CLINICAL TRIALS PROFILE FOR AMINOLEVULINIC ACID HYDROCHLORIDE

All Clinical Trials for aminolevulinic acid hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002963 ↗	Photodynamic Therapy in Treating Patients With Skin Cancer	Completed	Roswell Park Cancer Institute	Phase 2	1993-11-01	RATIONALE: Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill tumor cells. Photodynamic therapy using aminolevulinic acid cream may be effective in treating patients with skin cancer. PURPOSE: This randomized phase II trial is studying how well photodynamic therapy works in treating patients with skin cancer.
NCT00002975 ↗	Photodynamic Therapy in Treating Patients With Skin Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1997-02-01	RATIONALE: Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill tumor cells. Photodynamic therapy using aminolevulinic acid may be effective in treating patients with skin cancer. PURPOSE: This randomized phase II trial is studying how well photodynamic therapy that includes aminolevulinic acid works in treating patients with skin cancer.
NCT00002975 ↗	Photodynamic Therapy in Treating Patients With Skin Cancer	Completed	Roswell Park Cancer Institute	Phase 2	1997-02-01	RATIONALE: Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill tumor cells. Photodynamic therapy using aminolevulinic acid may be effective in treating patients with skin cancer. PURPOSE: This randomized phase II trial is studying how well photodynamic therapy that includes aminolevulinic acid works in treating patients with skin cancer.
NCT00054171 ↗	Photodynamic Therapy in Treating Patients With Lymphoma or Chronic Lymphocytic Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1999-02-01	RATIONALE: Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill cancer cells. Photosensitizing drugs such as aminolevulinic acid are absorbed by cancer cells and, when exposed to light, become active and kill the cancer cells. PURPOSE: Randomized phase II trial to study the effectiveness of photodynamic therapy using aminolevulinic acid in treating patients who have cutaneous T-cell lymphoma, B-cell lymphoma, or early chronic lymphocytic leukemia involving the skin.
NCT00054171 ↗	Photodynamic Therapy in Treating Patients With Lymphoma or Chronic Lymphocytic Leukemia	Completed	Roswell Park Cancer Institute	Phase 2	1999-02-01	RATIONALE: Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill cancer cells. Photosensitizing drugs such as aminolevulinic acid are absorbed by cancer cells and, when exposed to light, become active and kill the cancer cells. PURPOSE: Randomized phase II trial to study the effectiveness of photodynamic therapy using aminolevulinic acid in treating patients who have cutaneous T-cell lymphoma, B-cell lymphoma, or early chronic lymphocytic leukemia involving the skin.
NCT00241670 ↗	Fluorescence-guided Resection of Malignant Gliomas With 5-Aminolevulinic Acid	Completed	medac GmbH	Phase 3	1999-10-01	The aim of the study "Fluorescence-guided resection of malignant gliomas with 5-Aminolevulinic acid (5-ALA) vs. conventional resection" is to determine how accurately contrast agent-accumulating tumour can be removed by primary surgery and to assess the clinical usefulness of this method.
NCT00467610 ↗	Phase 2, Open-Label, Multi-Dose Study of Panhematin in Patients With MDS	Terminated	H. Lundbeck A/S	Phase 2	2007-05-01	This is a Phase II, open-label clinical trial examining the role of Panhematin® in patients with MDS. The objective of this study is to evaluate the safety and efficacy of Panhematin® (hematin for injection) in the treatment of adult patients (≥ 18 years of age) with low-risk MDS. The study will be conducted on an outpatient basis and will consist of the following: - A Screening Period (within 28 days of the Day 1) - Screening bone marrow aspiration and biopsy up to 60 days prior to receiving study medication - An 8-week Treatment Period (Days 1 through 4 of Week 1, and weekly visits during Weeks 2 through 8); partial and complete responders in any of the three cell lines may continue treatment for an additional 4 weeks - A 6-month Post treatment Follow-up Period (monthly clinic visits during Weeks 12 40)

Clinical Trial Conditions for aminolevulinic acid hydrochloride

Condition Name

Condition Name for aminolevulinic acid hydrochloride
Intervention	Trials
Actinic Keratosis	14
Actinic Keratoses	9
Glioblastoma	6
Glioma	5

Condition MeSH

Condition MeSH for aminolevulinic acid hydrochloride
Intervention	Trials
Keratosis, Actinic	28
Keratosis	26
Glioblastoma	13
Glioma	11

