<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03776656</url>
  </required_header>
  <id_info>
    <org_study_id>P160902J</org_study_id>
    <secondary_id>2017-002155-28</secondary_id>
    <nct_id>NCT03776656</nct_id>
  </id_info>
  <brief_title>Evaluation of a Treatment With Allopurinol in Adenylosuccinate Lyase Deficiency</brief_title>
  <acronym>ADSL</acronym>
  <official_title>Evaluation of a Treatment With Allopurinol on Autistic Disorders and Epilepsy in Adenylosuccinate Lyase Deficiency (ADSL)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Assistance Publique - Hôpitaux de Paris</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>URC-CIC Paris Descartes Necker Cochin</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Assistance Publique - Hôpitaux de Paris</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The aim of this study is to evaluate the effectiveness of allopurinol treatment at 12 months&#xD;
      on the adaptive and cognitive functioning of patients with adenylosuccinate lyase deficiency&#xD;
      (ADSL). The psychiatric evaluation will involve the use of standardized tools prior to&#xD;
      initiation of treatment, and will be repeated 6 months and 12 months after the start of&#xD;
      treatment.&#xD;
&#xD;
      The decrease in the concentration of SAICAR and S-Ado metabolites, which are markers of&#xD;
      adenylosuccinate lyase (ADSL) deficiency, will also be quantified.&#xD;
&#xD;
      Similarly, the efficacy of allopurinol on epileptic seizures for epileptic patients and on&#xD;
      electrocardiogram abnormalities will be evaluated secondarily&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Adenylosuccinate lyase deficiency (ADSL) is a rare disorder of purine metabolism whose&#xD;
      symptoms are mental retardation, autistic disorders, epilepsy, related to the accumulation of&#xD;
      succinylpurines: succinylaminoimidazole carboxamide riboside (SAICAr) and succinyladenosine&#xD;
      (S- Ado). The S-Ado / SAICAr ratio in the cerebrospinal fluid (CSF) is correlated with the&#xD;
      clinical severity: the cerebral toxicity of SAICAr is incriminated. There is no specific&#xD;
      treatment.&#xD;
&#xD;
      Based on the work of Gertrude B. Elion (1988 Nobel Prize in Medicine), who reports that&#xD;
      allopurinol (a structural analogue of hypoxanthine) can be a substrate for hypoxanthine&#xD;
      phosphoribosyltransferase (HPRT) and thus produce allopurinol ribonucleotides with as a first&#xD;
      step in the de novo synthesis of purines, investigators tested the hypothesis that treatment&#xD;
      with allopurinol in children with ADSL deficiency would reduce the production of the toxic&#xD;
      metabolite SAICAr.&#xD;
&#xD;
      This hypothesis was validated in 3 minor patients with biological and clinical improvement.&#xD;
&#xD;
      So the investigators put the phase II, non-comparative study based on 4 visits to&#xD;
      Necker-Enfants malades Hospital or La Pitié-Salpêtrière Hospital: Month 0 (before treatment),&#xD;
      Month 3, Month 6 and Month 12 after the start of treatment.&#xD;
&#xD;
      After verification of the inclusion criteria and information of the parents or the patient or&#xD;
      guardian, signature of the consent and inclusion of the patient:&#xD;
&#xD;
        -  Clinical and neurological evaluation;&#xD;
&#xD;
        -  Psychiatric assessment with standardized tests;&#xD;
&#xD;
        -  Biological evaluation: determination of urinary and plasma metabolites (SAICAr, S-Ado,&#xD;
           ...) Experimental treatment: Allopurinol (Zyloric®) will be administered orally for 12&#xD;
           months without exceeding 400 mg / day in children and 900 mg / day in adults, with&#xD;
           dosage adjustment in case of renal failure.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 14, 2019</start_date>
  <completion_date type="Actual">June 17, 2022</completion_date>
  <primary_completion_date type="Actual">June 17, 2022</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Measurement of adaptive functional improvement : composite total score for Vineland II adaptive behaviour Scale</measure>
    <time_frame>12 months</time_frame>
    <description>to assess Efficacy of Allopurinol (Zyloric)® treatment from Baseline : For each scale : Mean : 100 SD : 15&#xD;
