Trial · NCT07903376
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
NCT07903376, Early phase 1, Not yet recruiting, Exir Gene Pharmed
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE). Trials, record NCT07903376. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07903376
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
- NCT number
- NCT07903376
- Status
- Not yet recruiting
- Phases
- Early phase 1
- Enrollment
- 1 participants, estimated
- Lead sponsor
- Exir Gene Pharmed
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- SPX-01
- First posted
- 2026-10-29
- Last updated
- 2026-10-29
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Occurrence and Severity of Adverse Events and Serious Adverse Events | Through Month 12 | Number, nature, severity, seriousness, timing, outcome, and assessed relationship to SPX-01 of adverse events, serious adverse events, and dose-limiting toxicities using DAIDS grading criteria |
| Primary | Change in Alanine Aminotransferase (ALT) Levels From Baseline | Baseline through Month 12 | Change in serum ALT levels from baseline to each scheduled post-treatment assessment. ALT concentrations will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Aspartate Aminotransferase (AST) Levels From Baseline | Baseline through Month 12 | Change in serum AST levels from baseline to each scheduled post-treatment assessment. AST concentrations will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Platelet Count From Baseline | Baseline through Month 12 | Change in platelet count from baseline to each scheduled post-treatment assessment. Platelet counts will be measured in ×10⁹/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Serum Creatinine Levels From Baseline | Baseline through Month 12 | Change in serum creatinine levels from baseline to each scheduled post-treatment assessment as an indicator of renal function. Serum creatinine will be measured in mg/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Cardiac Troponin I Levels From Baseline | Baseline through Month 12 | Change in cardiac troponin I levels from baseline to each scheduled post-treatment assessment as a marker of myocardial injury. Troponin I levels will be measured in ng/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Lactate Dehydrogenase (LDH) Levels From Baseline | Baseline through Month 12 | Change in serum lactate dehydrogenase (LDH) levels from baseline to each scheduled post-treatment assessment as a marker of hemolysis and tissue injury. LDH levels will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Hemoglobin Levels From Baseline | Baseline through Month 12 | Change in blood hemoglobin concentration from baseline to each scheduled post-treatment assessment as an indicator of hematologic safety and potential hemolysis. Hemoglobin levels will be measured in g/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Total Bilirubin Levels From Baseline | Baseline through Month 12 | Change in serum total bilirubin levels from baseline to each scheduled post-treatment assessment as an indicator of hepatic safety and potential hemolysis. Total bilirubin levels will be measured in mg/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Occurrence of Thrombotic Microangiopathy (TMA) Following SPX-01 Administration | Through Month 12 | Assessment of the occurrence of thrombotic microangiopathy (TMA) in the participant following SPX-01 administration, based on clinical and laboratory findings consistent with microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury. Suspected TMA will undergo clinical evaluation and diagnostic confirmation according to the study protocol. TMA occurrence will be reported as present or absent (Yes/No) during the follow-up period. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40522061 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07903376
GET https://conductscience.org/api/trials/v1/NCT07903376/registered?page=2