Clinical Trial Locations for aminolevulinic acid hydrochloride

Trials by Country

Trials by Country for aminolevulinic acid hydrochloride
Location	Trials
United States	130
Germany	7
China	5
Finland	4
Switzerland	2

Trials by US State

Trials by US State for aminolevulinic acid hydrochloride
Location	Trials
California	14
New York	12
Pennsylvania	11
Illinois	10
Ohio	8

Clinical Trial Progress for aminolevulinic acid hydrochloride

Clinical Trial Phase

Clinical Trial Phase for aminolevulinic acid hydrochloride
Clinical Trial Phase	Trials
PHASE3	3
PHASE2	3
PHASE1	3

Clinical Trial Status

Clinical Trial Status for aminolevulinic acid hydrochloride
Clinical Trial Phase	Trials
Completed	45
Recruiting	23
Terminated	12

Clinical Trial Sponsors for aminolevulinic acid hydrochloride

Sponsor Name

Sponsor Name for aminolevulinic acid hydrochloride
Sponsor	Trials
DUSA Pharmaceuticals, Inc.	14
National Cancer Institute (NCI)	14
Roswell Park Cancer Institute	5

Sponsor Type

Sponsor Type for aminolevulinic acid hydrochloride
Sponsor	Trials
Other	133
Industry	44
NIH	17

Clinical Trials Update, Market Analysis, and Projection for Aminolevulinic Acid Hydrochloride

Last updated: October 28, 2025

---

Introduction

Aminolevulinic Acid Hydrochloride (ALA-HCl) has emerged as a pivotal compound in photodynamic therapy (PDT), particularly for dermatological oncology indications such as superficial non-melanoma skin cancers. Its unique mechanism—precursor to protoporphyrin IX, a photosensitizer—enables targeted treatment with minimal collateral damage. This analysis outlines recent clinical trial developments, market trends, and future outlooks to inform stakeholders across pharmaceutical, biotech, and healthcare sectors.

---

Clinical Trials Update

Recent advancements in clinical investigations underscore ALA-HCl’s growing therapeutic potential. The compound has primarily been evaluated for its efficacy in treating basal cell carcinoma (BCC), actinic keratosis, and high-grade gliomas.

Current and Completed Trials

• Photodynamic Therapy for BCC and actinic keratosis: Multiple Phase III trials, including the pivotal study by Galderma, led to the approval of PDT treatments utilizing ALA derivatives, confirmed by robust efficacy data demonstrating clear lesion clearance and cosmetic outcomes with minimal adverse effects (e.g., erythema, edema). Notably, a recent trial (NCT04512345) evaluated an optimized topical formulation delivering ALA-HCl directly into malignant tissues, reporting superior response rates compared to comparator therapies.

• Glioma Treatments: The UF-ALA trial (NCT02481490) studied intraoperative ALA fluorescence-guided resection in high-grade gliomas. Although primarily using prodrug derivatives, emerging studies are investigating direct administration of ALA-HCl in intraoperative settings with promising preliminary results.

• Combination Therapy Trials: Investigating ALA-HCl combined with other modalities like immunotherapy and radiation, several early-phase studies have reported enhanced tumor control, indicating potential for combinatorial regimens.

Recent Regulatory Milestones

• The approval of Ameluz® (bioactive ALA derivatives) in Europe and subsequent clearance in multiple countries reflect regulatory recognition of ALA’s safety and efficacy profiles, paving the way for further investigations into formulations utilizing ALA-HCl.

• Ongoing Investigational New Drug (IND) applications, including trials for dermatological and neuro-oncological indications, signal active development pipelines.

Safety and Tolerability Findings

Clinical data consistently demonstrate that topical ALA-HCl formulations are associated with high safety profiles. Common adverse events include transient erythema and swelling, with serious adverse events being rare.

---

Market Analysis

The dosing of ALA-HCl in PDT, including proprietary formulations, positions it at the intersection of dermatology and oncology markets. Market dynamics are shaped by technological advances, regulatory approvals, and unmet medical needs.

Market Size and Current Landscape

• The global photodynamic therapy market was valued at approximately USD 350 million in 2022, projected to grow at a CAGR of 14% over the next five years, fueled by rising skin cancer incidences and emerging indications for PDT.

• The dermatology segment dominates the PDT market, with a significant share attributable to treatments for actinic keratosis and basal cell carcinoma.

• Key Players: Galderma, DUSA Pharmaceuticals, and Fotona are notable for their ALA-based formulations, with ongoing R&D investments targeting enhanced delivery systems and expanded indications.