Adaptive behaviour composite : Range 20 to 180 -Domains scores : Range 20 to 140 -Communication : Range 20 to 140 -Daily living skills : Range 20 to 140 -Socialization : 20 to 140 -Motor skills : 20 to 140 For each scale values are considered to be better or worse outcome :High 130 to 140 -Moderately High 115 to 129 -Adequate 86 to 114 -Moderately Low 71 to 85 -Low 20 to 70 Total score is obtained by summing the subdomains scores</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Scores of different subdomains Vineland II scale from baseline</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Clinical evolution for developmental and cognitive assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Psycho-Educative Profile (PEP III/R) from baseline</measure>
    <time_frame>at 0, 12 months</time_frame>
    <description>Clinical evolution for developmental and cognitive assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Score ADI-R (Autism Diagnostic Interview-Revised) from baseline</measure>
    <time_frame>at 0, 12 months</time_frame>
    <description>Clinical evaluation for autistic symptoms : scale ranges :&#xD;
A : Social interactions: 0 to 30 (significative if &gt;10)&#xD;
B : Communication : 0 to 26 (significative if &gt; 7 or 8)&#xD;
C : Repetitive and restricted interests : 0 to 16 (significative if &gt;3)&#xD;
D : Developmental abnormality present before 36 months : 0 to 5 (significative if &gt;1)&#xD;
better score : 0 (non autistic) - worse score : the higher score is the worst</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Score ADOS-2 (Autism Diagnostic Observation Schedule 2) from baseline</measure>
    <time_frame>at 0, 12 months</time_frame>
    <description>Clinical evaluation for autistic symptoms. Scale ranges :&#xD;
A : Socialization : social interactions and communication : 0 to 20 (social interactions : 0 to 6 -communication : 0 to 14)&#xD;
B : Restricted and repetitive interests : 0 to 8&#xD;
Total : A+B : 0 to 28&#xD;
Autism : total score&gt;12 - Autism Spectrum Disorder : total score &gt;8&#xD;
Better score : 0 - Worse score : the highest score is the worst</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Score on Conners hyperactivity Scale</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Clinical evolution for behavioral disorders and adaptive functioning&#xD;
Conners Scale for Parents :&#xD;
- Subscales : Behavioural difficulties (items 2-8-14-19-20-27-35-39) : 0 to 24 Learning difficulties (items 10-25-31-37) : 0 to 12 Somatisation (items 32-41-43-44) : 0 to 12 Impulsivity, hyperactivity (items 4-5-11-13): 0 to 12 Anxiety (items 12-16-24-47): 0 to 12&#xD;
-Hyperactivity index : sum of the items (4-7-11-13-14-25-31-33-37-38) divided by 10 : 0 to 3&#xD;
Conners Scale for teachers :&#xD;
- Subscales : Behavioural difficulties (items 4-5-6-10-11-12-23-27) : 0 to 24 Impulsivity, hyperactivity (items 1-2-3-8-14-15-16) : 0 to 21 Inattention, passivity (items 7-9-18-20-21-22-26-28) : 0 to 24&#xD;
-Hyperactivity index (sum of the items 1-5-7-8-10-11-14-15-21-26 divided by 10) : 0 to 3&#xD;
Better score : 0 - Worse score : the highest score is the worst -Hyperactivity index : Significative if&gt; 1,5</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the Score on ABC scale (Aberrant Behaviour Checklist)</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Clinical evolution for behavioral disorders and adaptive functioning scale ranges :&#xD;
Irritability : 0 to 45&#xD;
Lethargy : 0 to 48&#xD;
Stereotypy : 0 to 21&#xD;
Hyperactivity : 0 to 48&#xD;
Inappropriate speech : 0 to 12&#xD;
Better score : 0 -Worse score : the highest score is the worst on each scale- there is no total score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of SAICAr levels in the urine</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Evolution of the quantity of urinary metabolites from Baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of S-Ado levels in the urine</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Evolution of the quantity of urinary metabolites from Baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of SAICAr levels in the blood</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Evolution of the quantity of plasma metabolites from Baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of S-Ado levels in the blood</measure>
    <time_frame>at 0, 6 months and 12 months</time_frame>
    <description>Evolution of the quantity of plasma metabolites from Baseline</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of the number of seizures from Baseline for epileptic patients</measure>
    <time_frame>at 0 and 12 months</time_frame>
    <description>at baseline, performing neurological examinations and interrogation</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of antiepileptic treatments from Baseline for epileptic patients</measure>
    <time_frame>at 0 and 12 months</time_frame>
    <description>at baseline, performing neurological examinations and interrogation</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evolution of electroencephalogram tracing from Baseline for epileptic patients</measure>
