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).
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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
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)
Spedali Civili
Brescia, Italy
University La Sapienza
Rome, Italy
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
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.
Time frame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population
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.
Time frame: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population
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.
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Time frame: At visit 7 (final visit, day 180)
Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population
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.
Time frame: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - ITT Population
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).
Time frame: At visit 7 (final visit, day 180)
Mean Change From V4 to V7 in Ocular Motility - PP Population
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).
Time frame: At visit 7 (final visit, day 180)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population
VABS aggregate scale is used. VABS is divided into 4 adaptive domains and 11 subdomains: 1. Communication: Receptive, Expressive, Written; 2. Daily Living Skills: Personal, Domestic, Community; 3. Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills; 4. 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)
Time frame: At visits 4 (90 days) and 7 (180 days, final visit)
Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population
VABS aggregate scale is used. VABS is divided into 4 adaptive domains and 11 subdomains: 1. Communication: Receptive, Expressive, Written; 2. Daily Living Skills: Personal, Domestic, Community; 3. Socialisation: Interpersonal Relationships, Play \& Leisure Time, Coping Skills; 4. 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)
Time frame: At visits 4 (90 days) and 7 (180 days, final visit)
Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)
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
Time frame: Throughout the study, until the end of 6 months of treatment (day 180)