Autism Spectrum DisorderPhase 2NCT06058962
Double Blind Trial in Children With Autism Spectrum Disorder
A Phase 2 study testing Suramin Sodium in people with Autism Spectrum Disorder, run by Paxmedica at 1 site in United States.
Trial summary
This study investigated the safe and efficacious use of suramin as a novel treatment for ASD by testing two suramin doses vs placebo Read more about this study
- Who is it for?
- Ages 4 to 17 years, men only
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Suramin Sodium
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2019to 15 Mar 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male children aged 4 - 17 years
- Participants with or without treatment interventions for ASD
- Participants must have been diagnosed with ASD by Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-V)
- Autism diagnostic observation schedule, version 2 (ADOS-2) Comparison scores in the
- moderate and high level as evaluated on the ADOS-2
- Stable treatment intervention for ≥ 2 months
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- Participants agreed to not change their treatment interventions throughout the study
- duration
- Participants on Ritalin and Risperdal or similar medication agreed to not change their
- dose during the study
Who cannot join
- Hospitalization within the previous 2 months
- An acute medical problem, Rett syndrome, microcephaly, tuberous sclerosis, neurofibromatosis, epilepsy, or clinically significant liver, kidney, or adrenal disease or persons suffering from any serious acute condition where, in the investigator's opinion, the participant's well-being may have been compromised
- Planning to start a new drug, diet, or behavioral intervention during the study
- Weight under the 5th percentile for age
- Unable to tolerate venipuncture, urine collection, or an indwelling IV catheter for 3-4 hours
- Plasma creatinine above normal for age and weight according to the laboratory reference ranges.
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- Liver function alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 1.5-fold above the upper limit of normal
- Known intolerance to suramin or other antipurinergic drugs
- Unable to perform or cooperate with study requirements
- Children with known syndromic forms of ASD caused by DNA mutation or chromosomal copy number variation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Three-Arm, Prospective, Randomized, Double Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of 2 Doses of Suramin vs. Placebo in Male Children With ASD Receiving Standard Treatment
This study investigated the safe and efficacious use of suramin as a novel treatment for ASD by testing two suramin doses vs placebo
Read the detailed description
PUR-ONQ-ASD-001 was a multicenter, 3-arm, prospective, randomized, double blind, placebo-controlled multiple dose trial involving 2 dose levels of suramin vs. placebo. The study randomized 52 male participants who received treatment interventions for ASD; each treatment arm was assigned approximately 17 participants. The number of participants was increased to 52 participants (48 originally planned) due to early withdrawals related to the COVID-19 global pandemic lockdowns and other matters. Participants were stratified by age, ADOS-2, and Non-verbal Intelligence Quotient (NVIQ). Participants who met all the inclusion criteria and none of the exclusion criteria were randomized through an electronic system to either arm A (10 mg/kg suramin) or Arm B (20 mg/kg suramin) or Arm C (Placebo) in a targeted 1:1:1 ratio, as per the randomization schedule and stratified by age, ADOS-2, and NVIQ. All participants received a 50 mg test dose at their first administration (0.5 mL of freshly reconstituted 10% solution in 5 mL saline) to check for allergic reactions. The test dose was given by slow intravenous (IV) infusion for 30 minutes then flushed with 10 mL saline. Vitals were checked for 30 min and if there were no changes or evidence of allergic reaction, the rest of the study drug dose was administered (10 mg/kg or 20 mg/kg minus test dose [minus 50 mg] in a 50 mL saline up to a maximum of 1 g given over 30 minutes). Arm A - 50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.
Arm B - 50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.
Arm C - Test dose of saline placebo, then saline placebo IV infusion of 50 mL administered over 30 minutes was given at Visits 2, 4 and 5
- Conditions
- Autism Spectrum Disorder
- Being tested
- Suramin Sodium
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Suramin SodiumDrug
Also called Placebo
Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.
| Study group | Role | What they receive |
|---|---|---|
| Suramin 10 mg/kg | Experimental | 50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5. Receives: Drug: Suramin Sodium |
| Suramin 20 mg/kg | Experimental | 50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5. Receives: Drug: Suramin Sodium |
| Placebo | Placebo comparator | Test dose of saline placebo, then saline placebo infusion of 50 mL administered over 30 minutes was given at Visits 2, 4, and 5. Receives: Drug: Suramin Sodium |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary objective was to evaluate the safety of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.
Adverse Events and Laboratory Results
Measured: 98 Days
Secondary outcomes (1)
The primary objective was to evaluate the efficacy of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.
Aberrant Behavior Checklist (ABC) and (Clinical Global Impression) CGI scales used to evaluate efficacy.
ABC scoring is a 0 as improvement with a 3 as a problem being severe.
For each item, decide whether the behavior is a problem for your child/adolescent and select the appropriate number/response:
"0 = not at all a problem
- = the behavior is a problem but slight in degree
- = the problem is moderately serious
- = the problem is severe in degree
Measured: 98 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start1 Jun 2019
- Primary completion15 Jan 2021
- Study completion15 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The participants were randomized to one of three double-blind treatment groups, i.e., Arm A (10 mg/kg suramin) or Arm B (20 mg/kg suramin) or Arm C (placebo) in a targeted 1:1:1 ratio, as per the randomization schedule and stratification plan. The stratification plan was to match patients by age (\< 7 vs ≥ 7), ADOS-2 comparison scores (≤ 8.5 vs \> 8.5) and NVIQ (≤ 80 vs \> 80)
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Paxmedica
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- jennifer L bonfrisco, Study director, Paxmedica
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06058962
- Sponsor's protocol ID
- PUR-ONQ-ASD-001
- Status last verified
- Nov 2023
- First submitted
- 18 Sep 2023
- Last updated
- 13 Nov 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Abstract and CSR
Publications
Hough D, Mao AR, Aman M, Lozano R, Smith-Hicks C, Martinez-Cerdeno V, Derby M, Rome Z, Malan N, Findling RL. Randomized clinical trial of low dose suramin intravenous infusions for treatment of autism spectrum disorder. Ann Gen Psychiatry. 2023 Nov 6;22(1):45. doi: 10.1186/s12991-023-00477-8.