    <time_frame>at 0 and 12 months</time_frame>
    <description>normal/abnormal</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">8</enrollment>
  <condition>Adenylosuccinate Lyase Deficiency</condition>
  <arm_group>
    <arm_group_label>Allopurinol</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Oral administration of Allopurinol (Zyloric®) for 12 months without exceeding 400 mg / day in children and 900 mg / day in adults, with dosage adjustment in case of renal failure</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Allopurinol</intervention_name>
    <description>Daily oral administration</description>
    <arm_group_label>Allopurinol</arm_group_label>
    <other_name>Zyloric®</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Child (minimum age 18 months) or adult with adenylosuccinate lyase; deficiency (ADSL)&#xD;
             confirmed by quantification of SAICAr and S-Ado urinary;&#xD;
&#xD;
          -  Girls / women of childbearing age must:&#xD;
&#xD;
               -  have a negative pregnancy test;&#xD;
&#xD;
               -  agree to use a reliable method of contraception from the baseline visit to the&#xD;
                  last dose of study treatment&#xD;
&#xD;
          -  Consent of the patient, his parents or his legal representative;&#xD;
&#xD;
          -  Beneficiary of social security (affiliated or entitled).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Refusal of the child, his parents or the patient or his representative;&#xD;
&#xD;
          -  Allergy known to allopurinol or to one of the constituents of the product (lactose in&#xD;
             particular);&#xD;
&#xD;
          -  Patients treated with Antipurines (azathioprine, mercaptopurine);&#xD;
&#xD;
          -  Patients treated with vidarabine, cytotoxic drugs (eg cyclophosphamide, doxorubicin,&#xD;
             bleomycin, procarbazine, alkyl halides), ciclosporin, or didanosine&#xD;
&#xD;
          -  Renal failure characterized by creatinine clearance &lt;80 ml/mn&#xD;
&#xD;
          -  Hepatic insufficiency&#xD;
&#xD;
          -  Medullary insufficiency but possibly serious&#xD;
&#xD;
          -  Breastfeeding&#xD;
&#xD;
          -  Pregnancy or wishing to conceive during the study period&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Months</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Pascale De LONLAY, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Assistance Publique - Hôpitaux de Paris</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Irène CEBALLOS-PICOT, MD, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Assistance Publique - Hôpitaux de Paris</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Laurence ROBEL-GALLI, MD</last_name>
    <role>Study Director</role>
    <affiliation>Assistance Publique - Hôpitaux de Paris</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>LA PITIE-SALPETRIERE Hospital, AP-HP</name>
      <address>
        <city>Paris</city>
        <zip>75013</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Pediatry. Reference centre of Hereditary diseases of the metabolism of child and adult. Necker - Enfants malades Hospital</name>
      <address>
        <city>Paris</city>
        <zip>75015</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>October 2022</verification_date>
  <study_first_submitted>August 13, 2018</study_first_submitted>
  <study_first_submitted_qc>December 12, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 17, 2018</study_first_posted>
  <last_update_submitted>October 10, 2022</last_update_submitted>
  <last_update_submitted_qc>October 10, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">October 12, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Adenylosuccinate lyase</keyword>
  <keyword>Deficit</keyword>
  <keyword>Allopurinol</keyword>
  <keyword>Autism</keyword>
  <keyword>Epilepsy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Purine-Pyrimidine Metabolism, Inborn Errors</mesh_term>
    <mesh_term>Autistic Disorder</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Allopurinol</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03776656
org study id: P160902J
secondary id: 2017-002155-28
nct id: NCT03776656
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The aim of this study is to evaluate the effectiveness of allopurinol treatment at 12 months
      on the adaptive and cognitive functioning of patients with adenylosuccinate lyase deficiency
      (ADSL). The psychiatric evaluation will involve the use of standardized tools prior to
      initiation of treatment, and will be repeated 6 months and 12 months after the start of
      treatment.