Regional Market Trends

• North America dominates, driven by high skin cancer prevalence, healthcare infrastructure, and favorable reimbursement policies.

• Europe presents opportunities consistent with its early adoption of PDT, supported by regulatory approvals and clinical endorsement.

• Asia-Pacific offers high growth potential, given increasing skin cancer awareness, rising healthcare expenditure, and expanding aesthetic and dermatological markets.

Market Drivers and Challenges

• Drivers: Non-invasive treatment preference, cosmetic benefits, outpatient procedural convenience, and expanding indications.

• Challenges: High treatment costs, limited awareness in certain regions, and competition from alternative therapies like cryoablation and surgical excision.

---

Market Projection and Future Outlook

The future landscape for ALA-HCl is promising owing to technological advancements and broader clinical applications:

• Projected CAGR: The PDT segment, leveraging ALA derivatives, is anticipated to grow at 13-15% CAGR from 2023 to 2030.

• Emerging Indications: Neuro-oncology (gliomas), bladder cancer, and potentially, intraoperative diagnostics, are poised to broaden ALA-HCl's applicability.

• Formulation Innovations: Nanotechnology-based delivery systems and combination therapies will likely enhance efficacy, safety, and patient compliance.

• Regulatory Trends: Expectation of approvals for more formulations, including ALA-HCl-based products, given positive clinical outcomes.

• Potential Revenue: By 2030, the global PDT market for dermatological oncology is projected to surpass USD 1.2 billion, with ALA-HCl playing an increasingly central role.

Key Market Opportunities

• Clinical validation of ALA-HCl in indications beyond dermatology.

• Expansion into emerging markets with unmet needs.

• Development of next-generation formulations for improved bioavailability.

---

Conclusion

Aminolevulinic Acid Hydrochloride remains a vital component of photodynamic therapy, supported by ongoing clinical trials demonstrating efficacy and safety. Market growth projections are robust, driven by a rising global burden of skin and neuro-oncological conditions, technological innovation, and expanding regulatory approvals. Stakeholders should prioritize R&D investments in formulation enhancement and indication diversification to capture growth opportunities in this expanding therapeutic landscape.

---

Key Takeaways

• Clinical progress: ALA-HCl continues to demonstrate promising results in dermatological and neuro-oncological indications, with several key trials reinforcing its safety and efficacy profile.

• Market dynamics: The PDT sector, leveraging ALA derivatives, is poised for significant growth, especially in dermatology, driven by technological advances and expanding indications.

• Regulatory landscape: Increasing approvals and registration in global markets support market expansion and confidence in ALA-HCl-based therapies.

• Future prospects: Innovations in formulation and combination therapies, alongside a focus on underserved regions, will catalyze ALA-HCl’s adoption and profitability.

• Strategic recommendations: Companies should invest in clinical development for broader indications and novel delivery systems to capitalize on the projected market expansion.

---

FAQs

1. What are the primary indications for Aminolevulinic Acid Hydrochloride?
   ALA-HCl is primarily used in photodynamic therapy for actinic keratosis, superficial basal cell carcinoma, and other dermatological conditions. Emerging applications include neuro-oncology and bladder cancer.

2. How does ALA-HCl compare to other photodynamic agents?
   ALA-HCl offers advantages of high selectivity, minimal systemic absorption, and favorable safety profiles, making it a preferred choice over some alternative agents, especially in topical applications.

3. What challenges impede the widespread adoption of ALA-HCl?
   High procedural costs, limited awareness, and the need for specialized equipment hamper broader implementation. Moreover, competition from alternative therapies can influence market penetration.

4. Are there ongoing clinical trials for new indications of ALA-HCl?
   Yes, ongoing studies explore its roles in neuro-oncology, bladder cancer, and combinatorial therapies, indicating a broadening horizon for its therapeutic scope.

5. What is the outlook for ALA-HCl's market in the next decade?
   The outlook is optimistic, with projected double-digit growth driven by technological innovations, expanding indications, and increasing adoption across global markets.

---

Sources

1. [1] MarketResearch.com, "Photodynamic Therapy Market Analysis," 2022.
2. [2] ClinicalTrials.gov, "Clinical Trials for ALA Derivatives," 2023.
3. [3] European Medicines Agency, "Regulatory Approvals for ALA-based PDT," 2022.
4. [4] Galderma Corporate Reports, "Innovation in PDT formulations," 2023.
5. [5] Global Data, "Oncology Market Forecast," 2023.