame: At visit 7 (final visit, day 180)

    An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.

    At V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).

  4. Mean Change From V4 to V7 in Ocular Motility - PP Population

    Time frame: At visit 7 (final visit, day 180)

    An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.

    At V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).

  5. Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population

    Time frame: At visits 4 (90 days) and 7 (180 days, final visit)

    VABS aggregate scale is used.

    VABS is divided into 4 adaptive domains and 11 subdomains:

    • Communication: Receptive, Expressive, Written;
    • Daily Living Skills: Personal, Domestic, Community;
    • Socialisation: Interpersonal Relationships, Play & Leisure Time, Coping Skills;
    • Motor Skills: Gross, Fine.

    For each *question* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):

    • 0 = the subject never performs the behaviour without help or reminders;
    • 1 = rarely performs (as above);
    • 2 = sometimes performs (as above);
    • 3 = often performs (as above);
    • 4 = almost always performs (as above); The total subdomain score is the sum of each *question* scores and the total domain score is the sum of each subdomains' scores.

    Domains min-max interval scores:

    0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)

  6. Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population

    Time frame: At visits 4 (90 days) and 7 (180 days, final visit)

    VABS aggregate scale is used.

    VABS is divided into 4 adaptive domains and 11 subdomains:

    • Communication: Receptive, Expressive, Written;
    • Daily Living Skills: Personal, Domestic, Community;
    • Socialisation: Interpersonal Relationships, Play & Leisure Time, Coping Skills;
    • Motor Skills: Gross, Fine.

    For each *question* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):

    • 0 = the subject never performs the behaviour without help or reminders;
    • 1 = rarely performs (as above);
    • 2 = sometimes performs (as above);
    • 3 = often performs (as above);
    • 4 = almost always performs (as above); The total subdomain score is the sum of each *question* scores and the total domain score is the sum of each subdomains' scores.

    Domains min-max interval scores:

    0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)

  7. Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)

    Time frame: Throughout the study, until the end of 6 months of treatment (day 180)

    The effect of ERY-DEX on treatment emergent adverse events was expressed as the number of patients showing at least one TEAE or 1 serious AE

Sponsors and collaborators

Lead sponsor

Quince Therapeutics S.p.A.

Industry

Registry information

Official study title

Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients

Acronym: IEDAT-01

Important dates

Study start
2011
Primary completion
2011
Study completion
2011
First posted
Dec 7, 2010
Registry last updated
Oct 10, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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