      The decrease in the concentration of SAICAR and S-Ado metabolites, which are markers of
      adenylosuccinate lyase (ADSL) deficiency, will also be quantified.

      Similarly, the efficacy of allopurinol on epileptic seizures for epileptic patients and on
      electrocardiogram abnormalities will be evaluated secondarily
Adenylosuccinate lyase deficiency (ADSL) is a rare disorder of purine metabolism whose
      symptoms are mental retardation, autistic disorders, epilepsy, related to the accumulation of
      succinylpurines: succinylaminoimidazole carboxamide riboside (SAICAr) and succinyladenosine
      (S- Ado). The S-Ado / SAICAr ratio in the cerebrospinal fluid (CSF) is correlated with the
      clinical severity: the cerebral toxicity of SAICAr is incriminated. There is no specific
      treatment.

      Based on the work of Gertrude B. Elion (1988 Nobel Prize in Medicine), who reports that
      allopurinol (a structural analogue of hypoxanthine) can be a substrate for hypoxanthine
      phosphoribosyltransferase (HPRT) and thus produce allopurinol ribonucleotides with as a first
      step in the de novo synthesis of purines, investigators tested the hypothesis that treatment
      with allopurinol in children with ADSL deficiency would reduce the production of the toxic
      metabolite SAICAr.

      This hypothesis was validated in 3 minor patients with biological and clinical improvement.

      So the investigators put the phase II, non-comparative study based on 4 visits to
      Necker-Enfants malades Hospital or La Pitié-Salpêtrière Hospital: Month 0 (before treatment),
      Month 3, Month 6 and Month 12 after the start of treatment.

      After verification of the inclusion criteria and information of the parents or the patient or
      guardian, signature of the consent and inclusion of the patient:

        -  Clinical and neurological evaluation;

        -  Psychiatric assessment with standardized tests;