PubMed 37932739
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Three-Arm, Prospective, Randomized, Double Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of 2 Doses of Suramin vs. Placebo in Male Children With ASD Receiving Standard Treatment
- ClinicalTrials.gov ID
- NCT06058962
- Acronym / sponsor ID
- PUR-ONQ-ASD-001
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- PaxmedicaIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2019 (actual)
- Primary completion
- 15 Jan 2021 (actual)
- Study completion
- 15 Mar 2021 (actual)
- First posted
- 28 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study investigated the safe and efficacious use of suramin as a novel treatment for ASD by testing two suramin doses vs placebo
Detailed description
PUR-ONQ-ASD-001 was a multicenter, 3-arm, prospective, randomized, double blind, placebo-controlled multiple dose trial involving 2 dose levels of suramin vs. placebo. The study randomized 52 male participants who received treatment interventions for ASD; each treatment arm was assigned approximately 17 participants. The number of participants was increased to 52 participants (48 originally planned) due to early withdrawals related to the COVID-19 global pandemic lockdowns and other matters. Participants were stratified by age, ADOS-2, and Non-verbal Intelligence Quotient (NVIQ). Participants who met all the inclusion criteria and none of the exclusion criteria were randomized through an electronic system to either arm A (10 mg/kg suramin) or Arm B (20 mg/kg suramin) or Arm C (Placebo) in a targeted 1:1:1 ratio, as per the randomization schedule and stratified by age, ADOS-2, and NVIQ. All participants received a 50 mg test dose at their first administration (0.5 mL of freshly reconstituted 10% solution in 5 mL saline) to check for allergic reactions. The test dose was given by slow intravenous (IV) infusion for 30 minutes then flushed with 10 mL saline. Vitals were checked for 30 min and if there were no changes or evidence of allergic reaction, the rest of the study drug dose was administered (10 mg/kg or 20 mg/kg minus test dose [minus 50 mg] in a 50 mL saline up to a maximum of 1 g given over 30 minutes). Arm A - 50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.
Arm B - 50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.
Arm C - Test dose of saline placebo, then saline placebo IV infusion of 50 mL administered over 30 minutes was given at Visits 2, 4 and 5
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- The participants were randomized to one of three double-blind treatment groups, i.e., Arm A (10 mg/kg suramin) or Arm B (20 mg/kg suramin) or Arm C (placebo) in a targeted 1:1:1 ratio, as per the randomization schedule and stratification plan. The stratification plan was to match patients by age (< 7 vs ≥ 7), ADOS-2 comparison scores (≤ 8.5 vs > 8.5) and NVIQ (≤ 80 vs > 80)
| Arm | Type | Receives |
|---|---|---|
| Suramin 10 mg/kg | Experimental | Drug: Suramin Sodium50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5. |
| Suramin 20 mg/kg | Experimental | Drug: Suramin Sodium50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5. |
| Placebo | Placebo comparator | Drug: Suramin SodiumTest dose of saline placebo, then saline placebo infusion of 50 mL administered over 30 minutes was given at Visits 2, 4, and 5. |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Male children aged 4 - 17 years
- Participants with or without treatment interventions for ASD
- Participants must have been diagnosed with ASD by Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-V)
- Autism diagnostic observation schedule, version 2 (ADOS-2) Comparison scores in the
- moderate and high level as evaluated on the ADOS-2
Key exclusion criteria (10 listed)
- Hospitalization within the previous 2 months
- An acute medical problem, Rett syndrome, microcephaly, tuberous sclerosis, neurofibromatosis, epilepsy, or clinically significant liver, kidney, or adrenal disease or persons suffering from any serious acute condition where, in the investigator's opinion, the participant's well-being may have been compromised
- Planning to start a new drug, diet, or behavioral intervention during the study
- Weight under the 5th percentile for age
- Unable to tolerate venipuncture, urine collection, or an indwelling IV catheter for 3-4 hours
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Suramin SodiumAlso: PlaceboSuramin sodium is a sodium salt form of suramin, an antitrypanosomal compound. | Drug | Placebo, Suramin 10 mg/kg, Suramin 20 mg/kg |
Primary outcome
The primary objective was to evaluate the safety of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.
Time frame: 98 Days
Adverse Events and Laboratory Results
1 secondary outcome
The primary objective was to evaluate the efficacy of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.
Time frame: 98 Days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2023.
- Derived from this study: PubMed 37932739Hough D, Mao AR, Aman M, Lozano R, Smith-Hicks C, Martinez-Cerdeno V, Derby M, Rome Z, Malan N, Findling RL. Randomized clinical trial of low dose suramin intravenous infusions for treatment of autism spectrum disorder. Ann Gen Psychiatry. 2023 Nov 6;22(1):45. doi: 10.1186/s12991-023-00477-8.
- Individual participant dataSharing planned: No. Abstract and CSR
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
jennifer L bonfrisco study director
Paxmedica
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Suramin Sodium in people with Autism Spectrum Disorder, run by Paxmedica at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary objective was to evaluate the safety of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment. (time frame: 98 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06058962, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Nov 2023.