        -  Biological evaluation: determination of urinary and plasma metabolites (SAICAr, S-Ado,
           ...) Experimental treatment: Allopurinol (Zyloric®) will be administered orally for 12
           months without exceeding 400 mg / day in children and 900 mg / day in adults, with
           dosage adjustment in case of renal failure.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Measurement of adaptive functional improvement : composite total score for Vineland II adaptive behaviour Scale
time frame: 12 months
description: to assess Efficacy of Allopurinol (Zyloric)® treatment from Baseline : For each scale : Mean : 100 SD : 15
Adaptive behaviour composite : Range 20 to 180 -Domains scores : Range 20 to 140 -Communication : Range 20 to 140 -Daily living skills : Range 20 to 140 -Socialization : 20 to 140 -Motor skills : 20 to 140 For each scale values are considered to be better or worse outcome :High 130 to 140 -Moderately High 115 to 129 -Adequate 86 to 114 -Moderately Low 71 to 85 -Low 20 to 70 Total score is obtained by summing the subdomains scores
measure: Evolution of the Scores of different subdomains Vineland II scale from baseline
time frame: at 0, 6 months and 12 months
description: Clinical evolution for developmental and cognitive assessment
measure: Evolution of the Psycho-Educative Profile (PEP III/R) from baseline
time frame: at 0, 12 months
description: Clinical evolution for developmental and cognitive assessment
measure: Evolution of the Score ADI-R (Autism Diagnostic Interview-Revised) from baseline
time frame: at 0, 12 months
description: Clinical evaluation for autistic symptoms : scale ranges :
A : Social interactions: 0 to 30 (significative if >10)
B : Communication : 0 to 26 (significative if > 7 or 8)
C : Repetitive and restricted interests : 0 to 16 (significative if >3)
D : Developmental abnormality present before 36 months : 0 to 5 (significative if >1)
better score : 0 (non autistic) - worse score : the higher score is the worst
measure: Evolution of the Score ADOS-2 (Autism Diagnostic Observation Schedule 2) from baseline
time frame: at 0, 12 months
description: Clinical evaluation for autistic symptoms. Scale ranges :
A : Socialization : social interactions and communication : 0 to 20 (social interactions : 0 to 6 -communication : 0 to 14)
B : Restricted and repetitive interests : 0 to 8
Total : A+B : 0 to 28
Autism : total score>12 - Autism Spectrum Disorder : total score >8
Better score : 0 - Worse score : the highest score is the worst
measure: Evolution of the Score on Conners hyperactivity Scale
time frame: at 0, 6 months and 12 months
description: Clinical evolution for behavioral disorders and adaptive functioning
Conners Scale for Parents :
- Subscales : Behavioural difficulties (items 2-8-14-19-20-27-35-39) : 0 to 24 Learning difficulties (items 10-25-31-37) : 0 to 12 Somatisation (items 32-41-43-44) : 0 to 12 Impulsivity, hyperactivity (items 4-5-11-13): 0 to 12 Anxiety (items 12-16-24-47): 0 to 12
-Hyperactivity index : sum of the items (4-7-11-13-14-25-31-33-37-38) divided by 10 : 0 to 3
Conners Scale for teachers :
- Subscales : Behavioural difficulties (items 4-5-6-10-11-12-23-27) : 0 to 24 Impulsivity, hyperactivity (items 1-2-3-8-14-15-16) : 0 to 21 Inattention, passivity (items 7-9-18-20-21-22-26-28) : 0 to 24
-Hyperactivity index (sum of the items 1-5-7-8-10-11-14-15-21-26 divided by 10) : 0 to 3
Better score : 0 - Worse score : the highest score is the worst -Hyperactivity index : Significative if> 1,5
measure: Evolution of the Score on ABC scale (Aberrant Behaviour Checklist)
time frame: at 0, 6 months and 12 months
description: Clinical evolution for behavioral disorders and adaptive functioning scale ranges :
Irritability : 0 to 45
Lethargy : 0 to 48
Stereotypy : 0 to 21
Hyperactivity : 0 to 48
Inappropriate speech : 0 to 12
Better score : 0 -Worse score : the highest score is the worst on each scale- there is no total score
measure: Evolution of SAICAr levels in the urine
time frame: at 0, 6 months and 12 months
description: Evolution of the quantity of urinary metabolites from Baseline
measure: Evolution of S-Ado levels in the urine
time frame: at 0, 6 months and 12 months
description: Evolution of the quantity of urinary metabolites from Baseline
measure: Evolution of SAICAr levels in the blood
time frame: at 0, 6 months and 12 months
description: Evolution of the quantity of plasma metabolites from Baseline
measure: Evolution of S-Ado levels in the blood
time frame: at 0, 6 months and 12 months
description: Evolution of the quantity of plasma metabolites from Baseline
measure: Evolution of the number of seizures from Baseline for epileptic patients
time frame: at 0 and 12 months
description: at baseline, performing neurological examinations and interrogation
measure: Evolution of antiepileptic treatments from Baseline for epileptic patients
time frame: at 0 and 12 months
description: at baseline, performing neurological examinations and interrogation
measure: Evolution of electroencephalogram tracing from Baseline for epileptic patients
time frame: at 0 and 12 months
description: normal/abnormal
arm group label: Allopurinol
arm group type: Experimental
description: Oral administration of Allopurinol (Zyloric®) for 12 months without exceeding 400 mg / day in children and 900 mg / day in adults, with dosage adjustment in case of renal failure
intervention type: Drug
intervention name: Allopurinol
description: Daily oral administration
arm group label: Allopurinol
other name: Zyloric®
criteria: 
gender: All
minimum age: 18 Months
maximum age: N/A
healthy volunteers: No
last name: Pascale De LONLAY, MD, PhD
role: Principal Investigator
affiliation: Assistance Publique - Hôpitaux de Paris
last name: Irène CEBALLOS-PICOT, MD, PhD
role: Study Director
affiliation: Assistance Publique - Hôpitaux de Paris
last name: Laurence ROBEL-GALLI, MD
role: Study Director
affiliation: Assistance Publique - Hôpitaux de Paris
facility: 
facility: 
country: France
responsible party type: Sponsor
mesh term: Purine-Pyrimidine Metabolism, Inborn Errors
mesh term: Autistic Disorder
mesh term: Allopurinol
sharing ipd: No